List of Adeno-associated viral vector (AAV) Companies in California - 72

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.

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.

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.

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.

Antibodies Incorporated is a biotechnology company that specializes in manufacturing and distributing monoclonal and polyclonal antibodies, diagnostic reagents, diagnostic kits, and developing ImmunoAssays.

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.

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.

Aves Labs produces high-affinity, custom chicken IgY and other immunoreagents for biomedical research and antibody manufacturing.

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.

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.

Biomiga, Inc. is an innovative biotech research and service company based in San Diego, California. Our mission is to provide the highest quality laboratory products while maintaining the best possible service and support to our end-users. Our products maximize efficiency and cost-effectiveness through our cutting-edge technology and process. Biomiga owns the manufacturing process, enabling us to control both the quality of the products as well as the price. Our products are more reliable and effective while often costing less than half the price of our competitors. Biomiga can customize its products to meet a client’s exact specifications and requirements. Biomiga provides full service support to all our clients through the entire process. Our support and sales staffs are available for consultation 24 hours a day, 7 days a week. We stand behind all our products with a full guarantee. Our mission is to ensure our client’s satisfaction. Biomiga emphasizes on product development. Our R&D team is highly trained in the biological sciences to quickly develop new products. Our products have been proven to meet our clients' high demands with greater cost savings and effectiveness. EzgeneTM and ViraTrapTM are just some examples.

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.

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

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.

Caribou Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the development of genome-edited allogeneic cell therapies for the treatment of hematologic malignancies and solid tumors in the United States and internationally. Its lead product candidates are CB-010, an allogeneic anti-CD19 CAR-T cell therapy that is in phase 1 clinical trial to treat relapsed or refractory B cell non-Hodgkin lymphoma; and CB-011, an allogeneic anti-BCMA CAR-T cell therapy for the treatment of relapsed or refractory multiple myeloma. The company also develops CB-012, an allogeneic anti-CD371 CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia; and CB-020, an allogeneic CAR-NK cell therapy for the treatment of solid tumors. Caribou Biosciences has collaboration with AbbVie Manufacturing Management Unlimited Company to develop CAR-T cell therapies. The company was incorporated in 2011 and is headquartered in Berkeley, 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.

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).

CHO Plus is creating regulatory compliant genetically engineered mammalian cells to be used by biopharmaceutical companies for therapeutic protein manufacturing. Early proof of concept experiments demonstrate CHO Plus cells to be 4.5-fold more productive than parent cells in model system. The CHO Plus approach includes patented methods for genetically engineering mammalian cells; proprietary and patented targeted transfection technology; and efficient clone screening technology.

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.

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

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.

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

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.

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.

Gilead Sciences, Inc., a research-based biopharmaceutical company, discovers, develops, and commercializes medicines in the areas of unmet medical need in the United States, Europe, and internationally. The company provides Biktarvy, Genvoya, Descovy, Odefsey, Truvada, Complera/ Eviplera, Stribild, and Atripla products for the treatment of human immunodeficiency virus (HIV) infection; Veklury, an injection for intravenous use, for the treatment of coronavirus disease 2019; and Epclusa, Harvoni, Vosevi, Vemlidy, and Viread for the treatment of liver diseases. It also offers Yescarta, Tecartus, Trodelvy, and Zydelig products for the treatment of hematology, oncology, and cell therapy patients. In addition, the company provides Letairis, an oral formulation for the treatment of pulmonary arterial hypertension; Ranexa, an oral formulation for the treatment of chronic angina; and AmBisome, a liposomal formulation for the treatment of serious invasive fungal infections. Gilead Sciences, Inc. has collaboration agreements with Arcus Biosciences, Inc.; Pionyr; Tizona; Tango Therapeutics, Inc.; Jounce Therapeutics, Inc.; Galapagos; Janssen; Japan Tobacco, Inc.; Gadeta; Bristol-Myers Squibb Company; Merck; and Novo Nordisk A/S. The company was founded in 1987 and is headquartered in Foster City, California.

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.

ImmunityBio (previously NantKwest (NASDAQ: NK)) is a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases. NK cells are the body’s first line of defense due to the innate ability of NK cells to rapidly identify and destroy cells under stress, such as cancer or virally-infected cells. ImmunityBio is uniquely positioned to implement precision cancer medicine, with the potential to change the current paradigm of cancer care. Natural Killer cells are ancient cells in the human body designed to recognize and detect cells under stress or infected. Our “off-the-shelf” activated Natural Killer (NK) platform have the capacity to destroy cancer and virally infected cells from the body. The safety of our NK cells as well as their activity against a broad range of cancers have been tested in multiple phase 1 clinical trials in the United States, Canada and Europe. In addition to our NK cells capability to be administered in the outpatient setting as an “off-the-shelf” living drug, it serves as a universal cell-based therapy without need for individualized patient matching. Moreover, our NK cell based platform has been bioengineered to incorporate chimeric antigen receptors (CARs) and antibody receptors to further optimize targeting and potency in the therapeutic disease. Our targeted therapeutic areas include: (1) cancer, focusing on bulky hematological cancers and solid tumors as well as cancer stem cells, (2) infectious diseases, including viral, fungal and bacterial infections, and (3) inflammatory diseases, ranging from rare inherited diseases to more prevalent autoimmune disorders.

Innovative NeuroTechnologies, Inc. (INT) is a development-stage biotechnology company operating in “virtual mode” that is currently pursuing a pipeline of novel biologically-based and disease-modifying therapeutic solutions to prevent, delay, or mitigate the underlying dementia or cognitive decline associated with a spectrum of neurodegenerative disorders referred to as tauopathies, which includes: • Alzheimer’s disease (AD), • Frontotemporal dementia (FTD or Pick’s disease), • Chronic traumatic encephalopathy (CTE) or dementia pugilistica, • Progressive supranuclear palsy (PSP), • Corticobasal degeneration (CBD), • Familial form of frontotemporal dementia and parkinsonism linked to chromosome 17 (FTDP-17), and • Parkinson's disease dementia (PDD). The pathology of these seemingly disparate conditions is similar in that they involve dysregulation of the Tau protein and development of neurofibrillary tangles (NFTs), synaptic dysfunction, and/or neuronal loss, which underlie the observed dementia or cognitive deficits. Unfortunately, there are no effective therapies presently available for this spectrum of disorders, and neurodegenerative tauopathies remain a very significant socioeconomic burden and tremendous unmet medical need worldwide.

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.

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.

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.

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.

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.

Okogen is a clinical stage ophthalmic pharmaceutical company. Our lead product is in clinical trials as a “one stop shop” treatment for all forms of acute infectious conjunctivitis. Okogen is focused on developing ophthalmic therapeutics for unmet needs in ophthalmic infectious diseases.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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

At SBI, our mission is to accelerate your science by turning the latest insights and technologies into robust research products and services to advance exosome studies, biomarker research, gene delivery, SARS-CoV-2 studies, and more.

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.

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.

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.

Via Nova Therapeutics is committed to discovering novel therapies for viral diseases, especially those arising from acute & subacute infections.

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.

Xencor, Inc., a clinical stage biopharmaceutical company, focuses on the discovery and development of engineered monoclonal antibody and other protein therapeutics to treat severe and life threatening diseases. It develops its antibody product candidates to treat cancer and autoimmune diseases. The company’s product candidates include XmAb5871, an immune inhibitor, which has been completed Phase II clinical trial for the treatment of autoimmune diseases; AIMab7195, which is in development and commercialization stage. Its product candidates also comprise XmAb14045, a bispecific antibody which is in Phase I clinical trial for the treatment of acute myeloid leukemia; plamotamab (XmAb13676) that is in Phase I clinical trial to treat B-cell malignancies; and XmAb18087, which is in Phase I clinical trial for neuroendocrine tumors and gastrointestinal stromal tumors. In addition, the company offers Tafasitamab, an antibody drug candidate for the treatment of patients with relapsed/refractory diffuse; AMG424, a bispecific antibody that targets CD38 and CD3, which is in Phase I clinical trial for the treatment of various myeloma; and AMG509, a bispecific antibody that is in preclinical development stage to treat prostate cancer. Further, it is developing bispecific antibodies to treat various cancers, such as XmAb20717, XmAb22841, and XmAb23104 which are in preclinical Phase. Additionally, it offers XmAb24306, initial cytokine candidate which is in Phase I clinical trial. The company has collaboration and license agreement with Novartis Institutes for BioMedical Research, Inc.; Amgen Inc.; Catalent Pharma Solutions LLC.; Janssen Biotech, Inc.; MorphoSys AG; Incyte Corporation; and The University of Texas MD Anderson Cancer Center. Xencor, Inc. was founded in 1997 and is headquartered in Monrovia, California.