List of Gene Therapy Companies in California - 202

4DMT is a clinical stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large market and rare diseases. Our vector discovery platform Therapeutic Vector Evolution harnesses the power of directed evolution, a Nobel Prize-winning technology, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors with improved therapeutic profiles compared to wild-type vectors. Our product design, development and manufacturing engine empowers us to efficiently create our valuable and diverse product pipeline. This combination of bold innovation and relentless execution gives 4DMT the capability to revolutionize genetic medicines and to strive for potential curative therapies. We have built a deep portfolio of genetic medicine product candidates, with five product candidates in clinical trials for seven patient populations. We are taking genetic medicine to new heights and into new disease areas to overcome challenges not addressed by existing medicines. This incredible challenge drives our relentless and talented team. Our people are the essence of our inspiring, innovative and collaborative environment that encourages individual contributions and provides opportunities for career development. We seek the best in the industry who identify with our mission and our bold and innovative results-driven approach. Our 4 Guiding Principles drive the way we work together: - Dare to Cure - Break Boundaries - Beyond Yourself - Prepare & Execute Relentlessly Join us in creating the future of genetic medicine.

High throughput engineering of cell lines to enhance the design and manufacture of next-generation therapeutics.

A2 is dedicated to pushing the boundaries of science to transform medicine and turn the tide on cancer. We are focused on the next frontier in cell therapy: solid tumors. A2 engineers T cells that target the loss of genetic material in tumors, enabling the selective killing of tumor cells while leaving normal cells unharmed. The company was founded in 2018 by Alexander Kamb, Michael Gallo and Paul Kang and has raised $136M since inception. Our investors include The Column Group, Vida Ventures, Samsara BioCapital, Nextech Invest, Casdin Capital, Euclidean Capital, UC Investments (Office of the Chief Investment Officer of the Regents) and Hartford HealthCare Endowment.

Driven by a bold founding vision: Empowering patients from within, Abintus is pioneering potentially curative, off-the-shelf genetic medicines that engineer cells directly inside the patient’s body, also termed in vivo. These therapies are based on Abintus' clinically proven Modular Delivery Platform (MDP). The company’s two initial, fit-for-purpose, In Vivo programs are: 1. Highly selective gene expression targeting cell type/state via synthetic biology (e.g., In Vivo CAR-NK/T/Treg, In Vivo TCR-T) with applications across multiple gene delivery systems (e.g., AAV, lenti, adeno, retro) and therapeutic areas including leukemia, solid tumors, autoimmune, infectious, and genetic disease. 2. Simultaneous engineering of a diverse repertoire of active innate and adaptive immune cells (e.g., In Vivo CAR-X) with applications in diseases such as lymphoma and solid tumors. Abintus is leveraging over 20 years and $275 million of prior technology advancement for in vivo engineering including the treatment of over 350 patients through Phase 3 registration trials. Our company name comes from the Latin words “ab intus”, which means “from within”. We chose this name for 3 reasons: 1) Patients: We are pursuing product candidates that are designed to directly empower patients’ own immune cells to fight their disease from within. 2) Science: Our team is committed to scientific excellence and continual learning. Our differentiated technologies have the potential to directly reprogram activated immune cells “in situ”, or from within. 3) Culture: We believe our ability to achieve our vision will come from within - by building a performance-driven culture that prioritizes career development and rewards outstanding contributions. Abintus’ initial funding syndicate included Takeda Ventures and The Leukemia & Lymphoma Society’s Therapy Acceleration Program® (LLS TAP). Abintus Bio, Inc. (“Abintus”) is headquartered in San Diego. www.abintusbio.com.

Acepodia is a clinical-stage biotechnology company developing innovative and highly effective cell therapies for cancer that are broadly accessible for patients. Leveraging its Antibody-Cell Conjugation (ACC) technology, the company links tumor-targeting antibodies to its proprietary immune effector cells, such as gamma delta T cells and natural killer (NK) cells to create novel antibody cell effector (ACE) therapies, which have increased binding strength against tumors that express low levels of tumor antigens. Acepodia is made up of seasoned leadership and scientific experts dedicated to advancing its robust pipeline of off-the-shelf ACE therapies with the potential to bring powerful, accessible cell therapies to a broad population of patients across a variety of solid tumors and hematologic cancers.

ADARx Pharmaceuticals is a newly launched next-wave genetic medicine company focusing on oligonucleotide therapeutics for inhibition, degradation and base editing of mRNA transcripts. With RNA as the target of our drug discovery, we are capable of working on a wide range of diseases and are currently focusing on genetic, cardiometabolic, and central nervous system (CNS) diseases. We are driven by our mission to turn cutting-edge science into life-saving therapeutics and our desire to provide hope for patients with intractable disease.

Adverum Biotechnologies, Inc., a clinical-stage gene therapy company, engages in developing gene therapy product candidates to treat ocular and rare diseases. Its pipeline of product candidates includes ADVM-022, an adeno- associated virus (AAV). 7m8-aflibercept for the treatment of wet age-related macular degeneration; ADVM-043, an investigational gene therapy candidate for the treatment of alpha-1 antitrypsin deficiency; and ADVM-053, a preclinical gene therapy product candidate for the treatment of hereditary angioedema. The company has collaboration agreements with Editas Medicine, Inc. to leverage its proprietary AAV vectors for genome editing technologies to treat up to five inherited retinal diseases; and Regeneron Pharmaceuticals, Inc. for the development of up to eight distinct ocular therapeutic targets. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was founded in 2006 and is headquartered in Redwood City, California.

Allogene Therapeutics, Inc., a clinical stage immuno-oncology company, develops and commercializes genetically engineered allogeneic T cell therapies for the treatment of cancer. The company is developing UCART19, an allogeneic chimeric antigen receptor (CAR) T cell product candidate, which is in Phase I clinical trials for the treatment of pediatric and adult patients with R/R CD19 positive B-cell ALL; ALLO-501, an anti-CD19 allogeneic CAR T cell product candidate that is in Phase I clinical trial for the treatment of R/R non-Hodgkin lymphoma; and ALLO-501A for the treatment R/R large B-cell lymphoma or transformed follicular lymphoma. It is also developing ALLO-715, an allogeneic CAR T cell product candidate that is in a Phase 1 clinical trial for treating R/R multiple myeloma; ALLO-819, an allogeneic CAR T cell product candidates for the treatment of acute myeloid leukemia; ALLO-647, an anti-CD52 monoclonal antibody; CD70 to treat renal cell cancer; and DLL3 for the treatment of small cell lung cancer and other aggressive neuroendocrine tumors. The company has license and collaboration agreements with Pfizer Inc.; Servier; Cellectis S.A.; and Notch Therapeutics Inc., as well as clinical trial collaboration agreement with SpringWorks Therapeutics, Inc. Allogene Therapeutics, Inc. also has a strategic collaboration agreement with The University of Texas MD Anderson Cancer Center for the preclinical and clinical investigation of AlloCAR T candidates across a portfolio of hematologic and solid tumors. The company was founded in 2017 and is headquartered in South San Francisco, California.

Alto Neuroscience is a clinical-stage biopharmaceutical company that integrates the biology of the patient into drug development to improve the lives of people with mental health conditions. Through its AI-enabled biomarker platform, Alto Neuroscience combines rich sources of information on patients’ brain activity and behavior to rapidly develop

Altos Labs is a biotechnology company focused on restoring cell health and resilience through cell rejuvenation to reverse disease, injury, and the disabilities that can occur throughout life. The company comprises a community of leading scientists, clinicians, and leaders from academia and industry working together towards this common mission. Altos operates in the San Francisco Bay Area, San Diego and Cambridge, UK. Note: Altos Labs will not ask you to download a messaging app for an interview or spend your own money to get started as an employee. If this sounds like your interaction with people claiming to be with Altos, it is not legitimate or a practice within our organization.

Appia Bio is an early stage biotechnology company based in Los Angeles, California. Founded in 2020, Appia Bio is focused on discovering and developing engineered allogeneic cell therapies across a broad array of indications with a scalable technology platform that increases access for patients. With its ACUA (Appia Cells Utilized for Allogeneic) technology platform, Appia Bio leverages the biology of lymphocyte development with chimeric antigen receptor (CAR) and T-cell receptor (TCR) gene engineering to generate CAR-engineered invariant natural killer T (CAR-iNKT) cell subtypes from hematopoietic stem cells (HSCs).

Applied Cells, Inc., was founded in 2018 in Silicon Valley to create revolutionary solutions to cell separation and enrichment products, which increase cell separation efficiently by changing how physics is employed in the process. We are supporting industry experts in Cell Therapy Development with MARS® immunomagnetic cell separation Platform. Our innovative, column-free, immunomagnetic cell iso

Advancing Gene and Cell Product Development Through iPSC and Genome Editing Technologies Applied StemCell strives to advance gene-editing and stem cell innovation for biomedical research and the biotechnology industry to assist in the development of breakthrough therapeutic approaches to cure intractable diseases. ASC is a trailblazing CRO/CDMO committed to building a complete iPSC-Gene Editing platform for the production of iPSC-derived products for gene and cell therapy research and development. With the integration of our unique iPSC approach and IP-based site-specific large cargo insertion technology, TARGATT™, we (1) develop immune-compatible master iPS cell lines and (2) deliver large DNA fragments to pluripotent cells that have proven differentiation capabilities. We stand at the forefront of the iPSC-based therapy revolution delivering research- and GMP-grade iPSC solutions along with various downstream assay services. Current Offerings: • iPSC Cell Line Models: iPSC generation, CRISPR & TARGATT™ genome editing, differentiation • Cell Gene Product CDMO: TARGATT™ iPSC Platform for allogeneic cell therapy products/GMP iPSC reprogramming, cell banking, gene editing, and differentiation • Bioproduction: TARGATT™ CHO cell system for antibody screening and bioproduction/TARGATT™ HEK293 cell system for library/antibody screening • Animal Models: Custom and off-the-shelf CRISPR & TARGATT™ mouse models For patent licensing, business development, and investment inquiries, please contact us at info@appliedstemcell.com.

Applied Viromics is a biotech company aiming to support early drug discovery and development activities. Applied Viromics specializes in custom-made recombinant viral vectors for efficient gene delivery both in vitro and in vivo.

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

ArsenalBiosciences, Inc., is a privately held, clinical stage programmable cell therapy company focused on the realization of solid tumor cell therapy to defeat cancer. Our discovery engine comprises precise CRISPR-enabled genome editing, integrated circuits incorporating logic gates for improved tumor targeting, and therapeutic enhancements enabling multiple pharmaceutical functions. With our programmable and computationally driven approach, we aim for enhanced and broader efficacy, increased patient safety, reduced provider costs and expanded market access. Our team is engineering living medicines to attack cancer’s inherent multi-faceted nature and bring dramatic improvements to the lives of patients.

Recombinant T Cell Receptor Ligands

At Artiva, our mission is to deliver to cancer patients highly effective cellular immunotherapies that are also safe and immediately accessible. Towards this end, Artiva is advancing a pipeline of off-the-shelf, allogeneic NK cell therapies, including CAR-NK cell therapies, that leverage the company’s unique manufacturing and engineering capabilities. Artiva is headquartered in San Diego.

The company supports cell and gene therapy manufacturing and process development projects from design through to clinical and commercial scale.

ASC Therapeutics focuses on the development of curative therapies for rare diseases. Our gene, CRISPR and cell therapies were developed at Applied StemCell, a life sciences company for over 12 years, including proprietary gene editing technologies. Our development pipeline includes several preclinical stage projects focusing on monogenic blood disorders that exhibit a high genetic penetrance and conditions requiring immunomodulatory interventions. Contact us for partnership opportunities

Aspa Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on developing a treatment for Canavan disease, a devastating inherited condition without an FDA-approved therapy. Aspa is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe and effective treatment for Canavan disease to patients as quickly as possible.

Aspen is developing induced-pluripotent stem cells (iPSCs) to address diseases with high unmet medical need, beginning with autologous neuron replacement for Parkinson’s disease (PD) and extending across the brain and affected organs.

Atara Biotherapeutics Inc., is a leading allogeneic T-cell immunotherapy company pioneering the development of transformative off-the-shelf therapies for patients with serious diseases including solid tumors, hematologic cancers, and autoimmune diseases.  With our lead program receiving marketing authorization in Europe, Atara is the most advanced allogeneic T-cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other serious diseases through incorporation of engineered CARs (chimeric antigen receptors) or TCRs (T-cell receptors). Atara is applying this one platform, which does not require TCR or HLA gene editing, to create a robust pipeline including: tab-cel for Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) and other EBV-driven diseases; ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation chimeric antigen receptor T-cell (CAR-T) immunotherapies for both solid tumors and hematologic malignancies.

Avellino turns genomic analytics into actionable healthcare insights, creating a healthier future for all. Powered by artificial intelligence and machine learning, its genetic discovery engine drives disruptive healthcare innovations. Avellino’s expertise spans a broad spectrum of healthcare specialties, including the impact of genetics in ophthalmology, oncology, pathogenomics and infectious diseases. As a global leader in leveraging artificial intelligence-powered algorithms, Avellino’s diagnostics provide eye care professionals with personalized genetic data to predict eye disease, prevent disease progression and ultimately preserve vision. Avellino has completed more than one million genetic eye tests since 2008, commercialized in the United States, South Korea and Japan. Its world class CLIA-certified laboratory in California has tested more than four million patients for COVID and RSV. Avellino’s research and development team has made groundbreaking discoveries leveraging genetic data, with programs in early detection of oncology, a polygenic risk analysis for glaucoma, and a siRNA gene-silencing therapeutic for the treatment of granular corneal dystrophy type II (currently in preclinical development). With a focus on health equity, Avellino prioritizes the inclusion of ethnically diverse populations in its studies to maximize the impact of its genomics breakthroughs for global healthcare. Avellino is headquartered in Silicon Valley, California, with operations in Korea, Japan, and the UK.

Aveni Foundation is a biotechnology company that focuses on expediting the development of gene-targeted therapies for cancer and Parkinson's disease.

Avid Bioservices (NASDAQ:CDMO), is a dedicated contract development and manufacturing organization (CDMO) focused on development and CGMP manufacturing of biologics. The company provides a comprehensive range of process development, CGMP clinical and commercial manufacturing services for the biotechnology, biopharmaceutical, and cell & gene therapy industries. With over 30 years of experience, Avid's services include CGMP clinical and commercial drug substance manufacturing, bulk packaging, release and stability testing and regulatory submissions support. For early-stage programs the company provides a variety of process development activities, including upstream and downstream development and optimization, analytical methods development, testing and characterization. The scope of our services ranges from standalone process development projects to full development and manufacturing programs through commercialization. Please be aware that the FBI recently issued a warning about an increased number of fake job postings for remote work employment that target applicants' personally identifiable information. All approved Avid Bioservices positions are accessible via the Avid Bio Careers page at https://avidbio.com/careers/.

The company providesservices inbioanalytic, molecular and cellbiology, in vitro and in vivo pharmacology.The company's capabilities include assay development and in vitro/in vivo pharmacologystudies forantibodies, small molecules, siRNAs, proteins, and gene therapy products.

Benitec is a biotechnology company developing a proprietary therapeutic technology platform that combines RNA interference (RNAi) with gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration. Benitec is using its technology, called DNA-directed RNA interference (ddRNAi) to develop a pipeline of product candidates for the treatment of several chronic and life-threatening human diseases , including oculopharyngeal muscular dystrophy (OPMD) and hepatitis B. By combining the specificity and gene silencing effect of RNAi with gene therapy, ddRNAi has the potential to produce long-lasting silencing of disease-causing genes from a single administration, which could minimize the requirement for patients to take regular doses of medicine. Benitec’s objective is to become the leader in discovering, developing, clinically validating and commercializing ddRNAi-based therapeutics for a range of human diseases with high unmet clinical need or large patient populations and, as a result, provide a better life for patients with these diseases.

We are a clinical-stage regenerative medicine company developing novel therapeutics for cardiovascular diseases with large unmet medical needs. BioCardia is developing proprietary comprehensive biotherapeutic solutions for cardiovascular disease in its CardiAMP™ and CardiALLO™ therapies. Clinical results supporting both therapeutic programs are compelling and build upon the most rigorous data in the field to date with intramyocardial delivery of marrow-derived cells. These programs are enabled by the Company's Helix™ transendocardial delivery systems and Morph® vascular access products, which are partnered to enable other promising biotherapeutic programs.

BioMap is an AI for life sciences company, unmasking nature’s rules to generate proteins for a wide range of therapeutic and industrial applications. We build super-scale Foundation models supported by access to unrivaled computational power through our strategic collaboration with Baidu, with whom we share our Founder and Chairman, Robin Li. We supplement our AI capabilities with a leading team of scientists, disease experts and cutting-edge high-throughput labs which generate large volumes of high quality proprietary data for our AI models. We partner with pharma, biotechs and industry to enable their discovery ambitions across a wide range of therapeutic and synthetic biology modalities through access to our AI technology platform, with a variety of flexible collaboration models. Our mission is to leverage AI to radically improve lives.

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

Biomonadic is an early stage techbio startup leveraging AI and IoT to resolve critical supply chain bottlenecks affecting the cell and gene therapy (CGT). They are creating a next generation AI empowered data platform for biotech manufacturing, leveraging reinforcement learning and LLMs to augment their capabilities.

BridgeBio Pharma, Inc. engages in the discovery, development, and delivery of various medicines for genetic diseases. The company has a pipeline of 30 development programs that include product candidates ranging from early discovery to late-stage development. Its products in development programs include BBP-265, a small molecule stabilizer of transthyretin, or TTR, that is in an ongoing Phase 3 clinical trial for the treatment of TTR amyloidosis-cardiomyopathy, or ATTR-CM; BBP-831, a small molecule selective FGFR1-3 inhibitor which is an ongoing Phase 2 clinical trial for the treatment of achondroplasia in pediatric patients; an AAV5 gene transfer product candidate for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD; and Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, an ongoing phase 2 proof-of-concept clinical trial for Autosomal Dominant Hypocalcemia Type 1, or ADH1. The company also engages in developing products for Mendelian, genetic dermatology, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; The Regents of the University of California; Leidos Biomedical Research, Inc.; Johns Hopkins University and University of Florida; University of Colorado Anschutz Medical Campus; Salk Institute for Biological Studies; Maze Therapeutics; and UC San Francisco. The company was founded in 2015 and is headquartered in Palo Alto, California.

Buck Institute for Research on Aging is a biomedical research institute focused on studying the aging process and its effects on health.

Calidi Biotherapeutics is a clinical-stage biopharmaceutical company revolutionizing oncolytic viral therapies with stem cell-based platforms.

California Institute of Technology - Caltech is an educational and research institution that focuses on science, technology, and engineering.

Founded in 1901, California Polytechnic State University (Cal Poly) is one of five comprehensive polytechnic universities in the U.S., with about 18,000 undergraduate and 900 graduate students. Located in San Luis Obispo, 200 miles north of Los Angeles, Cal Poly maintains a guiding philosophy of “Learn By Doing” that is reflected in the school’s comprehensive curriculum combining technical and professional training with the arts and humanities. When you graduate from Cal Poly, you won’t just know theory you will be able to think critically and know how to solve real-world problems. Today, Cal Poly is among the most selective public universities in the United States. Under President Armstrong’s leadership, Cal Poly’s national reputation is growing and, according to payscale.com, the return on investment for graduating students is consistently on a par with some of the most prestigious universities in California. Visit www.calpoly.edu to learn more. For Cal Poly Jobs, Visit: http://ow.ly/THgQc Awards & Recognition in 2014: - #1 Public-Master's University in the West by U.S. News. 22 consecutive years - #70 out of 1,500 colleges for "best overall value"​ - #13 among public schools for graduates'​ median salaries - #7 among public universities for return on investment - #4 in the nation for Aerospace & Computer Engineering - #5 in the nation for ROI, Orfalea College of Business - #1 in the nation for Industrial/Manufacturing Engineering - #3 in the nation for Civil Engineering - #1 in the nation for Architecture and Design - #4 Aerospace Engineering program in the nation for universities that do not offer a doctorate - Top 10 colleges for highest paid graduates in the field of Education

Welcome to the Cancer Center of Southern California. Contact us today to schedule an appointment with a leading oncology expert in Los Angeles!

Capricor Therapeutics, Inc., a biotechnology company, focuses on the development of transformative cell- and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases. The company’s lead candidate, CAP-1002, an allogeneic cardiac-derived cell therapy, which has completed phase II clinical trial for the treatment of patients with late-stage Duchenne muscular dystrophy (DMD); CAP-1002, which is in Phase II clinical trial for the treatment of cytokine storm associated with COVID-19; and completed various trials investigating the use of CAP-1002 for the treatment of cardiac conditions, including heart failure and post myocardial infarction with cardiac dysfunction. It is also developing CAP-2003 that is in pre-clinical development for the treatment of trauma related injuries and conditions; and two vaccine candidates, which are in preclinical stage for the potential prevention of COVID-19. Capricor collaborates with Lonza Houston, Inc. for the development of CAP-1002, its cell therapy candidate for the treatment of DMD and other indications. The company was founded in 2005 and is headquartered in Beverly Hills, California.

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology.

Capstan Therapeutics is a biotechnology company dedicated to advancing in vivo cell reprogramming. The core platform technology comprises proprietary targeted lipid nanoparticles conjugated with a recombinant protein binder such as monoclonal antibodies (tLNPs) designed to deliver payloads, including mRNA or gene editing tools, that can reprogram specific cell types in vivo. The platform technology has the potential to generate transformative therapies with applications across a broad range of disease areas, including oncology, autoimmune disorders, fibrosis, and monogenic blood disorders.

CARGO Therapeutics is engineering best-in-class CAR T-cell therapy to overcome resistance to cancer treatment and address barriers to access so that more patients may benefit from potentially curative therapies. CARGO is on a mission to outsmart cancer by advancing a new generation of best-in-class chimeric antigen receptor (CAR) T-cell therapies. Despite advances made by commercially available autologous CARs, these treatments are curative for fewer than half of all cancer patients. In addition, far too many patients are unable to access these potentially curative therapies due to other challenges, including manufacturing, supply constraints, slow turnaround time and reimbursement hurdles. The team at CARGO is intensely focused on pursuing novel solutions to achieve better cancer treatment outcomes and experiences.

We are an ISO 13485:2016 Certified Manufacturer developing innovative media formulations for cell cryopreservation, cell culture, and cell handling in cell therapy, gene therapy, and regenerative medicine. We deliver high-quality products with batch-to-batch consistency and rigorous quality controls from raw materials to final product release. We offer manufacturing services, scientific assistance in media formulation, and optimization of existing formulations to meet your specific needs.

• The Cedars-Sinai Biomanufacturing Center (CBC) is a new 25,000 square feet, state-of-the-art biomanufacturing facility located in West Hollywood, California. • Manufacturing the next generation of cell and gene therapies for human IND-enabling clinical trials. • cGMP cell manufacturing facility housing multiple cleanroom cell production suites and process development laboratories. • Combined expertise in a cGMP-compliant approach to manufacture cells at scale with innovative technologies and equipment. • Transferring laboratory protocols to cGMP, up-scaling of processes and achieving Food and Drug Administration standards to bring innovative cell therapy solutions to researchers and translational scientists. • World-class expertise in stem cell (iPSC) biology, gene editing and bioprocessing. • Supporting California Institute for Regenerative Medicine (CIRMN) programs, CBC is providing a much-needed cell biomanufacturing center in California

Cellastra Inc., a developer of disruptive gene therapies, is a private biotech company based in the San Francisco Financial District. It was founded and managed by proven industry executives with a track record of execution. Cellastra’s mission is to develop Disruptive, First or Best in Class Gene Therapies. Cellastra Proprietary Technology - Gene Vector- Anti-scarring peptide: Cellastra is developing viral gene vector-mediated intervention to address an area of great unmet medical needs in scarless wound healing after surgery and respiratory infections: 1. Surgery: Prevent excessive dermal scarring, e.g., after breast augmentation surgery, and C section. 2. Respiratory Infections: Prevent an excessive immune response, tissue damage, and scarring/fibrosis in high-risk patient groups with respiratory infections such as COVID-19 (coronavirus disease 2019) and RSV (respiratory syncytial virus). Scarless wound healing can be achieved by using a gene vector encoding for endogenous continuous production of the anti-scarring peptides at the site of injury. Thus, two gene vectors are planned for development (Cellexa applied under the skin after surgery and Virexa inhaled into the lungs in COVID-19 and other respiratory infections). The development of these vectors is built and expands upon a foundation of scientific proof of concept data of the anti-scarring peptide ensereptide.

CellFE is a cell engineering startup that enables development and manufacturing of the new generation of medicines – cell therapies. CellFE has developed a unique microfluidic platform to solve one of the most pressing issues of therapeutic cell engineering –efficiently delivering gene-editing molecules into cells while preserving cell viability. CellFE’s technology: • Enables development of previously inaccessible cell therapies by providing means for transporting hard-to-deliver molecules into the cells. • Accelerates commercialization of existing cell therapies by significantly reducing cost, complexity, and processing time, while improving the efficacy and safety of the cell engineering process.

CG Oncology, Inc. (Nasdaq: CGON), is a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients afflicted with bladder cancer. We see a world where urologic cancer patients can benefit from our innovative oncolytic immunotherapies to live with dignity and have an enhanced quality of life. Our lead candidate, cretostimogene grenadenorepvec, is an intravesically delivered oncolytic immunotherapy agent in a Phase 3 trial for the treatment of BCG-unresponsive non-muscle invasive bladder cancer. Cretostimogene grenadenorepvec is also in a Phase 2 study in combination with KEYTRUDA® (pembrolizumab) in the same indication. Other types of bladder cancer are being evaluated with cretostimogene grenadenorepvec in combination with OPDIVO® (nivolumab).

Chimera's mission is to reprogram life to end cancer, using therapies that restore dignity to patients. We do this with next generation, safer and more potent, CAR T therapeutics.

Cirsium Biosciences is democratizing gene therapy through transformative viral vector production methods and systems.

Coastar Therapeutics is a pre-clinical biotech company developing biological payload delivery technologies for cancer immunotherapy and gene therapies. Our proprietary ENHEnS technology can be used to coat biological payloads with cell membranes, which help them evade recognition and clearance by the immune system and be successfully delivered to tumor or other disease sites.

Correlia Biosystems develops innovative microscale tools that accelerate the rate of molecular interactions for rapid measurements of biomolecules. The customizable PIXI​ platform opens up new biological questions for interrogation from less than two microliters of sample, with rapid results that allow for dynamic monitoring of protein expression profiles.

CytomX Therapeutics, Inc. is committed to changing the treatment of cancer with our novel Probody® therapeutic platform. We have a broad pipeline comprised of five clinical-stage programs, with even more in development. Our commitment to transforming lives with safer, more effective therapies is driven by our curiosity and passion for innovation, and our belief that by acting with integrity in an honest, respectful, ethical manner, we have the power to change lives. Our workplace embodies collaboration, open communication, celebrating our successes and holding each other to the highest possible standards. CytomX embraces diversity and seeks to enhance and maintain our culture of equity and inclusion which encourages individuals to be themselves, feel involved, respected and connected. We at CytomX believe that we benefit from each other’s ideas and experiences. We accept our differences and learn from one another, improve together, and embrace change to achieve our corporate and individual objectives. This belief applies to how we work together at CytomX, how we work with others outside CytomX, how we represent CytomX in our communities, and how we endeavor to bring our scientific innovations to a diverse patient population. CytomX acknowledges the importance of diversity, equity and inclusion initiatives and that they require an evolving and ongoing commitment. CytomX is an equal opportunity employer and does not discriminate against any individual or potential candidate because of race, color, religion, sex, national origin, sexual preference or any other legally protected category. CytomX is located in South San Francisco, California, the birthplace of biotechnology. Learn more about us and how we are advancing science to treat cancer differently at www.cytomX.com.

DeciBio Consulting is a boutique strategy consulting firm focusing on the life science industry. DeciBio's mission is to provide strategic insights that accelerate innovation in precision medicine. Headquartered in Los Angeles, CA, DeciBio serves clients and customers across the globe, ranging from incubator-stage startups to Fortune 500 life science corporations. DeciBio addresses business solutions that range from market landscape analyses to full commercial strategies, including organic and inorganic growth opportunities (commercial due diligences). DeciBio’s work is typically supported by a mix of primary and secondary research leveraging Dexter, its in-house expert network, and DeciBio Analytics’ proprietary data intelligence products. DeciBio’s Analytics division offers numerous data products to support strategic insights, including in clinical diagnostics (the DxBooks), oncology biomarkers, liquid biopsy, NGS, spatial biology, omics market segmentation (Market Reports), the entire life sciences industry (BioTrack), and more. Internal teams such as the Culture Committee, Women* in Consulting, and Queers in Consulting enrich the employee experience. DeciBio’s Social Impact board and the DeciBio Impact Lab work collaboratively across divisions and externally to uproot discrimination and increase access to precision medicine. DeciBio Consulting, LLC recently launched DeciBio Ventures. This venture capital and creation firm invests in and supports ground-breaking companies in the precision medicine space.

Dendreon is a commercial-stage biopharmaceutical company and pioneer in the development of immunotherapy. Dendreon’s flagship product, PROVENGE (sipuleucel-T), was the first FDA-approved immunotherapy made from a patient’s own immune cells. More than 40,000 men with advanced prostate cancer have been prescribed PROVENGE in the U.S. since 2010. Dendreon also is evaluating the use of PROVENGE in early-stage prostate cancer, with the hope of curing more men of the disease. Dendreon is headquartered in Seal Beach, Calif.

Diagnostic testing for patients across all rare genetic diseases, including Cystic fibrosis, Fabry disease, Epilepsy, Hemophilia, all types of hereditary cancers, and cell and gene therapy.

DNARx is a company developing multiple therapeutics for major unmet medical needs, with demonstrated vaccine capabilities as well as protein expression. The company has been awarded a DARPA contract for up to $10.7 million to develop medical countermeasures against biological threats.

We are now at the dawn of the synthetic biology revolution, which will spawn new applications, markets, and business models, improving human lives in countless ways. This revolution will be driven by new technologies that enable DNA writing at speed and scale. To catalyze this revolution, Elegen is creating a suite of ground-breaking technologies to achieve DNA writing with unparalleled speed, cost, and quality. Elegen is unlocking the power to reprogram biology and create biologic solutions in order to solve some of humanity's greatest challenges. Founded and led by Dr. Matthew Hill and located in San Carlos, CA, Elegen is a stealth-mode company that is well capitalized by top life science investors, including Andreessen Horowitz, 8VC, and KdT. The company is advised and staffed by leading biotechnology scientists and entrepreneurs, including Dr. Marc Unger, inventor of the Nanoflex™ valve and former CSO of Fluidigm. Dr. Hill has a PhD from Stanford and a proven track record of advancing innovative technologies from invention to commercial success.

Elpida Therapeutics is a biopharmaceutical company that focuses on developing treatments for rare diseases, offering hope to children and families affected.

Encellin is a biotechnology company specializing in cell encapsulation technology for cell-based therapeutics. The company's initial pipeline focuses on endocrine disorders with a clear mechanism of action, particularly focusing on Hypoglycemia. Encellin was founded based on the technology developed in the lab of Dr. Tejal Desai at the University of California in San Francisco.

Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers potentially unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders.

Engage Bio is unleashing the potential of DNA medicines to eliminate disease. Our platform represents a new class of non-viral DNA medicines that are highly potent, non-immunogenic, durable, and ready to scale.

Epic Bio is a biotechnology company that focuses on biomedical research and genetics.

Eurekabio stands as a pioneering force in the realm of cell and gene therapy, serving as a leading upstream core supplier.Our primary focus lies in the research and development of pivotal biotechnology and autonomous equipment in the field of gene and cell therapy (CGT). By seamlessly integrating automation technology, artificial intelligence, and diverse disciplines with biotechnology, we have successfully addressed crucial technological challenges in gene therapy, cell therapy, life science, and pharmaceutical R&D. Through the synergies of cross-disciplinary collaboration and technological innovation, Eurekabio has consistently pushed the boundaries of therapy.Eurekabio has brought forth advanced cellular drug production solutions and the EuLV lentiviral vector platform. These breakthroughs, including high-titer stable lentiviral vector packaging and producer cell lines, are poised to play a pivotal role in the global commercialization of CGT drugs. Our ultimate aim is to bring tangible benefits to patients worldwide by ushering in a new era of advanced and accessible therapies.

Eureka Therapeutics is a privately held biotechnology company located in the San Francisco Bay Area, focused on breakthrough immunotherapies for the treatment of cancer. Utilizing our antibody drug discovery and engineering technologies, we are advancing safe and effective therapies for targeting previously inaccessible cancer antigens. We have built an early stage pipeline of innovative drugs, with the most advanced candidates at pre-clinical stage. Eureka Therapeutics is a leader in the discovery of novel antibody immunotherapies. We utilize a paradigm-changing technology platform to develop fully-human antibodies against intracellular cancer antigens. In collaboration with Memorial Sloan-Kettering Cancer Center, we were the first-to-demonstrate in vivo efficacy of a human IgG1 antibody against an intracellular cancer antigen. Our most advanced program is at preclinical stage and being developed in partnership with Novartis.

Excellos accelerates gene therapies by improving the quality, breadth, and variance of donor samples, and providing end-to-end cell therapy services: from customized collection to engineered working cell banks. Our proprietary Excellos 360 technology provides deep characterization of cells to better match patient and therapeutic development needs.

ExcepGen is a venture-backed, early stage biotechnology company located in San Francisco. The company is focused on solving fundamental underlying problems in drug discovery and applying those solutions to create a new generation of therapeutics.

Excision BioTherapeutics is a private company focused on developing advanced gene editing therapeutics to address the medical need for the treatment of life-threatening disease caused by viral infections. Our mission is to advance gene editing therapeutics for our indications into safe and efficacious medicines that will greatly improve the lives of infected individuals around the world.

Challenges with physical cell isolation limit adoption of single-cell sequencing. So we figured out how to prepare complete libraries within intact cells.

Fate Therapeutics (Nasdaq: FATE) is a clinical-stage biopharmaceutical company dedicated to the development of next-generation cellular immunotherapies for cancer and immune disorders.

A Biotechnology Research private company. Mission is to take on one of the largest problems in all of medicine – the leading cause of disability, chronic pain, and a significant risk factor for opioid addiction – osteoarthritis (OA).

Gene Biotherapeutics is a biotechnology company that focuses on the clinical development and commercialization of DNA-based biotherapeutics for the treatment of cardiovascular disease.

GenEdit is revolutionizing genetic medicines by developing innovative therapies through targeted delivery of genetic medicines.

GeneFab was formed in 2023 with a vision to combine industry leading expertise in synthetic biology with advanced cGMP capabilities to accelerate the development and commercialization of genetic medicines. GeneFab spins out from more than seven years at Senti Biosciences as the manufacturing and CMC arm of an innovative drug maker. Combining R&D collaboration, CDMO capabilities, drug development, and regulatory expertise is a key differentiator for GeneFab to support clients from early development to clinical and commercial manufacturing. The team is led by CEO Philip Lee who brings 20 years of experience as a biotech innovator and was most recently Co-Founder and CTO of Senti. GeneFab employs over 90 employees with a collective experience of 350+ years developing cell and gene therapies from discovery to commercialization.

Genlantis is a biotechnology company that develops innovative reagents for various research purposes including transfection, gene expression, primary cell culture, and proteomics.

GenVivo will make first- and best-in-class immune therapies that are immediately and easily deployable, highly effective, and which improve survival and quality of life beyond current therapies.

GigaMune is a therapeutics company developing a novel technology for in vivo, T cell targeted gene delivery of T cell receptors (TCRs) and chimeric antigen receptors (CARs) for serious diseases such as cancer and autoimmune diseases. Our initial goal is to challenge conventional ex vivo cell therapy manufacturing by reducing costs and improving outcomes. Our eventual goal is to deliver any gene to any cell in any tissue location at any time. See our high throughput immune screening technology highlighted in Nature Biotechnology (DOI: 10.1038/s41587-020-0438-y), and our petabase-scale protein search algorithm, published in Nature (DOI: 10.1038/s41586-021-04332-2).

Process, analyze, and annotate genomics and biomedical data at scale using containerized workflows.

Gritstone Oncology, Inc., an immuno-oncology company, engages in developing tumor-specific cancer immunotherapies to fight various cancer types. Its lead product candidate is GRANITE, which is in Phase I/II clinical trial for the treatment of solid tumors, including metastatic non-small cell lung cancer, as well as gastroesophageal, bladder and microsatellite stable, and colorectal cancers. The company is also developing SLATE, an off-the-shelf immunotherapy candidate which is in Phase I/2 clinical study for the treatment of common solid tumors, including metastatic non-small cell lung cancer, colorectal cancer, pancreatic cancer, and other mutation-positive tumors. Gritstone Oncology, Inc. has a strategic collaboration with bluebird bio, Inc. The company was founded in 2015 and is headquartered in Emeryville, California.

Develop instruments and methods for quantifying stability, purity, and quality in protein therapeutics and cell and gene therapy products.

HIBM (Hereditory Inclusion Body Myopathy) is a progressive and debilitating muscle wasting disorder caused by a gene defect. It touches those betwen the ages of 20 to 40 and, although progression is slow, it typically leads to total disability within 10-15 years. HIBM Research Group is laboratory funded byARM (the Advancement of Research for Myopathies,a leading supporter in research toward the cure).

ImmPACT Bio is dedicated to the discovery of transformative CAR-T cell therapies for cancer patients who have exhausted their treatment options. Our technology is designed to precisely distinguish cancerous cells from normal cells; thereby eliminating tumors without damaging normal tissues. Our goal is to eradicate tumors without generating the severe side effects that are difficult, sometimes impossible, for cancer patients to endure.

ImmuneBridge is developing allogeneic immunotherapies for cancer. Our core technology drives the expansion of cord blood derived immune stem cells and establishes a novel, flexible, consistent and abundant cellular source for immunotherapies. We are currently pursuing natural killer cell products for hematological malignancies. Next we will use the unique advantages of our cellular source to tackle solid tumors. Platform ImmuneBridge is creating a platform of scalable allogeneic immune cell therapies from cord blood. Our proprietary technology, including a novel small molecule, expands cord blood HSPCs over 100,000-fold while maintaining full immune multipotency, with the ability to differentiate into cell types including NK cells, T cells and macrophages. Pipeline In the near term, our goal is to validate the economic and clinical benefits of our expansion technology by taking an expanded-HSC-derived NK cell to clinical trials for hematological malignancies. In the long term, our unique access to the modified and unmodified immune tree allows us to tackle the challenge of solid tumors with combinations of modified cells. Agile Discovery This agile and flexible allogeneic platform reduces costs of discovery and manufacturing, expanding commercially viable indications for cellular immunotherapy. Starting from cord blood has speed and cost advantages over iPSCs, including the ability to quickly match HLA types to target populations or select beneficial genotypes without the need to edit those features into a clonal iPS line. This enables rapid iterations of abundant and consistent sources of therapeutic cells.

ImmunoVec is a biotechnology company based in Santa Monica, California, founded in 2019. The company specializes in developing gene therapy treatments for terminal genetic diseases that affect the immune system, particularly those related to the hematopoietic system, which includes blood and bone marrow disorders. ImmunoVec focuses on modifying or replacing genes to treat conditions such as hemic and lymphatic diseases. Their work targets specific genes like CYBB and CD34, aiming to provide life-saving therapies for patients with these genetic disorders. The company has raised $4.3 million in funding and has filed a patent application related to enhancers and vectors, indicating its commitment to advancing gene therapy technologies.

Founded by Jennifer Doudna, the Innovative Genomics Institute advances genome engineering to cure disease and ensure food security.

We combine synthetic biology and machine learning to control cellular stress responses for next-generation therapeutics.

At InvivoGen, we strive to provide life scientists worldwide with innovative, high quality and reliable reagents. As specialists in cell culture engineering and innate immunity, we are passionate about designing, developing, and bringing to market new tools and services for cutting-edge research. At our three regional headquarters — in Toulouse (France), San Diego (USA) and Hong Kong (China) — and through our dedicated multilingual customer support staff, we can provide researchers around the globe with the reagents and product information that they need to make the scientific breakthroughs of tomorrow. InvivoGen is a privately held company founded by university researchers in 1977 in Toulouse, France. Since then, we have gradually expanded with the opening of InvivoGen San Diego, in 1997, and InvivoGen Hong Kong, in 2014. Our unparalleled skills in microbial fermentation enable us to produce a wide range of bioactive molecules, including ultra-pure antibiotics, novel mycoplasma treatments and the largest available collection of pattern recognition receptor agonists, from various micro-organisms. Over 25% of InvivoGen’s employees hold a PhD in biology or chemistry. Through specialized R&D teams, they work together seamlessly to design, produce and validate novel plasmids, cell lines, vaccine adjuvants and synthetic ligands for research in areas as diverse as gene therapy, molecular immunology and vaccination. Moreover, based on our first-hand knowledge of innate immune receptor signaling pathways, we have devised an ever-expanding collection of reporter cell lines, including cytokine reporter cells and knockout (KO) cell lines for high-priority innate immune targets. Scientists depend on InvivoGen for quality, consistency and reliability. Whatever research challenges you are facing, count on InvivoGen to provide you with the tools and solutions that you need.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It is involved in developing neurology products that include Tominersen for Huntington’s diseases; Tofersen for amyotrophic lateral sclerosis (ALS); IONIS-MAPTRx for Alzheimer's diseases; IONIS-C9Rx for genetic form of ALS; ION859 for Parkinson’s disease; ION464 for multiple system atrophy and Parkinson’s disease; ION541 for amyotrophic lateral sclerosis; and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing products for cardiometabolic disease, such as IONIS-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; IONIS-APOCIII-LRx for familial chylomicronemia syndrome and TG- driven diseases; IONIS-APOCIII-LRx for treatment resistant hypertension; Pelacarsen for Lp(a)cardiovascular disease risk reduction; Vupanorsen for sHTG/CVDRR; IONIS-FXI-LRx for clotting disorders; ION449 for cardiovascular diseases; and IONIS-GCGRRx for diabetics. In addition, it develops IONIS-ENAC-2.5Rx for cystic fibrosis and chronic obstructive pulmonary disease; IONIS-PKK-LRx for hereditary angioedema; Danvatirsen for cancer; IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity; IONIS-HBVRx for hepatitis B virus; IONIS-AR-2.5Rx for prostate cancer; IONIS-FB-LRx for age-related macular degeneration and IgA nephropathy; and ION357 for retinitis pigmentosa. It has a collaboration agreement with AstraZeneca; GlaxoSmithKline plc; Janssen Biotech, Inc.; Bayer AG; Novartis AG; Pfizer, Inc.; Biogen Inc.; and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

Iovance Biotherapeutics (NASDAQ: IOVA) aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. We are adding to our talented team as we execute our commercial launch, advance our research and development efforts, and build our internal manufacturing capabilities. Iovance is headquartered in the San Francisco Bay Area. Our Iovance Cell Therapy Center (iCTC), a state-of-the-art manufacturing facility, is located in Philadelphia. We also have a research site in Tampa, Florida. Any information expressed by Iovance is subject to the risk factors and information on forward-looking statements contained in its filings with the Securities and Exchange Commission and available here: https://ir.iovance.com/sec-filings

FUJIFILM Irvine Scientific is a worldwide leader in the innovation and manufacture of cell culture media, reagents, and medical devices for researchers and clinicians. The company provides unrivalled service and quality to scientists working in cell therapy and regenerative medicine, assisted reproductive technology and cytogenetics, and industrial cell culture for the large-scale production of biotherapeutics and vaccines. FUJIFILM Irvine Scientific adheres to both ISO and FDA regulations and operates dual cGMP manufacturing facilities in California, USA and Tokyo, Japan. The company's consultative philosophy combined with expertise in cell culture and compliance provides customers with unique capabilities and support. For over 50 years FUJIFILM Irvine Scientific has remained uniquely flexible and focused on media while becoming a strategic global leader in media products and services.

Jasper Therapeutics is a biotechnology company focused on targeting mast cells and stem cells to treat chronic diseases. Their mission is to make a significant difference in the lives of patients suffering from these conditions.

Jaya Biosciences is an early stage life sciences company developing CNS-directed gene therapies for genetically defined neurodegeneration, including Alzheimer's, FTD and Parkinson's diseases.

jCyte is a clinical-stage company focused on the application of progenitor cell-based technology in retinal diseases. The Company's allogeneic product candidate, jCell, is delivered by simple intravitreal injection without the need for surgery or immune suppression. jCyte has completed a phase 1/2a, phase 1/2a extension and phase 2b clinical trials testing jCell in patients with Retinitis Pigmentosa.

Keck Graduate Institute is a graduate education institution focused on biotechnology, life sciences, healthcare, and research.

At Kite, a Gilead Company, our singular focus is cell therapy to treat and potentially cure cancer. Our goal is to bring the promise of cell therapy to as many eligible patients as possible who may benefit. Since 2009, Kite has been at the forefront of cancer immunotherapy. Today, we are proud to be the leader in engineered T cell therapy for hematologic malignancies, but we recognize that this is just the beginning. Patients are at the heart of our focus on the cure, which is why we are committed to pursuing cell therapy as a transformative treatment option for as many people with cancer as possible who may benefit. Cancer is personal to us. Unlike traditional pharmaceutical manufacturing, our therapies are not created on an assembly line. Cell therapy specialists at our manufacturing facilities reengineer a patient’s own immune cells to attack and fight their cancer cells — one patient at a time. That sense of connection to the people we serve motivates us daily at Kite. Our corporate headquarters are located in Santa Monica, California, and we have dedicated cell therapy manufacturing facilities in California, Maryland and the Netherlands. If you’re passionate about changing the future of cancer treatment, let’s talk.

Kriya Therapeutics, Inc. is a biopharmaceutical company developing gene therapies for diseases affecting millions of people around the world. With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $450 million to advance a broad pipeline of gene therapies for ophthalmology, neurology, and metabolic diseases.

Kriya Therapeutics, Inc. is a biopharmaceutical company developing gene therapies for diseases affecting millions of people around the world. With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $450 million to advance a broad pipeline of gene therapies for ophthalmology, neurology, and metabolic diseases.

Kyverna Therapeutics is a clinical-stage cell therapy company with the mission of engineering a new class of therapies for autoimmune and inflammatory diseases. The Kyverna therapeutic platform combines advanced T cell engineering and synthetic biology technologies to suppress and eliminate the autoreactive immune cells at the origin of autoimmune and inflammatory diseases. Kyverna’s pipeline includes KYV-101, a fully human, autologous chimeric antigen receptor (CAR) T-cell therapy with properties well suited for use in B cell-driven autoimmune diseases and KYV-201, a fully human, allogeneic CAR T-cell therapy. By offering more than one mechanism for taming autoimmunity, and with patients currently in treatment for multiple indications in the U.S. and Europe, Kyverna is positioned to act on its mission of transforming how autoimmune diseases are treated.

LevitasBio is a premier end-to-end sample processing and analysis provider to the life sciences market. Our advanced LeviCell system is a groundbreaking cellular research and analysis tool for scientists, researchers, and labs. For the first time ever, researchers can examine, analyze and understand true biological signatures without altering, stressing, or damaging cells—all while preserving the original samples. LeviCell delivers true biology for real cellular insights to power cellular research.

Life-saving therapies. Critical diagnostics. Breakthrough clinical trials. Life Couriers places hope in the hands of patients everywhere by being the world's most trusted logistics partner for crucial pharmaceutical and medical products. We are the worldwide market leader for radiopharmaceutical and stem cell logistics. We have taken this experience with the most time-critical shipments in the world to expand our services to biopharmaceuticals, cell and gene therapies, lab specimens, clinical trials, organ transplant, medical devices, and more. We understand a fundamental reality - that missing a delivery can mean losing a life. That is why we have intentionally curated our culture to be solely focused on patient outcomes. Our people care more and they are deeply committed to enhancing, enriching, and saving lives through the power of logistics. We are Life Couriers. Logistics for Life.

Ligandal is a genetic medicine company that uses nanotechnology to develop targeted and personalised therapies. As a regenerative medicine biotechnology company, Ligandal develops ultra-rapid therapeutics and vaccines for outbreaks and pandemics, in addition to its work in immuno-oncology and rare genetic diseases. Our current focus is on delivering a peptide-based cure to the COVID-19 pandemic (SARS-CoV-2 coronavirus), as part of a broader mission of enabling personalized medicine on a global scale through the power of nanomedicine and gene therapies. Given that increasingly complex disease states require precise and custom-tailored medicine, much of our technology development has focused on combining gene therapy with targeting peptides to enable completely new paradigms in evolutionary medicine. Currently, we are utilizing a peptide nanoscaffold based approach for treating COVID-19 without a gene therapy component, with the goal of creating a combined antidote-vaccine. My expertise lies in targeted gene delivery, nano- and biomaterials, computational modeling of peptides, binding simulations, immunoengineering, as well as CRISPR and TALEN based gene editing. At Ligandal, we have demonstrated peptide-based delivery of CRISPR, RNA, DNA, siRNA, and a range of genes or proteins to virtually any cell type.

Lyell Immunopharma, Inc., a T cell reprogramming company, engages in developing T cell therapies for patients with solid tumors. The company develops therapies using technology platforms, such as Gen-R, an ex vivo genetic reprogramming technology to overcome T cell exhaustion; and Epi-R, an ex vivo epigenetic reprogramming technology to generate population of T cells with durable stemness. Its pipeline includes LYL797, a T cell product candidate for the treatment of non-small cell lung cancer and triple negative breast cancers; LYL845 for multiple solid tumors; and NY-ESO-1 for synovial sarcoma and other solid tumor indications. Lyell Immunopharma, Inc. was incorporated in 2018 and is headquartered in South San Francisco, California.

The comapanies' research and development efforts are focused exclusively on rare genetic neurodevelopmental disorders, a group of serious diseases with few or no treatment options.

Mammoth Biosciences is a biotechnology company focused on leveraging its proprietary ultracompact CRISPR systems to develop long-term curative therapies, as well as other applications such as decentralized precision diagnostics. Founded by CRISPR pioneer and Nobel laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington, the company’s ultracompact proteins are designed to enable in vivo gene editing in difficult to reach tissues utilizing both nuclease applications and new editing modalities beyond double stranded breaks, including base editing, gene writing, and epigenetic editing. The company is building out its pipeline of potential in vivo gene editing therapeutics and capabilities and has partnerships with leading pharmaceutical and biotechnology companies to broaden the reach of its innovative and proprietary technology. Mammoth’s deep science and industry experience, along with a robust and differentiated intellectual property portfolio, have enabled the company to further its mission to transform the lives of patients and deliver on the full promise of CRISPR technologies.

Mana.bio is an AI based drug delivery startup, creating a platform for Lipid Nanoparticle (LNP) development for oligonucleotide therapies including mRNA-based therapeutics, vaccines and gene therapy.

Marin Biologic blends the fields of cell biology, immunology, molecular biology and biochemistry to tackle complex projects in an innovative and timely manner. We simultaneously utilize our in-depth scientific expertise to collectively approach projects to complement our client’s approach and meet the client’s needs. Marin Biologic is a woman PhD owned and managed Contract Research Laboratory which specializes in the research, pre-clinical and clinical development and validation of cell based assays and immunoassays for biotech and pharmaceutical companies in Phase I/II or Phase III stages. Our unique approach to collaboration encourages scientists to talk to scientists to create better science. We are compliant with U.S. FDA cGMP/GLP standards as well as ICH for all levels of clinical development. We employ these standards that are phase appropriate for your clinical development or research. CEO and President Tania Weiss PhD founded Marin Biologic Laboratories in 1995 to be able to apply her enthusiasm for good scientific process to a broad array of client projects. As a result, the client list as well as the project list is very diverse over the history of the Company. Marin Biologic seeks out and attracts interesting and different scientific opportunities, which puts them at the forefront of scientific development. Our Lab is located in Novato, California, halfway between San Francisco and the Napa and Sonoma Valleys. Peter Ralph. PhD, VP Science, peter@marinbio.com Tania Weiss, PhD, CEO, tania@marinbio.com 415 883-8000 Visit: www.marinbio.com to connect with us, or ask us a question.

Matrix Biomed, Inc. is a biopharmaceutical company focused on delivering gene-based therapeutics. Gene-based therapeutics are directed to address both the dysregulation of multiple genes as well as their interactions in gene regulatory networks. Only by addressing the dysregulation of multiple genes and their interactions in gene regulatory networks, Matrix Biomed, Inc. is able to deliver a therapy designed to truly revert a disease state to healthy state. Currently, Matrix Biomed, Inc. gene-based therapeutics are directed to therapies and drug products to make cancer treatment more manageable and successful through the elimination of the dose-limiting, life-threatening, and treatment-inhibiting toxicities associated with chemotherapy and radiotherapy while targeting the master genes responsible for cancer growth and development, hypoxia inducible factor -1 (HIF-1) and hypoxia inducible factor-2 (HIF2).

Maze Therapeutics is translating novel genetic insights into lifesaving medicines, through an approach that integrates human genetics and functional genomics. Advances in these areas have enabled us to decode the mysteries of genetic modifiers in a range of severe diseases, which we aim to turn into medicines that mimic protective mutations. We have assembled a world-class team that we need to realize this vision, including creative scientific pioneers in genetics, experienced company builders, and biotech industry leaders.

Gene editing is changing the world. Founded by pioneers in the CRISPR/CAS revolution, Metagenomi is discovering the next generation of gene-editing systems for use in treating genetic diseases.

Minaris Regenerative Medicine is a global contract development and manufacturing organization for the production of cell and gene therapy products. We offer our clients clinical and commercial manufacturing services, development solutions, and technologies. With more than 20 years’ experience in the field of regenerative medicine, we are trusted partner ensuring scalability, delivering high quality results, and achieving cost effective manufacturing. Our state-of-the-art facilities in the United States, Europe, and Asia, as well as our committed employees, enable us to supply patients worldwide with lifechanging therapies. At Minaris, we are creating cell therapy miracles together. Minaris Regenerative Medicine is wholly owned by the Resonac Group.

Minerva specializes in quality investigations, product development, technology transfer and productivity improvements services for the cell and gene therapy, biotech and medical device industries, using advanced data, knowledge and risk management. Our team of interdisciplinary professionals supply us and our clients with cutting-edge knowledge while keeping our rates very competitive. Our team includes Quality Investigators, Engineers, Data Analysts, Information Scientists, and Biotechnology professionals. We also have a network of advisors in Quality Investigations, Life Sciences, Data Sciences, and Engineering. We tailor our services according to our client's location and budget needs: • Full-time Remote Work with On-Location Manager • Part-time Remote work with partial On-Location • Virtual Person-to-Person Advisory Services • Full-time On-Location • Other arrangements as needed Minerva uses process characterization and data structuring to help companies grow through continuous improvement and effective change management. Our data-driven and risk-based reports on product development and sustaining are fast, reliable, and compliant, and designed to facilitate your analysis and decision-making. We are best known for our agile implementation of databases and information management systems for tracking and visibility of medical device development, drug development and approval, methods development, sample testing, and operations sustaining. Let Minerva help your company achieve its development, operations and quality objectives through our services in Knowledge Management, Data Management, Risk Management, and Language Management.

We're Mirai Bio, biotech's first open end-to-end development platform company for co-creation of optimized #GeneticMedicines. Learn more at MiraiBio.com.

Mitera is a maternal-fetal medicine (MFM) telehealth company. We help inform and educate our patients about their pregnancy-related risks and options through obstetrical screenings and at-home genetic testing kits, empowering them along their family-forming journey.

Moonwalk Biosciences is an early-stage therapeutics company co-founded by Dr. Feng Zhang and leading experts in cell state epigenetics including Alex Aravanis, the former CTO of Illumina. The company has developed a differentiated approach to mapping the epigenome of cellular states at the whole genome, single cell resolution level, as well as proprietary strategies for precise epigenetic engineering. The combination enables both a novel discovery platform and a path to developing therapeutics that include both epigenetic engineering and other traditional modalities.

Biotech startup developing cutting edge therapies for muscle diseases

We aspire to be the leading healthcare company focused on redefining care for women’s health and prostate cancer. Our experienced team brings significant breadth and depth of expertise in drug development, combined with a commitment to develop new treatments for women suffering from uterine fibroids, endometriosis, and infertility, and men suffering from prostate cancer. We strive to improve the lives of millions of people, while building an innovative, dynamic, and collaborative company where employees thrive.

We have developed a patented, non-permanent gene therapy to target pain that is non-addictive, highly specific and long-lasting. So how does this work? We were inspired by nature: there are humans that have a mutation in their genome that feel no pain whatsoever. We have imitated this process by utilizing a novel gene therapy to target pain in a non-permanent way. In our proof of concept, we have demonstrated that the mice that received our therapy have an increased pain tolerance and lower pain levels. Importantly, mice are not numb and can continue feeling mechanical stimulation, such as touch.

Neurona is a biotherapeutics company developing neural cell therapies with single-dose curative potential for the treatment of chronic neurological disorders. Based on a novel neural cell lineage pioneered by the company’s scientific founders, Neurona has built a robust regenerative platform and is developing neuronal, glial, and gene-edited cell therapy candidates that provide long-term repair of dysfunctional neural networks for multiple neurological disorders.

Nkarta is a clinical-stage biotechnology company advancing the development of allogeneic natural killer (NK) cell therapies. By combining its cell expansion and cryopreservation platform with proprietary cell engineering technologies, Nkarta is building a pipeline of cell therapy candidates generated by efficient manufacturing processes, and engineered to enhance tumor targeting and improve persistence for sustained activity in the body. For more information, please visit the company’s website at www.nkartatx.com.

Nucelis is a provider of ingredient solutions for personal care, nutrition, and flavor and fragrance markets, emphasizing high-value, performance-focused, and sustainably-sourced products.

Nucleus Biologics is a San Diego based biotechnology company and a global provider of media and supplements for the cell and gene therapy space.  Their mission is to speed the time from scientific discovery to cure by driving reproducibility through Precision Cell Culture products and tools. Nucleus Biologics delivers innovative, transparent and cGMP products and services with the goal of disrupting the market and eliminating antiquated practices and products. Ultimately, Nucleus Biologics strives to create a new paradigm that serves both scientists and clinicians, while reducing the environmental footprint of cell culture.

Our mission is to develop effective and affordable preventive vaccines for Alzheimer’s disease, Parkinson’s disease, and other neurodegenerative disorders. Using our universal vaccine platform for Alzheimer’s, our goal is to induce antibody titers high enough to penetrate the blood-brain barrier and prevent pathological plaques, fibrils, and oligomers to delay disease onset in cognitively unimpaired people at risk of Alzheimer’s.

Nurix Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of small molecule therapies for the treatment of cancer and immune disorders. The company develops NX-2127, an orally available Bruton’s tyrosine kinase (BTK) degrader with immunomodulatory drug (IMiD) activity for the treatment of relapsed or refractory B-cell malignancies; NX-5948, an orally bioavailable BTK degrader without IMiD activity for the treatment of relapsed or refractory B-cell malignancies; and NX-1607, an orally available Casitas B-lineage lymphoma proto-oncogene-B (CBL-B) inhibitor for immuno-oncology indications. It also develops NX-0255, a CBL-B inhibitor for ex vivo use to enhance adoptive T-cell therapy; DeTIL-0255 for tumor infiltrating lymphocytes therapy; KINASE-CTM3 to treat T cell malignancies and autoimmune diseases; and LIGASE-INH2 for immuno-oncology. In addition, the company develops programs, such as COVID-CTM1, COVID-CTM2, and COVID-CTM3 that are designed for protein degradation to SARs CoV2 targets. Nurix Therapeutics, Inc. has a strategic collaboration agreement with Gilead Sciences, Inc. for cancer and other challenging diseases patients; and Sanofi S.A. The company was formerly known as Nurix Inc. and changed its name to Nurix Therapeutics, Inc. in October 2018. Nurix Therapeutics, Inc. was incorporated in 2009 and is headquartered in San Francisco, California.

NysnoBio ia dedicated to developing the therapeutic potential of the Parkin protein for patients with serious medical conditions. We envision a future where the power of the Parkin pathway is directed toward specific disease areas, where the activation of the repair pathway can have maximum therapeutic benefits. Our core team of industry professionals and network of academic experts are working together to deliver on the scope of therapeutic potential for Parkin cellular repair.

Oculogenex is a biopharmaceutical company founded and directed by an ophthalmologist retinal surgeon with patients in mind. It was founded out of passion and frustration shared with patients losing vision due to lack of therapeutic options. We have professional and scientific experience with retinal diseases, genetic variations in retinal and optic nerve diseases. We have built a team of experts in animal models of macular degeneration, antioxidant therapies for macular degeneration, medical and surgical treatments of retinal diseases and drug delivery methods. This specialized team has been built to utilize genetic engineering technology to improve vision for patients with degenerative eye diseases.

Building a Healthier Future Our mission is to improve the health, vitality, and longevity of human beings through bioengineering. Who We Are At Ossium, we believe that science is humanity’s best weapon in the fight against disease, and we embrace the challenge of mobilizing the world’s healthcare ecosystem to win that fight. In pursuit of this goal, we empower our employees, maintain the highest standards of excellence, and are a force for good. PRINCIPLES The values we live by Impact We are mission-driven and results-oriented. We believe that a healthier world is a better one and that our work should drive positive change. Truth Embracing the truth empowers us to learn from both our successes and our mistakes. We follow the data wherever it leads. Ownership We hold ourselves and each other to the highest bar. We care deeply about the work we do and know that even the smallest details can make a difference.

At PacBio, we’re devoted to empowering you. Pioneering the future through biology takes vision. You have to look beyond the status quo to what’s possible. We know your vision can reveal the full potential of the genome, advance human health and the health of our planet. And that is why your vision and pursuit are at the very center of what our company does — our product design, our customer support model, our partnerships, and even our identity. We create the world’s most advanced sequencing systems to provide you the most complete and accurate view of genomes, transcriptomes, and epigenomes. Our founders invented a new way to study the synthesis and regulation of DNA, RNA, and proteins. Driven by a desire to advance human health, we’re harnessing advances in biochemistry, optics, nanofabrication, and more. We are passionate about developing products that empower scientists to explore the full spectrum of genetic variation in any organism — from unraveling the mystery of rare diseases to improving the world’s food supply. With world-class service and support, we keep your research progressing so you can focus on finding answers. We encourage you to see the whole picture — and consider us the partner you trust to see it too.

A start-up developing novel ophthalmic therapeutics to treat major unmet needs for patients.

PhorMed is a biopharmaceutical company leading the charge in the field of genomic medicine. We are working to utilize gene therapy to treat diseases, allowing the body to heal, and give new hope to patients suffering from diseases like AML, Hodgkin’s Lymphoma, Parkinson’s disease, and viruses.

Our mission at Poseida Therapeutics is to create cell and gene therapies with the capacity to cure. We are developing novel products using our broad gene engineering platform technologies, with the goal of bringing new and better treatments to patients with serious diseases, first in the blood-based cancer Multiple Myeloma and next in solid tumors like prostate cancer.

Profluent is an AI-first protein design company. Founded in 2022, we develop deep generative models to design and validate novel, functional proteins to revolutionize biomedicine. Based in Emeryville, CA, we are backed by leading investors including Spark Capital, Insight Partners, Air Street Capital, AIX Ventures, and Convergent Ventures. To learn more about our mission to decode the language of life with AI, visit profluent.bio.

PYC Therapeutics (ASX: PYC) is a clinical stage biotechnology company pioneering a new generation of RNA therapeutics for genetic diseases. It works by combining two complementary platforms: RNA-based drug design and PYC’s proprietary drug delivery technology. PYC's library of naturally derived cell penetrating peptides provide its delivery platform to overcome the major challenges of RNA therapeutics - getting into the target cell. PYC believes its PPMO (Peptide conjugated Phosphorodiamidate Morpholino Oligomer) technology enables a safer and more effective RNA therapeutic to address the underlying drivers of a range of genetic diseases for which no treatment solutions exist today. The Company is leveraging its leading-edge science to develop a pipeline of novel therapies including two programs focused on inherited eye diseases and preclinical discovery efforts focused on neurodegenerative diseases. PYC’s discovery and laboratory operations are located in Australia, and the Company’s preclinical and clinical operations are based in San Fransisco California.

Ranked as one of the 10 best children’s hospitals in the nation by U.S. News and World Report in 2024, Rady Children’s Hospital includes a 511-bed pediatric hospital that serves as the largest provider of comprehensive pediatric medical services in San Diego, southern Riverside and Imperial counties. With more than 40 locations, Rady Children’s is the only health system in the San Diego area dedicated exclusively to pediatric health care and is the region’s only designated pediatric trauma center. Rady Children’s is a nonprofit organization that relies on donations to support its mission. For more information, visit rchsd.org.

Rampart Bioscience is evolving a new class of medicines for patients with genetically driven diseases. They integrate capabilities from a variety of key sources to develop gene medicines to provide long-lasting treatments for a wide range of diseases.

Ray Therapeutics Inc (RayTx) is a late-stage preclinical Bay Area biotechnology company pioneering the first-to-market precision optogenetic therapy for vision restoration that is highly active in ambient lighting (and will not require light-enhancing goggles). Our studies have shown for the first time that our proprietary ChRown optogenetic protein can restore high levels of visual acuity, contrast sensitivity and visual field. RayTx lead asset, RTx-015, is expected to address the huge unmet medical in people suffering from many forms of retinal blindness (independent of the underlying genetic cause), including those with advanced age-related macular degeneration. RayTx has partnered with a world-leading AAV gene therapy manufacturer is utilizing their proprietary commercial manufacturing process and analytics to maximally de-risk clinical development. RayTx anticipates clinical trials will begin by the end of 2023 in our initial indication, retinitis pigmentosa.

Regulus Therapeutics Inc. (Nasdaq:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus is located in San Diego, California and is led by a seasoned executive team experienced in corporate management, business, science, drug discovery and development. Regulus'​ scientific advisory board consists of world-class scientists and some of the foremost authorities in the field of microRNA research.

ReIGNITE is a biotechnology company using Directed Evolution to develop systemic high-capacity viral vector immunotherapies and gene therapies. Our unique platform leverages the Nobel Prize-winning technologies of Directed Evolution to develop customized high-capacity vectors with tissue-specific tropism.

Rejuvenate Bio is targeting the core drivers of chronic age-related diseases by unlocking the power of gene expression and epigenetic reprogramming to reverse pre-existing heart disease, metabolic disease, and kidney failure in humans.

Rejuvenation Technologies is a biotechnology company that developed nucleoside-modified TERT mRNA to safely and rapidly extend telomeres. The company is focused on developing mRNA therapies to extend the healthspan by preventing and treating chronic age-related diseases.

Renovaro Biosciences, Inc. is a biotechnology company committed to developing advanced allogenic cell and gene therapies to promote stronger immune system responses potentially for long-term or life-long cancer remission in some of the deadliest cancers, and potentially to treat or cure serious infectious diseases such as HIV and Hepatitis B virus (HBV) infection.

Founded by pioneers in biopharmaceuticals, Renova Therapeutics is developing a portfolio of definitive gene and peptide therapies to restore health to people suffering from cardiovascular and metabolic diseases. Our investigational therapies are based on carefully validated and proprietary research into disease mechanisms and the founders’ nearly 50 combined years of work in the gene therapy field. This experience enables us to generate an entirely original platform of therapeutics and delivery systems that in a single dose are designed to provide durable, life-long treatments for chronic diseases. Our scientific research, led by Dr. H. Kirk Hammond, appears in numerous peer-reviewed articles that establish the scientific rationale and evidence supporting the development of our pipeline and our novel approach to delivering gene therapies. The first indications the company is pursuing are gene therapy treatments for congestive heart failure (CHF) and type 2 diabetes, two of the most common and devastating chronic diseases in the world. The company’s lead product, RT-100, is a treatment that delivers a therapeutic gene directly to the heart during a routine outpatient procedure and has the potential to increase heart function in millions of patients with CHF. The Renova Therapeutics pipeline also includes a groundbreaking gene therapy in preclinical stage for sufferers of type 2 diabetes, as well as a peptide infusion therapy for the treatment of acute decompensated heart failure. OUR MISSION: Creating transformational gene and peptide therapies that treat the most prevalent diseases to restore health and renew life OUR VISION: A future in which we are able to permanently treat chronic diseases with gene and peptide therapies

Replay is a genome writing company, which aims to define the future of genomic medicine through reprogramming biology by writing and reprogramming biology.

Replicate Bioscience is amplifying the power of RNA therapeutics by pioneering novel self-replicating RNA (srRNA) technology. Replicate’s off-the-shelf srRNAs contain two components: virally derived genetic code to drive controlled and self-limiting amplification, and the RNA encoding therapeutic proteins. Our library of viral vectors, selected for driving robust and sustained protein expression and orders-of-magnitude improved performance over linear mRNA, allow for the development of treatments in applications including oncology, infectious disease, and autoimmunity. Differentiated by a team of srRNA experts, a customizable library of synthetic srRNA vectors, and end-to-end development capabilities, Replicate is uniquely positioned to finally expand the reach of RNA treatments toward widespread use in infectious disease, immuno-oncology, autoimmune disease, and more.

Although ophthalmic medicine is progressing day by day, there are still some diseases neither treatment nor prevention methods has not been established yet. Ophthalmologists including me cannot do anything with such disease but just watching the patient go blind. As an ophthalmologist, I am struggling about the current limitation in ophthalmology and trying to overcome　this problem. There are more than 300,000 visually impaired patients in Japan, and the social cost is estimated to be more than 8 trillion yen, most of which are due to intractable diseases for which prevention nor treatment method has not yet been established. Please close your eyes. Can you imagine what the patients feel like losing their sight? It is said that vision occupies 80% of information that people obtain from the outside. Their quality of life will suffer considerably with blindness. In order to overcome these blindness diseases, we have established Restore Vision from Keio University School of Medicine to develop treatments for these intractable eye diseases using the latest technology. We will do our best for a bright future.

Aspiring to add 10 years to healthy human lifespan, starting with cellular reprogramming, autophagy, & plasma-inspired therapeutics

Launching Q1 2025. Sagittarius Bio is creating a new hope for immunological and inflammatory diseases and cancer.

Salk Institute for Biological Studies is a research institute focused on advancing biomedical studies and scientific research.

SanBio, Inc. - Founded in February 2001 SanBio's scope of business is development, production and sales of regenerative cell medicines. Our mission is to develop regenerative medicines, creating benefits for patients and value for stakeholders. Our vision is to achieve global leadership in the regenerative medicine field using best science and technology, building global revenues and sponsoring successful clinical trials. SanBio focuses on using regenerative cell medicine to treat the disabilies caused by diseases of the central nervous system. Most of these conditions currently have no effective medicinal treatment, and those treatment that do exist generally address disease symptoms and not the underlying cause of the disease. Such conditions afflict a vast number of patients worldwide.As of January, 2016, SanBio had introduced a product for the treatment of chronic motor impairment from ischemic stroke and traumatic brain injury into clinical development in both Japan and the US. In the future, SanBio plans to continue expanding the indications and geographical regions for its products, using both internal development, as well as partnerships and licenses.

Sangamo Therapeutics is a genomic medicine company conducting research & development across four distinct but complementary technology platforms – gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The optionality and diversity inherent to Sangamo’s technology platform enables us to design therapeutic approaches to resolve the underlying genetic causes of disease, using whichever technology is best suited to deliver that treatment. Because Sangamo’s technology platforms are related, we are applying learnings across studies to optimize each individual platform and uncover research and development paths forward. Sangamo is best known for developing our proprietary gene editing technology, zinc finger nucleases (ZFNs). Our technology team continues to optimize ZFNs for the three key dimensions of genome editing: precision, efficiency, and specificity. Ex vivo gene-edited cell therapy is the most straightforward application of ZFN technology. It builds on early experience with HIV clinical studies and is applied to hematological disorders and oncology. Our cell therapy portfolio also includes ex vivo engineered regulatory T-cells (CAR-Tregs) for application in autoimmunity and inflammation. The insights gained about molecular biology, cassette engineering, and delivery through in vivo application of ZFNs led to our gene therapy program using AAV, which is advancing in the clinic. Zinc finger protein engineering and AAV delivery are also foundational for our zinc finger protein-transcription factor (ZFP-TF) genome regulation technology, which we are applying in CNS diseases. With our robust history of genomic medicine firsts, unmatched technology platform, and growing manufacturing and clinical development capabilities, Sangamo believes we are the company to realize the vision of genomic medicine.

Scribe is building and applying a suite of CRISPR technologies designed for therapeutic use into a single continually evolving and expanding genetic modification platform. Specifically, the company is working on NK cell therapies.

Our mission is to create a new generation of smarter medicines that outmaneuver complex diseases in ways previously inconceivable. To accomplish this mission, we are building a synthetic biology platform that could enable us to program next-generation cell and gene therapies with what we refer to as “gene circuits”. These gene circuits, which are created from novel and proprietary combinations of DNA sequences, are intended to reprogram cells with biological logic to sense inputs, compute decisions and respond to their cellular environments. We are designing gene circuits to improve the “intelligence” of cell and gene therapies in order to enhance their therapeutic effectiveness against a broad range of diseases that conventional medicines do not readily address. For more information, please visit the Senti Bio website at https://www.sentibio.com.

Seragon Pharmaceuticals Inc. is a pharmaceuticals company based out of Irvine, CA. The Company was founded by scientists dedicated to leveraging their expertise in biomedical research to improve the quality of human health. Our products reflect years of experience in various clinical, biomedical and research environments. We strive to bring the most advanced health optimization and longevity products to the market by utilizing our knowledge and experience in various specialties. Additionally, we offer various laboratory and consulting services to our partners in industry. Seragon operates state-of-the-art biological research and testing facilities, which provide invaluable services to our strategic associates. Although our portfolio represents years of knowledge and progression, each step forward has shown us how much progress there is still to be made. Seragon is a growing team of gifted scientists, researchers, and creative minds dedicated to innovation, and playing a key role in redefining health care for the consumer. Our purpose is to drive innovation forward, with vigor and an unwavering passion to identify the gaps in modern medicine, by producing ethically sound, tangible health care options that make sense to the consumer. Utilizing collaboration, exceptional talent, principled business practices and a firm grasp of our industry. Seragon paves the way in expanding a universal understanding of Longevity, Metabolism, Gene Therapy and Immunology. Our employees drive the kinetic and creative energies in the Seragon work culture, laying the groundwork for a self-perpetuating, open-minded think-space that results in original and imaginative thought. We believe flexibility of ideas and open dialogue promotes a nimble mind and gives our employees the right kind of environment they need to invent, develop and create the very best technologies in healthcare.

Serotiny is a therapeutic discovery company that designs new genes for next generation cell & gene therapies. We invent therapeutic multi-domain proteins in high-throughput by orchestrating software, mammalian synthetic biology, and therapeutic expertise. At the heart of Serotiny’s technology is a discovery platform involving the close interworking of computationally aided protein design, high-throughput cellular assays, and sophisticated data-driven analytics, which together enable Serotiny to engineer multi-domain proteins for cell therapies, like Chimeric Antigen Receptors used in CAR-T immunotherapies and second-generation Cas9-based gene editing tools, like Base and Prime Editors. Rationally designing proteins for cell & gene therapies requires a different set of constraints and design tools than enzyme or antibody engineering. These therapeutic multi-domain proteins will not be purified, they will be expressed in a human cell, and they will affect the therapeutic phenotype of that cell. Serotiny encodes these constraints into its platform to create a scaled design-build-test-learn process to quickly and efficiently generate therapeutic candidates for next-generation therapies. Serotiny is a multidisciplinary team with expertise including molecular biology, immunology, machine learning, software engineering, data science, and bioinformatics. We’re looking for passionate and creative people to join our team!

Shinobi Therapeutics is developing a new class of hypoimmune CD8ab iPS-T cells for cancer and beyond by using immune evasion to unlock the full potential of iPSCs.

Shoreline's proprietary cell therapy technology platform is based on deep expertise in iPSC differentiation methods and genetic programming of the IL-15/CISH pathway for enhanced metabolic fitness and persistence of the engineered NK Cells to improve anti-cancer activity.

Siren Biotechnology is combining two transformative therapeutic technologies – AAV gene therapy and cytokine immunotherapy - into a single, reimagined modality that overcomes key challenges and redefines how we destroy tumor cells and elicit anti-tumor immunity.

Headquartered in Rancho Cordova, Calif., SK pharmteco is a global contract development and manufacturing organization specializing in the production of active pharmaceutical ingredients (APIs), advanced intermediates, and cell and gene therapy for the pharmaceutical industry. SK pharmteco is comprised of six companies: SK Biotek Korea, SK Biotek Ireland, AMPAC Fine Chemicals, AMPAC Analytical Laboratories in the U.S., Yposkesi and the Center for Breakthrough Medicines.

Sonoma Biotherapeutics is developing engineered regulatory T cell (Treg) therapies to treat serious autoimmune and inflammatory diseases driven by an imbalanced immune system. Founded by pioneers in Treg biology and cell therapy, the company is employing proprietary platform technologies and approaches to develop a new generation of targeted Treg cell therapies designed to induce durable immune tolerance to treat and prevent autoimmune and inflammatory diseases. Sonoma Biotherapeutics is based in South San Francisco and Seattle.

SonoThera is the world's first ultrasound-guided gene delivery platform designed to overcome limitations of current gene therapy approaches.

Sorrento Therapeutics is an antibody-centric, clinical stage biopharmaceutical company developing new treatments for cancer, inflammation and autoimmune diseases. Sorrento's lead products are multiple late-stage biosimilar and biobetter antibodies, as well as clinical CAR-T therapies targeting solid tumors.

Stanford University is a Higher Education institution that focuses on research, innovation, and providing educational services.

Switch Therapeutics is an emerging preclinical stage biotechnology company pioneering a new type of medicine that integrates nucleic acid nanotechnology and RNA interference (RNAi) science with the goal of treating a range of diseases – affecting the central nervous system and systemic indications – with significant unmet needs. Switch’s novel gene knockdown approach is based on technology developed by renowned researchers in the field of RNA from Caltech, Harvard and City of Hope. Based on the scientific discovery of Switch’s co-founders, the company has developed a novel proprietary platform known as CASi (Conditionally Activated siRNAs) that combines advantageous properties of both single and double-stranded RNAs in a single molecule, allowing for cell selective RNAi activity. To date, Switch has raised $52M in funding through Series A, and the company’s South San Francisco based team has continued to grow as its research has advanced.

Synthekine is an engineered cytokine therapeutics company developing disease-optimized treatments. The company uses immunological insights to guide targeted protein engineering to generate transformative medicines for cancer and autoimmune disorders. Using the principles of cytokine partial agonism and immunological specificity, Synthekine designs differentiated therapeutics to be both safe and efficacious. Its lead programs have shown promising efficacy and tolerability in preclinical studies, and it is developing additional cytokine partial agonists that selectively modulate key pathways of the immune system.

Targazyme Inc. is a San Diego-based, clinical-stage biopharmaceutical company developing novel enzyme-based platform technologies and products to improve clinical efficacy and cost of care outcomes for cell therapy, immunotherapies for autoimmune diseases and cancer, stem cell transplantation, gene therapy and regenerative medicine. The Company’s clinical-grade fucosyltransferase enzymes and small molecule products (TZ101 and TZ102) are off-the-shelf biologic products used at the point-of-care to treat therapeutic cells immediately before infusion into the patient using a simple procedure that is easily incorporated into existing medical practice. The Company has received worldwide patents, multiple FDA orphan drug designations, has an open investigational new drug application (IND) with multiple ongoing clinical studies and a Phase 3 Special Protocol Assessment (SPA) with the FDA. Targazyme’s fucosylation technology has received numerous medical and scientific awards from institutions such as NIH, NCI, US Treasury, CPRIT, ETF, OCAST, JDRF, Leukemia, Lymphoma Society. Targazyme has partnerships and collaborations with Kyowa Hakko Kirin and Florida Biologix, as well as various medical research institutions including The University of Texas M.D. Anderson Cancer Center, Oklahoma Medical Research Foundation, Texas Transplant Institute, Case Western/University Hospitals, Scripps Hospitals, Fred Hutchinson Cancer Research Center, University of California Los Angeles Medical Center, Stanford University Medical Center, University of Minnesota Medical Center, University of California San Diego Medical Center, Sanford-Burnham Medical Research Institute, Indiana University, Memorial Sloan Kettering Cancer Center, and New York Blood Center. Learn more at http://www.targazyme.com.

T-Cure is a clinical-stage immuno-oncology company developing the next generation of T cell therapies for patients with solid tumors. It is committed to bringing new targets and therapies to the T-Cell Receptor (TCR) field that have a high likelihood of clinical efficacy.

Teknova makes solutions possible. Since 1996, Teknova has been innovating the manufacture of critical reagents for the life sciences industry to accelerate the discovery and development of novel therapies that will help people live longer, healthier lives. We offer fully customizable solutions for every stage of the workflow, supporting industry leaders in cell and gene therapy, molecular diagnostics, and synthetic biology. Our fast turnaround of high-quality agar plates, microbial culture media, buffers and reagents, and water helps our customers scale seamlessly from RUO to GMP. Headquartered in Hollister, California, with over 200,000 square feet of state-of-the-art facilities, Teknova's modular manufacturing platform was designed by our team of scientists, engineers, and quality control experts to efficiently produce the foundational ingredients for the discovery and commercialization of novel therapies.

Telesis Bio is empowering researchers to accelerate the creation of novel synthetic biology solutions to address some of humanity's greatest challenges. With our breakthrough automation solutions for biological synthesis and expertise in DNA biology, we enable rapid, accurate, and reproducible writing of DNA and mRNA for wide-ranging biological applications. Around the world, innovators are leveraging Telesis Bio's technology to overcome current bottlenecks in synthetic biology workflows and advance the discovery of cutting-edge solutions for health and technology. We are proud of our growing collaborations with partners that include premier academic research institutions, emerging start-ups to nearly all of the Top 25 Biopharma companies. Telesis Bio is honored to play a part in several pioneering advances of our customers and collaborators ranging from novel infectious disease vaccines, precision immunotherapy for cancer and antibody therapeutics to creation of engineered meat substitutes and sustainable cellular agricultural products.

Tenaya Therapeutics, Inc. develops therapeutics for heart failure. It offers cellular regeneration platform, that enables in vivo reprogramming of cardiac fibroblasts into cardio myocytes by delivering proprietary transcription factors; gene therapy platform, that enables the targeted delivery of a payload to cardiac fibroblasts; and precision medicine platform, which provides personalized medicine approach to treat heart disease. The company was incorporated in 2016 and is based in South San Francisco, California.

The Angeles Clinic & Research Institute is a healthcare institution that specializes in providing clinical services and conducting medical research, with a focus on cancer treatment.

Theragene Pharmaceuticals is creating life-changing cancer and cardiology treatments through the development of our novel immunotherapy and Adeno-Associated Virus (AAV) gene therapy platforms. As a company operating in both the US and Asia, we deliver unique product development models, utilizing the strengths of each region, while delivering safe, effective treatments. Our mission is to advance cutting-edge science through efficient development plans while pursuing licensing or co-development collaborations.

Theragent is a full-service cGMP Contract Development and Manufacturing Organization delivering innovative cell and gene therapy products to clients and patients around the world. Our expert staff and state of the art facility serves as a “one stop shopâ€? for all biopharma stakeholders seeking assistance in manufacturing and product development. Theragent’s cGMP facility features four manufacturing clean suites equipped with Digital DES technology, in house QC/QA, Microbiology, MSAT capabilities, and custom designed ERP system with paperless MBRs for real time reporting.

ThinkCyte's mission is to provide scientists and biomedical professionals with a transformational single-cell technology that accelerates biological discoveries and enable the development of novel treatments and diagnostics through the integration of advanced hardware, machine learning, and biotechnology.

Launched in 2024, Trace Neuroscience is a biopharmaceutical company on a mission to expand the promise of genomic medicine for people living with neurodegenerative diseases. With an initial focus on ALS, the company is developing novel therapies to restore UNC13A protein function to re-establish healthy communication between nerves and muscle cells.

Trestle Biotherapeutics, Inc. is a preclinical stage company developing bioengineered therapies for patients living with end stage renal disease. Once implanted, these bioengineered stem cell-derived tissues will get patients off dialysis, delay their need for transplantation, and one day become replacement organs.

We are a biotechnology company focused on restoring cells’ youthful vigor to repair damage caused by the aging process. Using a proprietary ERA™ Platform, we develop mRNA medicines that are specially formulated to instruct specific cells in the body to fight disease or repair damaged tissue. We do this by reprogramming the epigenome to restore cell function that people often lose as they age. This approach can be used to revitalize different cells throughout the body – we are researching its applicability across multiple therapeutic areas, including dermatologic conditions, immunology, osteoarthritis and ophthalmology.

Turnstone Biologics is a clinical-stage biotechnology company developing new medicines to treat and cure solid tumors by pioneering a differentiated approach to TIL therapy. Our innovative TIL therapy is based upon the identification, selection, and expansion of the most potent tumor-reactive T cells, known as Selected TILs, and is designed to overcome the limitations of first-generation bulk TILs that have demonstrated objective responses only in limited tumor types. Our most advanced program, TIDAL-01, is currently being evaluated in two Phase 1 studies in patients with melanoma, breast cancer, and colorectal cancer. We are also actively advancing a pipeline of preclinical programs, including TIDAL-02, our next Selected TIL program, and our TIDAL-01 and viral immunotherapy combination program.

This page is no longer active or monitored. We are now Sumitomo Pharma America (SMPA), a science-based, technology-driven biopharmaceutical company focused on delivering therapeutic and scientific breakthroughs in areas of critical patient need. See pinned post for our new page and follow us!⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀⠀⠀⠀ ⠀ ==== We aim to advance urologic patient care through bold innovation, based on diversity of thought, openness to new ideas, adaptation to change, integrity and compassion. We develop innovative therapies and participate in exceptional licensing opportunities. Our goal is to be the leading specialty company: recognized and trusted by patients and providers for our commitment to urology. Urologic conditions can have a major impact on daily life, with those affected often suffering in silence as their conditions are overlooked, misunderstood or not adequately treated. Read more about our community guidelines: https://urovant.com/community-guidelines/

Vascular BioSciences (VBS), a biomedical company with operations in Research Triangle, NC, San Diego, and Goleta, CA, makes interventional catheters to obtain pulmonary endoarterial biopsies, provides molecular diagnostic services, and through its majority-owned subsidiary, VBS Pharmaceuticals, advances targeted therapies in order to enhance and prolong huma

We are a team of experts driven by complex image analysis problems. ViQi is modernizing image processing technology and expediting science discovery through the creation of AI-based assay development systems. Our technology not only generates better results, but our cloud-based platform allows us to scale quickly and interact with over 150 types of large-scale image formats. Currently our technology is employed in life sciences, specifically virology and high content image analysis; digital pathology and environmental monitoring. Our cloud based solutions enable any lab access to our software and reporting. Follow us to get updates on latest discoveries, customer collaborations such as webinars or publications and more.

Vironexis is focused on transforming the future of cancer treatment by pioneering AAV-delivered T-cell immunotherapy. Our TransJoin™ AAV Gene Therapy Platform enables the creation of off-the-shelf, single-dose gene therapies designed to overcome the key challenges and shortcomings of current immunotherapies, including CAR-T and bispecific antibodies. Our current pipeline includes more than ten product candidates for blood-based cancers, solid tumor metastasis prevention, and a cancer vaccine. Our lead program, VNX-101, for the treatment of CD19+ acute lymphoblastic leukemia, is anticipated to begin clinical trials in the fourth quarter of 2024.

Visgenx is developing therapeutics based on increasing expression of a gene known as ELOVL2 which has been shown to regulate aging in the retina and other tissues. Our lead indication is Dry Age-Related Macular Degeneration (Dry AMD).

Vybion developed a novel vectorized intrabody (INT41) for Huntington’s disease, a devastating inherited orphan disease that results in the progressive loss of both motor and cognitive function. INT41 is effective in both cell-based system and animal models. INT41 has Orphan Disease Designation from the FDA and a pre-IND meeting defined the basic elements of the IND enabling package. INT41 binds to an epitope that is unavailable in the functional molecule, but appears in the toxic degradation fragment that accumulates in the nucleus, binds to DNA via the polyglutamine and alters gene expression detectable years before the onset of symptoms. INT41 stabilizes this fragment and facilitates its degradation. In addition to Huntington’s disease, Vybion is testing INT41 as a therapy for spinobulbar muscular atrophy (SBMA) and spinocerebellar Ataxia 1, 3 and 7 (SCA), two closely related Orphan diseases with an identical target sequence and polyglutamine expansion as seen in Huntington’s disease.

Western University of Health Sciences is a higher education institution specializing in health sciences and medical education.

Wyatt Technology is a California-based developer and manufacturer of commercial light scattering instruments for sectors including medical and food and beverage.

X-Therma Inc. is solving unmet legacy needs in the storage of biological products using convergent biomimetic nanotechnology. This solution can better enable critical biomanufacture processes, safer transport and long-term biobanking for advanced cell therapies, in vitro fertilization, organ transplants, and other rapidly growing regenerative medicine areas.

At Xyphos, we’re working to deliver flexible and adaptable cancer therapies with the potential to revolutionize cancer care.