List of Gene Therapy Companies in Massachusetts - 139

Our name, 2seventy bio, reflects why we do what we do - TIME. Cancer rips time away, and our goal is to work at the maximum speed of translating human thought into action – 270 miles per hour – to give the people we serve more time. We are building the leading immuno-oncology cell therapy company, focused on discovering and developing new therapies that truly disrupt the cancer treatment landscape With a deep understanding of the human body’s immune response to tumor cells and how to translate cell therapies into practice, we’re applying this knowledge to deliver next generation cellular therapies that focus on a broad range of hematologic malignancies, including the first FDA-approved CAR T cell therapy for multiple myeloma, as well as solid tumors. Our research and development is focused on delivering therapies that are designed with the goal to “think” smarter and faster than the disease. Importantly, we remain focused on accomplishing these goals by staying genuine and authentic to our “why” and keeping our people and culture top of mind every day.

908 Devices is revolutionizing chemical analysis with its simple handheld and desktop devices, addressing life-altering applications. The Company’s devices are used at the point of need to interrogate unknown and invisible materials and provide quick, actionable answers to directly address some of the most critical problems in bioprocessing, pharma/biopharma, forensics, life sciences research and adjacent markets. The Company is headquartered in the heart of Boston, where it designs and manufactures innovative products that bring together the power of complementary analytical technologies, microfluidic sampling and separations, software automation, and machine learning.

Abata Therapeutics - We are bringing an entirely new approach to the treatment of autoimmune disease by engineering Tregs as targeted therapies that stop immune-mediated destruction, restore homeostasis – a state of harmony – and promote repair in the affected tissues. In addition to our lead program in progressive MS, Abata has early programs in Type 1 Diabetes (T1D) and inclusion body myositis (IBM). We bring together industry experts and deeply engaged pioneers in Treg biology, T cell receptor and antigen discovery, disease pathogenesis, and molecular and imaging biomarkers. We are bold in our mission and purpose and aim for nothing less than a transformative impact on people’s lives.

Our Mission To create best niche antibody-based biologics for fulfilling the research and development needs in biomedical and biopharmaceutical communities. Our long range goal is to develop therapeutic and diagnostic entities. Core Competence ABB's core competence is in protein designs of biologics for diagnostic and therapeutic applications of immuno-oncological disorders. Specifically, our R&D for therapeutics is focused on biologics that are capable of modulating activities associated with antigenic targets and effector molecules. In addition, ABB has designed and produced a panel of immune related protein reagents that are available to biomedical researchers as investigational tools. History Founded in 2007, AB Biosciences (ABB) has been dedicated to the engineering of therapeutic biologics and research protein reagents. With decades of combined experience in research and development, ABB's scientists have built an efficient platform for the development of these two protein classes. The recombinant replacement of the traditional IVIG (PRIM program) is a great example of our engineering expertise in R&D of an unique protein drug for replacing the traditional serum-derived IVIG. Our Community Massachusetts provides a vibrant and nurturing environment for life science ventures like ABB, in terms of training grants and tax benefits, as well as an abundant talent pool possessing a wide range of expertise. Discovering breakthrough biotechnology and exploring biologics for biomedical study is our mission at AB Biosciences. As part of our commitment to science education, we open our doors to intern students, who can experience firsthand the R&D employment possibilities in the Biotech Industry. More than 15 students/interns have visited our laboratory since 2009.

At Adicet (Nasdaq: ACET), we are defined by our mission to deliver best-in-class gamma-delta T cell therapies for patients fighting autoimmune diseases and cancer. Every one of our team members, whether in the lab, manufacturing, or general business role, comes to work every day with the patient in mind. As pioneers in the cell therapy space, we are honored and humbled by the opportunity to put our talents to bear in developing transformative therapies to defeat autoimmune diseases and cancer. We believe that the best way to do this is through discovering and developing allogeneic gamma-delta T cell therapies, a novel type of cell therapy. Adicet has chosen the gamma-delta T cell because it: • Preferentially traffics to tissues and provides B cell depletion, potentially leading to an immune reset for patients with autoimmune diseases. • Has multiple ways to target and kill cancer cells including solid tumors. We also chose to use cells from donors (allogeneic) to enable our potential therapies to be readily available when needed (taken “off-the-shelf” or available to the patient on demand). We’re not just blazing new trails in cell therapy, we’re having fun along the way. With regular team gatherings, many shared meals, larger company events, and a consistent commitment to our values, we have a strong culture! We are proud to attract and retain some of the greatest talent in the biopharma and cell therapy space, and we’re consistently giving back to our employees. The Adicet benefits package includes generous financial compensation, comprehensive health plans, generous paid time-off, educational reimbursement, and access to many amenities in our state-of-the-art facility. To learn more or inquire about joining our team in Boston, MA or Redwood City, CA, please visit http://www.adicetbio.com

Our focus on service, quality, and reliability has made Advanced Instruments a leading developer and manufacturer of analytical instruments and services for the biopharmaceutical, clinical, and food & beverage industries for more than 65 years. Globally, we provide innovative technologies aligned with the needs of the markets we serve, helping to improve efficiency, enhance productivity, and ensure best-in-class performance. Our Solentim portfolio of best-in-class imaging and single-cell deposition technologies enables the clonal isolation, outgrowth, and characterization of the highest value cells for monoclonal antibody upstream development and cell and gene therapy. Our robust line of micro-osmometers is built specifically for the biotech industry and seamlessly integrate into both non-regulated and regulated GMP workflows ensuring increased yield, quality, and purity. Similarly, clinical labs worldwide rely on our products to provide high-quality results and optimize workflow efficiency. With products for clinical chemistry, microbiology and hematology labs, our innovative solutions enable the delivery of accurate and timely patient results to help improve health outcomes and help simplify workflows to support an increase in lab productivity. Our newest addition, the Artel portfolio, includes leading analytical instruments, software and services that validate, automate, and calibrate critical liquid handling processes and instrumentation. We are a global leader with products supported by a worldwide network of direct salespeople and distributors, with 24/7 technical support. Our direct presence is growing as we continue to expand the team with a continued focus on being the supplier of choice. Contact us if you are interested in joining a dynamic team! For more information, visit www.aicompanies.com.

Aera was founded with a vision to unlock the potential of genetic medicines across a broad range of modalities and therapeutic areas. Our protein nanoparticle (PNP) delivery platform leverages the discovery of endogenous, human proteins that can self-assemble to form capsid-like structures and that can package and transfer nucleic acid cargo. This platform has the potential to address many of the limitations of today’s delivery technologies and to enable various genetic medicine modalities.

Affinia Therapeutics is an innovative gene therapy company focused on broadening the reach of gene therapy. Our proprietary Affinia Rationally-designed Therapies (ART) platform is created to address key limitations of conventional gene therapies, by developing novel capsids, novel promoters, and novel manufacturing approaches to developing gene therapies for rare and prevalent devastating diseases. We are backed by a strong syndicate of life science investors and have ambitious plans to have a dramatic impact on the lives of patients around the world.

Affini-T is a leading precision immunotherapy company targeting core oncogenic driver mutations to develop potentially curative therapies for patients with solid tumors. Our differentiated cell therapy platforms harness state-of-the-art engineering, synthetic biology, and gene editing capabilities to target even the most devastating cancer-driving mutations, beginning with KRAS. We leverage these tools to optimize T cell functions and rewrite the rules of the solid tumor microenvironment, enabling the potential for sustained clinical outcomes in patients. Building on the world-class innovation inherent in our leadership team, founders and technologies, we are powered to develop transformational medicines that last.

Founded in 2016, AffyImmune is a clinical-stage biotechnology company that optimizes CAR T cells for the treatment of solid tumors using our Tune & Track platform. AIC100, our first product, spares healthy cells through our unique affinity-tuning, enhancing its safety and efficacy. The initial indication for AIC100 is refractory thyroid cancer, which has a very poor prognosis and very few effective therapies. The antigen targeted by AIC100 is also overexpressed in a variety of additional cancers, including stomach, triple negative breast cancer and a number of others.

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss.

Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between FalconTM, the company’s proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and four novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone’s lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome, and gene replacement for the treatment of IGHMBP2-related disorders including spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S).

Aleta Biotherapeutics’ unique portfolio of multi-antigen targeting solutions for cell therapy address are designed to enable approved CAR T cell therapies to work better in B cell lymphoma, B cell leukemia and multiple myeloma. Our technology is designed to further enable CAR T cell therapies to successfully treat non-B cell cancers such as acute myeloid leukemia (AML), and solid tumors such as breast cancer, gastric cancer and pediatric brain tumors, an area where advances are desperately needed.

Arbor Bio is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines, with the most extensive toolbox of proprietary genomic editors in the industry. We have the unique ability to work backward from disease pathology and choose the optimal editors or combination of editors for the disease in question.

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With active discovery and preclinical programs in ophthalmology, neurological and neuromuscular disorders, and other rare diseases, Ascidian is opening new therapeutic possibilities for patients in need of breakthroughs.

Asimov's mission is to advance humanity's ability to design living systems, enabling biotechnologies with outsized societal benefit. The company is developing a synthetic biology platform from cells to software to design and manufacture next-generation therapeutics, including cell & gene therapies, through a combination of products, services, & collaborations. Founded by bioengineers from MIT and Boston University and headquartered in Boston, the company has raised over $200 million.

AvenCell - Based on our deep science and focused on patients in need, we develop next generation immunotherapies for hard-to-treat cancers.

Azenta (Nasdaq: AZTA) is a leading provider of life sciences solutions worldwide, enabling impactful breakthroughs and therapies to market faster. Azenta provides a full suite of reliable cold-chain sample management solutions and genomic services across areas such as drug development, clinical research and advanced cell therapies for the industry's top pharmaceutical, biotech, academic and healthcare institutions globally. Azenta is headquartered in Burlington, MA, with operations in North America, Europe and Asia. For more information, please visit www.azenta.com.

Beam Therapeutics Inc., a biotechnology company, engages in developing precision genetic medicines for patients suffering from serious diseases in the United States. The company is developing therapies for the development of sickle cell disease and beta-thalassemia; CAR-T cell therapies for pediatric T-cell acute lymphoblastic leukemia and pediatric acute myeloid leukemia; therapies for alpha-1 antitrypsin deficiency and glycogen storage disorder 1A; and therapies for ocular and central nervous system disorders. It has a research and clinical collaboration agreement with Magenta Therapeutics, Inc. Beam Therapeutics Inc. was founded in 2017 and is based in Cambridge, Massachusetts.

Be Bio is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies.

Through cutting-edge science and medicine, Biogen discovers, develops and delivers innovative therapies worldwide for people living with serious neurological and neurodegenerative diseases. Founded in 1978, Biogen is a pioneer in biotechnology and today the Company has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first and only approved treatment for spinal muscular atrophy, and is at the forefront of neurology research for conditions including Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral sclerosis. Biogen also manufactures and commercializes biosimilars of advanced biologics. With approximately 7000 people worldwide, we are truly a global organization, headquartered in Cambridge, Massachusetts, which is also home to our research operations. Our international headquarters are based in Zug, Switzerland and we have world-class manufacturing facilities in North Carolina and Denmark. We offer therapies globally through direct affiliate presence in 30 countries and a network of distribution partners in over 50 additional countries.

bluebird bio, Inc., a biotechnology company, engages in researching, developing, and commercializing of transformative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin for the treatment of ß-thalassemia and sickle cell disease; and Lenti-D for the treatment of cerebral adrenoleukodystrophy. The company’s product candidates in oncology include bb2121 and bb21217, which are chimeric antigen receptor T (CAR T) cell product candidates for the treatment of multiple myeloma. It has a strategic collaboration with Bristol-Myers Squibb to discover, develop, and commercialize disease-altering gene therapies in oncology; and Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize various immune cell therapies for cancer. The company also has collaborations with Medigene AG to discover T cell receptor (TCR) product candidates in the field of cancer; Gritstone Oncology, Inc. to discover TCR product candidates in the field of cancer; TC BioPharm Limited to research and develop gamma delta CAR T cells directed at hematologic and solid tumor targets; Forty Seven, Inc. to pursue clinical proof-of-concept for an antibody-based conditioning regimen; and Novo Nordisk A/S to develop in vivo genome editing treatments for genetic diseases, as well as clinical trial collaboration with Magenta Therapeutics. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was founded in 1992 and is headquartered in Cambridge, Massachusetts.

BlueRock Therapeutics is a leading engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock Therapeutics’ cell+gene platform harnesses the power of cells to create new medicines for neurology, cardiology, and immunology indications. BlueRock Therapeutics’ cell differentiation technology recapitulates the cell’s developmental biology to produce authentic cell therapies, which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock’s culture is defined by scientific innovation, the highest ethical standards and an urgency to bring transformative treatments to all who would benefit. The company strives to be a top employer of scientific talent, empowering every member of the team to make meaningful and lasting contributions to the burgeoning field of regenerative medicine. Become a BlueRocker! We are expanding our BlueRock team at all locations. Contact us via LinkedIn or our website to learn about rewarding career opportunities.

BIB is a full service CDMO partner offering cGMP and non-GMP mammalian, microbial, gene therapy and cell therapy services.

Boston University is a Higher Education institution that offers a wide range of academic programs and conducts research.

BrickBio's engineered RNA and protein-chemistry platform technology enables custom tailoring of any protein with homogenous, site-specific and site-selective bioconjugation modifications, enabling novel classes of biologics with superior therapeutic windows and characteristics such as half-life, dosage, and efficacy. Having validated its precise and unrestricted conjugation capabilities through pharmaceutical co-development partnerships, the company is building its internal oncology and immunology pipeline by creating unique antibody and bispecific-conjugates while additionally pioneering spatial protein assembly for Precise Protein Origami™.

Boston's Brigham and Women's Hospital (BWH) is an international leader in virtually every area of medicine and has been the site of pioneering breakthroughs that have improved lives around the world. A major teaching hospital of Harvard Medical School, BWH has a legacy of excellence that continues to grow. With two outstanding hospitals, over 150 outpatient practices, and over 1,200 physicians, we serve patients from New England, throughout the United States, and from 120 countries around the world. The BWH name is a reflection of our history. In 1980 three of Boston’s oldest and most prestigious Harvard Medical School teaching hospitals - the Peter Bent Brigham Hospital, the Robert Breck Brigham Hospital, and the Boston Hospital for Women – merged to form Brigham and Women’s Hospital. As a national leader in improving health care quality and safety, we have helped to develop some of the industry’s best practices including computerized physician order entry (CPOE) to prevent medication errors. The CPOE is now a nationally-accepted safety practice. The BWH Biomedical Research Institute (BRI) is one of the most powerful biomedical research institutes in the world and the second largest recipient of National Institutes of Health (NIH) funding among independent hospitals in the United States. BWH has long had great success in research as measured by the number of important discoveries made, the size and scope of its research portfolio and the volume of publications annually. BWH is a training ground for physicians, nurses, and allied health professionals. We have 1,100 trainees in over 140 of the most sought after training programs in the world, and also host Harvard Medical School students in rotations throughout our programs. As our global health services expand, our clinical trainees have rich opportunities to contribute and learn in challenging environments around the world. Brigham and Women's Hospital is an EEO, AA, VEVRAA Employer.

At Candel Therapeutics, we are developing immunotherapies that induce immunogenic cell death in cancer cells at the site of injection, unmasking tumor neo-antigens within an activated microenvironment. This process leads to a systemic, durable immune response against the tumor, with the potential to change disease outcomes across a variety of indications. Candel’s products are designed to improve survival while maintaining quality of life – from early- to late-stage disease.

Carbon Biosciences is a team of passionate drug developers extending the reach of genetic medicines for the treatment of devastating diseases. We combine the genetic diversity and potency of viruses that have evolved over millions of years with the recent clinical and manufacturing advances in the gene therapy field. Our proprietary CBN vector library has the potential advantages of exquisite tissue specificity, larger payloads and durable treatment strategies across multiple modalities. Our goal: realize the power of natural viral evolution and deliver on the promise of genetic medicines.

Carmine Therapeutics, headquartered in Cambridge MA, is pioneering a new class of non-viral gene therapies based on red blood cell extracellular vesicles, combined with proprietary payloads. In June 2020, Carmine inked a research alliance with Takeda Pharmaceuticals worth up to $900 million to develop non-viral gene therapies for 2 targets. Carmine has also been named one of the FierceBiotech’s Fierce 15 Biotech companies of 2020 and was also awarded the Bristol-Myers Squibb "Golden Ticket". Carmine was founded in 2019 by EVX Ventures, Professors Harvey Lodish (Whitehead, MIT), Minh Le and Jiahai Shi. Professor Lodish, who was a founding member of several successful biotech companies that include Genzyme and Millennium Pharmaceuticals, is also Chair of the Scientific Advisory Board of Carmine. For more information, visit www.carminetherapeutics.com Media Relations Media@carminetherapeutics.com Investor Relations Investors@carminetherapeutics.com

At Catamaran, our mission is to harness the power of our proprietary TAILWIND™ Platform to develop safe and effective, off-the-shelf, allogeneic immune cell therapies for the treatment of patients with cancer and immune disorders.

About Us: Cell BioEngines, Inc., founded in Dec 2022, is a clinical-stage biotech company focused on developing allogeneic 'off-the-shelf' stem cell and its derived therapies as ‘drugs’ for human disease treatment. The company utilizes a ‘plug-and-play’ proprietary platform technology that transforms universal ‘non-gene-modified’ donor blood stem cells from umbilical cords, to produce high-quality, pure and potent, multiple clinical grade cells at scale. This innovative method aims to make stem cells and its derived therapies more accessible and efficient. Its lead product is currently in Phase 1 trials for the treatment of hematopoetiic neoplasms not curable with available therapies, particularly acute leukemia and myelodysplasia. The lead pre-clinical stage product is a first-in- class ‘one-size-fits-all’ cell-based cancer vaccine, composed of dendritic cell type 1 (cDC1) to circumvent the barriers in solid cancers. Platform Technology: The company's platform technology is what makes it innovative and unique. It's "Cell BioEngineMAP" is a cutting-edge platform for developing multiple cell therapy assets, leveraging the remarkable potential of universal umbilical cord-derived blood stem cells. These cells serve as a foundational source, which we can expand, differentiate, and de-differentiate using our proprietary chemical reprogramming methods to produce pure, powerful rare cell states, at large scale for clinical applications. Complementing this, our "Cell BioEngineAI" platform employs artificial intelligence to predict donor availability and target characteristics, ensuring the safety and efficacy of HLA-compatible therapies. Our goal is to create a ready-to-use, 'off-the-shelf' cell bank with therapeutic products that cover over 85% of the population, available at selected blood centers. For more information, visit www.cellbioengines.com and follow us on www.twitter.com/cell_bioengines

At Charles River, we are passionate about our role in improving the quality of people’s lives. Our mission, our excellent science and our strong sense of purpose guides us in all that we do, and we approach each day with the knowledge that our work helps to improve the health and well-being of many across the globe.

Cimeio Therapeutics is a biotechnology company based in Massachusetts, specializing in gene editing, cellular, and immunotherapy. The company focuses on developing Shielded-Cell & Immunotherapy Pairs (SCIP), which are innovative immunotherapies aimed at treating serious diseases such as genetic disorders, hematologic malignancies, and severe autoimmune conditions. Cimeio's proprietary technology platform enables the discovery of novel protein variants that help cells maintain their function while resisting depletion from targeted immunotherapies. Their core products combine modified cell surface proteins with immunotherapies, ensuring the preservation of healthy cells during treatment. Additionally, their Cell-Shielding Technology protects hematopoietic stem cells from immunotherapy-induced depletion, allowing for effective treatment of diseased cells. Cimeio has formed significant partnerships, including a collaboration with Kyowa Kirin to develop novel cell therapies and a research partnership with the University of Pennsylvania to enhance their immunotherapy research capabilities. The company has received recognition for its innovative approach, with features in publications like Forbes and research published in Nature.

Comanche is a biopharmaceutical company developing novel siRNA molecules for the treatment of preeclampsia. Our purpose is to lower the risks of pregnancy and prematurity worldwide by safely sustaining natural pregnancy and allowing for fetal maturation. We envision a world where all pregnant women have access to safe and effective therapies that are evidence-based, cost-effective and sustainable. Our mission is to prevent or treat preeclampsia at its root cause.

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

Cure Rare Disease™ is developing advanced therapeutics for those who have been diagnosed with rare, genetic diseases that have no treatment or cures. Through partnerships and collaboration, our world-renowned researchers build life-saving therapeutics that are intended to stop or reverse the progression of disease.

Dragonfly Therapeutics is a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing therapies that use its novel multispecific antibody technology to harness the body's immune system to bring breakthrough treatments to patients. In addition to its wholly owned clinical assets, Dragonfly has a deep pipeline of wholly owned preclinical candidates discovered using its proprietary platform, as well as productive collaborations with Merck, AbbVie, Gilead and Bristol Myers Squibb in a broad range of disease areas. For more information, email info@dragonflytx.com.

Dyno Therapeutics is pioneering an artificial intelligence (AI) powered approach to gene therapy.Using machine learning and quantitative high-throughput in vivo experimentation, they are inventing new ways to design gene vectors with a focus on cell-targeting capsid proteins from adeno-associated virus (AAV), the most widely-used vector for gene therapies.

At Editas Medicine, we are pioneering the possible. Our mission and commitment is to harness the power and potential of CRISPR gene editing to develop a robust pipeline of medicines for people living with serious diseases around the world. Our goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases.

ElevateBio is a technology-driven company built to power transformative genetic medicines today and for many decades to come. The Company commercializes its enabling technologies, manufacturing capabilities, and industry-leading expertise through partnerships to accelerate development across a breadth of therapeutic approaches and modalities. The ElevateBio ecosystem combines multiple R&D technology platforms – including Life Edit, a next-generation, full-spectrum gene editing platform; comprehensive cell engineering technologies; and an expanded viral and non-viral therapeutic delivery platform – with BaseCamp®, its end-to-end genetic medicine cGMP manufacturing and process development business, to accelerate the discovery and development of advanced therapeutics.

Eterna Therapeutics is developing breakthrough mRNA cell engineering technologies to repair cellular dysfunction and treat a range of therapeutic indications. We and our strategic partners are advancing innovative nucleic acid and cell therapies that offer the hope of radically improving the health outcomes of patients with high unmet medical needs. We are committed to creating a world in which patients and their families have access to effective, life-changing treatments for serious illnesses.

Factor Bioscience develops new technologies in a little-explored area that we believed had enormous potential: messenger RNA ("mRNA").

Fractyl Health is a leader in creating innovative therapies to address the root cause of type 2 diabetes. We were founded based on groundbreaking research and biological insights showing the key role of the intestine in metabolic control. Our therapies are uniquely designed to target and control this controller for the treatment of metabolic disease. Our lead program, Revita DMR, is a first-in-class procedural therapy that is now in late-stage clinical trials for the treatment of type 2 diabetes. Our team is made up of passionate innovators at the intersection of biology and technology, working to deliver better real-world outcomes for people with metabolic disease.

Generation Bio Co., a genetic medicines company, develops gene therapies for the treatment of rare and prevalent diseases. The company is developing a portfolio of programs for rare and prevalent diseases of the liver and retina. It also focuses on the diseases of skeletal muscle, central nervous system, and oncology. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was founded in 2016 and is headquartered in Cambridge, Massachusetts.

GentiBio, Inc., is an early stage biotherapeutics company co-founded by pioneers in Treg biology and synthetic immunology to develop engineered regulatory T cells (EngTregs) programmed to treat autoimmune, alloimmune, autoinflammatory and allergic diseases. GentiBio's proprietary autologous and allogeneic EngTregs platform integrates key complimentary technologies needed to successfully restore immune tolerance and overcome major limitations in existing regulatory T-cell therapeutics. GentiBio is at the forefront of leveraging a unique therapeutic modality that can be used to address the fundamental cause of many diseases that result from overshooting and/or malfunctioning of the immune system.

Halloran Consulting Group is a life science consulting firm providing strategic development, regulatory, quality, clinical, and organizational support to industry leaders in the pharmaceutical, biotechnology, and medical device sectors. Our consultants are subject matter experts with technical and strategic expertise, who deliver a tailored approach to each engagement, successfully propelling our clients to their next inflection point.

HebeCell was founded in 2016 by a group of scientists who wanted to push research in regenerative medicine and immuno-oncology farther than ever before. Our team of researchers includes multiple world-leading experts in stem cell biology, as well as experienced entrepreneurs and business developers. We operate a fully-equipped, state-of-the-art laboratory in Natick, MA, one of the world’s largest biotech hubs. Since 2016, we have grown HebeCell into a leading innovator in our field. Most importantly, we have developed and patented a method of producing allogeneic natural killer (NK) cells indefinitely from induced pluripotent stem cells (iPS cells) by suspending them in a liquid solution within specially engineered bioreactors. No other company or lab has this technology or these capabilities.

HelixNano is building the world's most advanced mRNA platform to enable previously impossible applications across human and non-human biology

Hopewell Therapeutics is discovering, synthesizing and developing the next generation of tissue-targeted lipid nanoparticles (ttLNPs) to bring genomic medicines to patients. We are pursuing opportunities to redefine the non-viral delivery space for novel genomic medicines by designing systemically administered LNPs to specifically target extrahepatic tissues and cells throughout the body. Hopewell has built a robust intellectual property portfolio with an expansive library of ionizable lipids and has established partnerships with several industry leading companies. We are developing our own internal pipeline, initially targeting diseases of the lung, while concurrently exploring the potential of our ttLNP platform for patients with unmet needs in oncology, infectious diseases, rare genetic diseases and neurological disorders.

The Faculty of the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited disease for which there are no cures. They utilize state of the art technologies to correct the genetic mutations underlying the diseases. The Center focuses on AAT Deficiency, Amyotrophic Lateral Sclerosis (Lou Gehrig's Disease), Canavan Disease, Cystic Fibrosis, Tay-Sachs & Sandoff diseases, Retinitis Pigmentosa, Huntington's disease, Hypercholesterimia, and Cardiac Arrhythmia.The Center operates under the umbrella of the Advanced Therapeutics Cluster (ATC) of UMass, whose mission is to apply knowledge flowing from the latest biomedical discoveries, develop new ways to treat diseases, and drive those therapies into clinical trials.

Developing a gene therapy for Leber Congenital Amaurosis Type 16

Headquartered in Cambridge, Massachusetts, Hunterian Medicine LLC is a gene-editing company working to cure genetic diseases by correcting DNA mutations using its innovative CRISPR delivery technology. The company’s patented technology solves the problem of CRISPR delivery with a “2-for-1” genetic element that enables delivery via a single adeno-associated virus (AAV).

We are a pioneering, clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. Our lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to increase PPi and adenosine, enabling the potential treatment of multiple diseases caused by deficiencies in these metabolites. By targeting the PPi-Adenosine Pathway, INZ-701 aims to correct pathological mineralization and intimal proliferation, addressing the significant morbidity and mortality in these devastating diseases. It is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis.

Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology’s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic. View our social media community guidelines at https://www.intelliatx.com/terms-and-conditions/ .

Kelonia is pioneering a new wave of genetic medicines using its next generation gene delivery platform. The company’s simple and elegant cutting-edge in vivo gene delivery technology uses a few potent lentiviral vector-like particles to precisely and efficiently deliver in vivo genetic cargo to the desired target tissue, and only that tissue, every time. With an initial focus on developing transformational therapies for solid tumors and hematologic cancers, Kelonia is building a pipeline of genetic medicines for a wide range of diseases, with the bold goal of bringing genetic medicines to every patient in need.

Korro Bio was founded to turn extraordinary scientific insights into life-altering new treatments for patients. We harness the body’s natural RNA editing machinery to make precise, single-base RNA edits to modulate protein function, addressing the medical needs of patients. Our mission is to rewrite the future for patients living with debilitating disease by repairing the underlying genetic cause.

KSQ Therapeutics has built an industry-leading genome-scale precision functional genomics platform, called CRISPRomics, to pinpoint therapeutic nodes that directly correlate genomic function to disease with unprecedented certainty and speed. The company is deploying CRISPRomics for novel drug development across broad therapeutic areas and is currently advancing a proprietary pipeline of tumor- and immune-focused drug candidates.

Kytopenis an MIT startup developing their proprietary FlowfectTM technology for non-viral delivery of molecules into hard-to-transfect immune cells.

Landmark Bio is a collective endeavor launched by leaders from academia, the life sciences industry, and world-renowned research hospitals to accelerate development and industrialization of next-generation genomic medicine. Inspired by recent advancements in cell and gene therapy, Landmark Bio was established to remove barriers in drug development, create accessible capability, expertise, and solutions, and offer a collaboration platform to advance manufacturing technologies for the new generation of medicines to come. Founding partners include Harvard University, Massachusetts Institute of Technology (MIT), Cytiva, FUJIFILM Diosynth Biotechnologies (FDB), and Alexandria Real Estate Equities, Inc. Other collaborating institutions include Beth Israel Deaconess Medical Center, Boston Children’s Hospital, Mass General Brigham, and the Dana-Farber Cancer Institute. For more information please contact info@landmarkbio.com

Marengo Therapeutics, a clinical stage biotech company, is pioneering an entirely new way to activate the body’s own immune system to mount a rapid, effective, and durable response against cancer. Our unique therapeutic platform selectively boosts T cells, turbocharging potent anti-tumor activity and promoting long-term immunity to keep cancer at bay.

Mass Eye and Ear in Boston is a Harvard teaching hospital dedicated to eye (ophthalmology) and ear, nose, throat, head and neck (ENT) care and research.

MedTherapy is a Boston-based global biotechnology corporation manufacturing cell, gene and immunological therapies for treatment of cancer to make them accessible and affordable for every cancer patient in the world. MedTherapy works in collaboration with Harvard Medical School, Merck, and the 'International Health Organization'​ (IHO) and many leading private and public instutions, organizations and biotechs. Cell, gene and immunological therapies comprise the most sophisticated treatments modern technology has developed. While their success has been deemed a ‘miracle’, however, unfortunately, their cost is exorbitant often running into millions of dollars which is not sustainable for any individual patient, society or country. Therefore, MedTherapy works in collaboration with various organizations- private, public, academia and non-profits in US and globally to develop more streamlined processes, technologies and partnerships to manufacture these sophisticated medical therapies and made affordable for every patient in the world.

The METiS platform (AiTEM) combines state-of-the-art AI data-driven algorithms, mechanism- driven quantum mechanics and molecular dynamics simulations to calculate Active Pharmaceutical Ingredient (API) properties, elucidate API-target and API-excipient interactions, and predict chemical, physical and pharmacokinetic properties of small molecule and nucleic acid therapeutics in specific microenvironments. This enables efficient lead optimization, candidate selection and formulation design. Founded by a team of MIT researchers, serial entrepreneurs and biotech industry veterans, METiS develops and in-licenses novel assets with high therapeutic potential that could benefit from its data-driven platform.

We are a clinical stage biopharmaceutical company pioneering the discovery, development, and commercialization of allogeneic, off-the-shelf, invariant natural killer T (iNKT) cell therapies to treat cancer and other immune-mediated diseases. iNKT cells are a distinct T cell population that combine durable memory responses with the rapid cytolytic features of natural killer (NK) cells. iNKT cells offer distinct therapeutic advantages as a platform for allogeneic therapy in that the cells naturally home to tissues, aid clearance of tumors and infected cells and suppress Graft versus Host Disease (GvHD). Our proprietary platform is designed to facilitate scalable and reproducible manufacturing for off-the-shelf delivery. As such, we believe that our approach represents a highly versatile application for therapeutic development in cancer and immune diseases. We have leveraged our platform and manufacturing capabilities to develop a wholly owned pipeline for both native and engineered iNKT cells.

Modalis Therapeutics Corporation (Tokyo Mothers 4883) is developing precision genetic medicines through epigenetic gene modulation. Modalis is advancing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or histone modification without the need for double- stranded DNA cleavage, gene editing or base editing. Modalis is headquartered in Tokyo with laboratories and R&D facilities in Cambridge,

Multi-Regional Clinical Trials Center is a healthcare organization that engages expert stakeholders to address critical issues in the conduct and oversight of clinical trials.

Mustang Bio, Inc., a clinical-stage biopharmaceutical company, focuses on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. Its pipeline focuses on gene therapy programs for rare genetic disorders, chimeric antigen receptor (CAR) engineered T cell (CAR T) therapies for hematologic malignancies, and CAR T therapies for solid tumors. The company develop MB-107, a gene therapy program, potential curative treatment for X-linked severe combined immunodeficiency, a rare genetic immune system condition in which affected patients do not live beyond infancy without treatment. Its ex vivo lentiviral gene therapy is in two Phase 1/2 clinical trials.

At Myeloid, we have applied our extensive knowledge of advanced gene and cell therapies to build novel platform technologies that harness the innate abilities of myeloid cells. We have translated the learnings from our backgrounds and novel platform to create a clinical-stage, mRNA-immunotherapy pipeline focused on breakthrough therapies for cancer.

Myrtelle Inc. is a patient-centered, innovation-driven gene therapy company transforming the treatment of neurological diseases. Our mission is to develop and deliver novel treatments for devastating disorders of the central nervous system, beginning with our lead program, gene therapy treatment for Canavan disease. Myrtelle’s social media channels are places for community members to connect and learn about Canavan disease and Myrtelle initiatives. Discussion of medical and health topics should never be construed as medical advice. Contact a health care professional in your area for personal medical advice. Myrtelle Inc. does not represent that the information on our social media channels is accurate, complete, reliable, useful, timely, or current and Myrtelle does not undertake an obligation to update any such information. Myrtelle does not endorse any opinions or statements expressed by others on our social media channels. Myrtelle does not expressly endorse opinions or statements expressed by individuals, businesses, or channels we may ‘follow’, ‘like’, or ‘share’. At times, Myrtelle may link to external websites. Myrtelle is not responsible for the content or policies of these websites. Content submitted to our social media channels is public and will not place Myrtelle under any obligation to you.

Nanite is a next-generation non-viral gene delivery company developing a new class of programmable polymer nanoparticles for a range of modalities and indications. The company’s AI-driven platform, SAYER, combines cutting-edge high-throughput experimental and computational methods to design fit-for-purpose delivery vehicles delivering a broad range of genetic cargoes with tissue specificity.

nChroma Bio is a pioneering biotechnology company redefining the future of in vivo targeted genetic medicine to treat a wide array of diseases and bring cures to patients. The company’s integrated product engine tackles significant limitations of existing genetic medicine approaches by enabling safe, precise and specific in vivo delivery. nChroma’s near clinical-stage development candidate, CRMA-1001, is a liver-targeted therapy in development as a potential functional cure for chronic hepatitis B and hepatitis D that leverages the power of epigenetics, nature's innate mechanism for gene regulation. Guided by a world-class team at the forefront of genetic medicine, founded by renowned pioneers in the field, and supported by top-tier investors, the company is uniquely positioned to deliver groundbreaking therapies with programmable tissue specificity, unlocking highly potent, durable and targeted gene regulation for the liver and beyond.

We are developing an analytical system designed to reveal blind spots throughout your bioprocess by providing confident answers on antibody titers and excipient concentrations in 30 seconds with 1 workflow and 1 system.

Obsidian Therapeutics is pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian’s programs apply our cytoDriveTM technology in cell and gene therapy products to control expression of proteins for enhanced therapeutic efficacy, including our lead program cytoTIL15 engineered to make TILs more effective for more patients. Our aspirations are high, and we’re built to deliver – well-funded with blue chip investors, engaged field-leading advisors, strong partnerships with industry leaders, and a highly experienced, dynamic, innovative and collaborative team, collectively focused on delivering transformative therapies in areas of greatest clinical need. Located in the heart of Cambridge with a new laboratory facility in Bedford, we’re proud of our diverse talented team and committed to cultivating an environment of inclusion where we strive to instill a strong sense of belonging, and support each Obsidianite to continuously learn and contribute their best work. We offer competitive salary and benefits, and potential for employee ownership through stock options.

Omega Therapeutics, Inc. operates as a development-stage biopharmaceutical company. Its OMEGA Epigenomic Programming platform is designed to coopt nature’s operating system by harnessing the power of epigenetics, the mechanism for gene control and cell differentiation. The company is developing omega epigenomic controller (OEC) candidates to up-regulate the expression of HNF4a, a transcriptional master regulator as a potential way to restore liver-cell function in patients suffering from chronic liver diseases; to control the expression of genes that have been strongly linked to cell-growth inhibition in patients with diabetes and other conditions to restore the capacity for corneal regeneration; to down-regulate expression of the CXCL1, 2, 3, and IL-8 gene cluster; to control expression of genes implicated in patients with idiopathic pulmonary fibrosis to halt or reverse disease progression and improve disease outcomes; to down-regulate the expression of SFRP1, a protein that inhibits hair growth; and to treat non-small cell lung cancer and small cell lung cancer. It is also developing OTX-2002 to down-regulate c-Myc, an oncogene. The company was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Organogenesis™ is a leading regenerative medicine company focused on the development, manufacture, and commercialization of solutions for the advanced wound care and surgical and sports medicine markets. Organogenesis offers a comprehensive portfolio of innovative regenerative products to address patient needs across the continuum of care.

Orna Therapeutics is a biotechnology company reshaping RNA therapeutics. We are developing the leading circular RNA technology platform (oRNA®) and unprecedented lipid nanoparticle (LNP) delivery solutions – unlocking the therapeutic possibilities of circular RNA and pioneering the next chapter of safe and effective in vivo therapies for the treatment of disease. Website: www.ornatx.com

PepGen Inc., a clinical-stage biotechnology company, focuses on the development of oligonucleotide therapeutics for use in the treatment of severe neuromuscular and neurologic diseases. Its lead product candidate is PGN-EDO51, an EDO peptide that id in Phase I clinical trial to treat individuals with Duchenne muscular dystrophy (DMD). The company is also developing PGN-EDODM1, an EDO peptide-conjugated PMO, which is in Phase 1 clinical trial for the treatment of myotonic dystrophy type 1, as well as EDO therapeutic candidates, such as PGN-EDO53, PGN-EDO45, and PGN-EDO44 for the treatment of DMD. PepGen Inc. was founded in 2018 and is headquartered in Boston, Massachusetts.

Phio Pharmaceuticals Corp. (Nasdaq: PHIO) is a clinical stage biotechnology company whose proprietary INTASYL™ RNAi technology makes immune cells more effective in killing tumor cells. INTASYL is the only self-delivering RNAi technology focused on immuno-oncology therapeutics. INTASYL drugs precisely target specific proteins that reduce the body’s ability to fight cancer, without the need for specialized formulations or drug delivery systems. Phio was co-founded by Nobel Laureate Craig Mello, who was awarded the Nobel prize in 2006 for his discovery of RNAi, which has broad therapeutic applications. Our founders opened up a whole new field of biology when they discovered RNAi, and we continue their pioneering efforts today through our work with INTASYL. We’re committed to discovering and developing innovative cancer treatments for patients, creating new pathways for a cancer-free future. For additional information, visit the Company's website, www.phiopharma.com. #INTASYL #siRNA #RNAi #Phio #immunooncology

Phosphorex provides drug delivery solutions to the pharmaceutical and biopharmaceutical industries, which include drug encapsulation, nanosizing, microemulsion, and surface functionalization and coating. Following is a brief summary of our expertise: • Microencapsulation/nanoencapsulation of small molecule active pharmaceutical ingredients (APIs) or biomolecules into biodegradable microspheres or nanoparticles enables controlled or sustained drug release, protecting APIs from premature degradation as well as increasing circulation time and targeted drug delivery. • Nanosizing can help you transform poorly soluble APIs with low bioavailability into nanocrystals that can be delivered more easily with enhanced solubility and bioavailability. • Microemulsion, in which a poorly soluble API is dissolved in oil and micro-emulsified into an aqueous media to form a homogeneous mix. • Surface functionalization and coating in which we attach a biological entity on the surface of drug loaded microspheres or nanoparticles via either covalent bonding or physical adsorption to enable cell entry, brain-blood barrier crossing, and targeted drug delivery. The services we provide apply to all stages of your drug development efforts. Whether you are testing a new idea, conducting animal studies, or moving to clinical studies, we can assist. With our fully integrated cGMP system, we can assure you that our processes are completely reproducible and scalable. Please visit www.phosphorex.com for more information.

PlatformQ Health is committed to improving health outcomes by providing trusted digital education. We create video-first programs for clinicians, patients, caregivers, advocates and administrators that feature top clinical and advocacy specialists right alongside affected families. Our unique education platform allows participants to connect online from anywhere, and our integrated suite of engagement and outcomes tools allow us to measure the real-world impact of our modules.

Portal is a cell engineering platform company focused on enabling next generation cell engineering and cell analytics across research and clinical applications. We are implementing a simplified approach to intracellular delivery, focused on accelerating advances enabled by the new generation of RNA, gene editing, and AI-driven technologies. Portal’s initial product suite is based on a proprietary mechanical delivery system capable of delivering many different types of cargo to a broad range of cell types. The technology's ability to be used stand-alone or as an integrated component of third-party systems provides a significant opportunity to broadly impact the fields of biological research and cellular therapeutics

Prime Medicine was founded to bring the promise of gene editing to patients. We use Prime Editing, a next-generation technology that can “search and replace” to restore normal genetic function almost anywhere in the genome. Prime Editing searches to find the precise place in the genome to edit and replaces the segment of faulty DNA with a correct copy of DNA. A single Prime Editor can correct the individual mutations found across patients, meaning Prime Editing can potentially address more than 90 percent of known disease-causing genetic mutations. We envision a world where this technology can cure, halt, and ultimately prevent genetic diseases, providing lifelong benefit to patients. Prime Medicine offers the opportunity to shape the future of gene editing and transform how medicines are used to treat disease. Realizing the promise of Prime Editing requires a talented team with diversity of viewpoints and expertise. We’re building a group of dedicated, scientifically curious individuals who are passionate about helping patients live longer, healthier lives.

ProtaGene is a world-leading CRO partner for the biopharmaceutical and gene and cell therapy industries. From research through product commercialization, we provide the most advanced, integrated, and complete protein and gene analytic capabilities and packages for biologic therapeutics as well as gene and cell therapy platforms. Our deep history of analytical leadership in protein-based therapeutics stems from the merger of Protagen Protein Services in Europe and BioAnalytix in the US. In 2021, GeneWerk’s unique capabilities in vector safety, integration site analysis, and bioinformatics were added to our portfolio, establishing ProtaGene. Our combined protein- and gene-based analytical platforms make it the leading analytic service provider across the biopharmaceutical and gene and cell therapy development spaces. The organization operates four sites in Europe and North America and works in advanced therapeutic platforms with leading biopharmaceutical and gene therapy companies worldwide.

We bring together scientists and engineers from diverse fields to better understand the immune system and support human health.

Regel’s technology utilizes a deactivated Cas system (dCas) which targets the epigenome without editing or damaging the DNA. This approach harnesses the natural mechanisms of gene regulation, allowing for efficient and permanent restoration of normal gene expression.

Remedium Bio is a gene therapy company focused on the development of treatments for large unmet clinical needs. To enable this, we developed a revolutionary gene therapy platform technology, which enables safe, effective, and durable delivery of therapeutic genes while allowing for simple post-treatment dose adjustment. The Prometheus™ adjustable gene therapy platform is able to safely and durably replace multi-injection subcutaneously administered biologics, with a single treatment, at a fraction of the cost, and without genetic integration. Our lead product is a disease-modifying treatment for Osteoarthritis that is based on the only clinically validated chondro-regenerative mechanism. Remedium’s pipeline includes gene therapy treatments in the fields of Weight Loss, Type 2 Diabetes, and Stroke.

Repertoire Immune Medicines is a biotechnology company dedicated to creating treatments for diseases based on the power of the human T cell repertoire to eliminate cancer cells, target pathogens, and regulate immune function in autoimmune disease. The company’s proprietary DECODE™ platform provides a comprehensive understanding of the interactions between T cell receptors and their antigens in disease. DECODE uniquely elucidates the entire immune synapse, including the T-cell receptor-epitope pairs that defines it, the HLA context, and the T cell phenotype. This capability enables the creation of new and potentially transformative classes of immune-based medicines. Repertoire believes the ability to decipher these interactions represents one of the greatest opportunities for innovation in medical science. The company is utilizing the discoveries from DECODE to design and develop novel immune therapies for multiple therapeutic areas, such as autoimmune diseases, infectious diseases, and cancer. The team operates from sites in Cambridge, Massachusetts and Zurich. To learn more about Repertoire, please visit www.repertoire.com.

Replimune Group, Inc., a biotechnology company, develops oncolytic immune-gene therapies to treat cancer. It uses its proprietary Immulytic platform to design and develop product candidates that are intended to activate the immune system against cancer. The company's lead product candidate is RP1, a selectively replicating version of herpes simplex virus 1, which is in Phase I/II clinical trials for a range of solid tumors; and that is in Phase II clinical trials for patients with cutaneous squamous cell carcinoma. It is also developing RP2, which is in Phase I clinical trials for an anti-CTLA-4 antibody-like protein in order to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts.

Rhythm is a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity. The company recently announced topline results from pivotal Phase 3 clinical trials in POMC deficiency obesity and LEPR deficiency obesity. Rhythm is also conducting a pivotal Phase 3 trial in Bardet-Biedl syndrome and Alström syndrome. The company is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study in additional rare genetic disorders of obesity, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of these disorders. For healthcare professionals, visit www.UNcommonObesity.com for more information.

Powering a new era of gene therapy. An Important Note About Privacy: Ring Therapeutics is committed to your privacy and will not ask for sensitive personal information such as social security number, date of birth or bank account details via email or social media platforms. We do not interview candidates on Telegram, Wire, Google Hangouts or similar public platforms. Any questions about job listings can be directed to careers@ringtx.com.

RootPath is a synthetic biology company that elevates the throughput of gene function interrogation by orders of magnitude. Our molecular programming-based DNA fragment assembly technology gives life science researchers, drug developers and synthetic biologists unprecedented access to accurate, long synthetic genes and their functional readouts. We enable biology at scale.

We work at the frontier of programmable medicines. We power our bioplatform and product candidates by harnessing evolution and AI. We operate with purpose and urgency on behalf of people everywhere. We aim to generate life-changing impact for the world.

SalioGen is advancing a new category of genetic medicine we call Gene Coding, which works by adding a new genomic code to turn on, off or modify the functions of new or existing genes. We are advancing potentially curative therapies, with an initial focus on developing durable, safe and accessible genetic medicine to more patients with inherited disorders. At SalioGen, we are driven not only by our efforts to realize the immense potential of our science, but also by our dedication and commitment to developing medicines that can make a significant impact on patients’ lives. Our Gene Coding approach is guided by our Exact DNA Integration Technology (EDIT) platform, a genome engineering technology that leverages proprietary mammal-derived enzymes we call Saliogase. With broad applicability, non-viral delivery capabilities of any size gene and significant manufacturing advantages, EDIT has the potential to transform the treatment paradigm for inherited disorders and beyond. Looking ahead, SalioGen plans to explore Gene Coding’s capabilities to streamline cell therapy, cell engineering, and biologics manufacturing with the goal of helping an even broader population of patients in need.

SanegeneBio is an emerging RNAi-based company developing innovative RNA interference (RNAi) therapeutics driven by the cutting-edge delivery technology. Founded in 2021, SanegeneBio was propelled by a team of industry-leading experts and has operations in both the US and China. Since its inception, SanegeneBio has successfully established proprietary chemical modification platform, hepatic and extrahepatic delivery platforms, enabling tissue-specific delivery of a wide range of RNA therapeutics to efficiently knock down disease-causing genes. Our fast-growing RNAi portfolio covers a broad range of therapeutic areas including cardiovascular and metabolic diseases, immunology-related diseases, and nervous system diseases. Among them, our first RNAi drug has entered the clinical stage, and several pipelines are in progress simultaneously. SanegeneBio is committed to creating transformational RNAi medicines through striving for scientific innovation to address unmet medical needs worldwide and change the lives of patients and families.

Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For information on our Community Guidelines, please visit sarepta.com/community-guidelines. We want to share a reminder with all job seekers and candidates regarding the persistence of recruiting fraud. Please read a message about recruiting fraud and steps you can take to protect yourself here: https://www.sarepta.com/recruiting-fraud

SeQure Dx, Inc. is applying genomics technologies for gene editor target profiling to advance discovery, clinical development, and diagnostic solutions. Their GUIDE-seq and ONE-seq technologies enable identification of all potential off-target sites prior to initiating therapy, to allow comprehensive confirmation of actual edits.

seqWell was created to revolutionize NGS library prep to unlock the full potential of today’s DNA sequencing instruments. Our plexWell™ platform is a transformative library prep technology that allows for simple, scalable multiplexing of 100s to 1000s of samples without time- and cost-consuming normalization. plexWell has wide-ranging impact on applications from synthetic biology, targeted sequencing and single-cell analysis to low and high depth sequencing of human, plant, animal and microbial genomes. In addition to plexWell, seqWell is also developing library prep methods for reagent-based approaches for long-molecule phasing and read linking. We invite you to learn more at https://seqwell.com

siRNAgen Therapeutics is a next-generation RNAi platform products company focused on immunology and central nervous system (CNS) diseases. Their proprietary SAMiRNA™ platform leverages modular chemistry to overcome the challenges around delivery. SAMiRNA™ platform has been instrumental in the creation of innovative drugs such as SRN-001, which holds immense promise in treating fibrotic diseases globally. With a firm commitment to innovation and collaboration, siRNAgen continues to push the boundaries of scientific discovery to change the lives of patients worldwide.

Skylark Bio Gene Therapy Hearing Solutions: We believe in the power of innovative medicines to improve human lives by eliminating sensory limitations. Through gene therapy solutions, we aim to initially address the underlying genetic causes of hearing impairment to enhance, or even restore natural communication.

Skyline Therapeutics is a biotechnology company that focuses on the discovery, development and delivery of innovative gene therapy treatments.

SmartPharm Therapeutics, now a subsidiary of Sorrento Therapeutics, is a gene therapy company applying the next generation of non-viral DNA and RNA technologies to treat serious human diseases. Founded with the vision of producing “Biologics from Within,” SmartPharm is focused on bringing together unique technology and developmental capabilities for the treatment of rare diseases including lysosomal storage disorders. SmartPharm Therapeutics is a second-generation gene-encoded therapeutics company that has benefited from the first decades of learning in gene therapy development to produce an evolved therapeutic platform. We integrate these insights with key technologies and engineering capabilities to enable us to produce optimal gene-encoded therapeutics for patients.

SOLA Biosciences (SOLA) offers a new proprietary, TapBoost® technology, to improve target protein production in various pharmaceutical companies and researchers that share our ultimate goal to deliver new therapeutics faster and more efficiently, at a lower overall cost to patients around the world. TapBoost technology is able to efficiently increase the production of target proteins such as antibodies and Fc-fusion proteins as well as many “difficult-to-express” recombinant proteins including therapeutic biologics. In addition to protein production, we anticipate additional pharmaceutical applications of TapBoost including new gene therapies for conformational diseases, drug screening with new disease target proteins, development of innovative biologics, and GMP manufacturing of commercial biologics. SOLA provides access to TapBoost to help promote pharmaceutical research and development in two ways: 1. Manufacture and provide needed proteins to researchers and pharmaceutical companies 2. Licensing Researchers and pharmaceutical companies can license TapBoost to produce needed proteins in-house. Please see our License page for more information.

Solid Biosciences (Solid) is a life science company singularly focused on solving Duchenne muscular dystrophy (Duchenne). We aim to be the center of excellence across the entire disease spectrum, serving as a catalyst to bring together those with the expertise in science, technology, disease management and care. Disease-focused and founded by a family directly impacted, our mandate is simple yet comprehensive - attack the roots of the disease and improve daily life for patients.

Sonata Therapeutics is a new, Boston-based Flagship Pioneering company revolutionizing the world’s understanding of the cellular microenvironment and its importance to human biology and disease. Our product-platform creates drugs based on a systematic, comprehensive characterization of the microenvironment in diseases with high unmet medical need. Through our pioneering science, we identify new targets for therapeutics that induce the release of a programmed diversity of signals with the potential for significant patient impact. Our employees are exceptionally passionate, accountable, collaborative, and innovative. Sonata Therapeutics was founded by Flagship Pioneering, an organization dedicated to the origination and development of first-in-category life sciences companies.  Since 2000, Flagship has originated and fostered the development of nearly 100 scientific ventures resulting in $20+ billion in aggregate value, 500+ issued patents, and more than 50 clinical trials for novel therapeutic agents.  

Strand was founded by biological engineers working together at the Massachusetts Institute of Technology (MIT). Building on the idea of creating smart therapies capable of making sophisticated decisions, they sought to apply this concept to the emerging field of mRNA therapeutics. This area was previously untapped by traditional synthetic biology, leading us to build our very own mRNA programming language, creating the world’s first platform for mRNA smart therapies. Strand’s mRNA programming technology promises to make mRNA therapies safer and more effective by programming the location, timing, and intensity of therapeutic protein expression inside a patient’s body via mRNA-encoded logic circuits. These circuits have the ability to implement cell-type specific expressions by sensing and classifying the unique mRNA expression signatures of cells and control the dosage of protein expression through responses to exogenously administered small molecules.

Based in Boston, MA, STRM.BIO ("storm bio") is a pre-clinical, VC-backed biotechnology company that aims to fulfill the promise of gene therapy by leveraging extracellular vesicles (EVs) to deliver treatment directly to the patient (in vivo). Unlike viruses, EVs are natural carriers of nucleic acids and proteins with innate targeting capabilities, amenability to large scale/commercial manufacture, and low immunogenicity – which make them excellent cell messengers for gene therapies. Our proprietary EV platform is establishing a whole new class of therapeutics, yielding new therapies and making existing ones more effective. At STRM.BIO, we believe that our work has the potential to democratize gene therapy and bring hope for patients fighting rare diseases worldwide. We are currently recruiting team members who can help us make our vision a life-saving reality.

Stylus Medicine is developing transformative in vivo genetic medicines to unlock cures. Stylus combines engineered recombinases with non-viral delivery to specifically encode therapeutics. The company’s approach is versatile and modular, with potential therapeutic application across oncology, autoimmune, genetic diseases, and beyond.

Sumitomo Pharma America (SMPA) is a science-based, technology-driven biopharmaceutical company focused on delivering therapeutic and scientific breakthroughs in areas of critical patient need in psychiatry and neurology, oncology, urology, women’s health, rare disease, and cell and gene therapies. SMPA was formed through the consolidation of Sumitomo Pharma’s U.S. affiliate companies including Sunovion Pharmaceuticals, Inc., Sumitomo Pharma America Holdings, Inc., Sumitomo Pharma Oncology, Inc., Sumitovant Biopharma, Inc., Myovant Sciences, Inc., Urovant Sciences, Inc. and Enzyvant Therapeutics, Inc. SMPA is a Sumitomo Pharma company.

Syd Labs, a recombinant company based in Boston, Massachusetts, is dedicated to providing quality products and services for research and development in academy, industry, and government. We have always tried our best to let you, our valuable customers and partners, save money and work better. Syd Labs, Inc provides very comprehensive and competitive services for antibody services, from peptide synthesis, gene synthesis, protein expression, rabbit and mouse polyclonal antibody development, mouse, rat, and rabbit monoclonal antibody development, cloning variable regions of hybridoma cell lines, antibody humanization and engineering, stable cell line generation, ELISA assay development, and so on. The lowest prices are guaranteed. If you find any US company providing the same quality services with lower prices, we will return the difference to you.

SynerK's founders are RNA-targeted therapy industry veterans with decades of drug development experience. Our expertise covers from discovery, preclinical and clinical development to commercial launch. Millions suffer from life threatening diseases, yet only few benefits from current treatment. There is so much we understood but also much more to understand. We strive to find a way to treat disease where a genetic defect is present. One patient at a time, the number will add up.

SYTE.bio is a Synthetic Biology Start-Up company developing precision DNA & RNA-based therapeutics and vaccines. The company has been incorporated as a Delaware C Corp in 2021 (SYTE.bio, Inc), and has labs & offices in Argentina's headquarter, that has been operating since 2017. SYTE.bio is pioneering the development of life-changing treatments for severe diseases and vaccines to treat and prevent diseases at the molecular and cellular level using engineered DNA, improving life through innovation. SYTE.bio’s powerful redosable DNA & RNA delivery platforms will unleash the potential of personalized genetic medicine in multiple potential applications, such as Gene Therapy (Oncology, Monogenic Diseases, Cardiovascular Diseases, Vaccines, Orphan Diseases), Cell Therapy (CAR-T Therapy, Induced Pluripotent Stem Cells (iPSC)) and Gene Editing (CRISPR/Cas, TALEN, ZFN).

Tessera Therapeutics is pioneering Gene Writing™—a new genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source.

Pioneering tRNA therapeutics to modulate mRNA function and cure a broad range of genetic diseases.

Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD.

TransCode's lofty goal is delivering a cancer-free future for all cancer patients. Our core belief is that cancer can be overcome through the intelligent design and efficient delivery of targeted therapeutics. Our lead therapeutic candidate, TTX-MC138, targets microRNA-10b, or miRNA-10b, considered the master regulator of metastatic cell viability in a range of cancers, including breast, pancreatic, ovarian, colon cancer, glioblastomas, and several others. Our other preclinical programs include two solid tumor programs, TTX-siPDL1, an siRNA-based modulator of programmed death-ligand 1, or PD-L1, and TTX-siLIN28B, an siRNA-based inhibitor of RNA-binding protein LIN28B. TransCode also has three cancer agnostic programs, TTX-RIGA, an RNA- based agonist of the retinoic acid-inducible gene I, or RIG-I, targeting activation of innate immunity in the tumor microenvironment; TTX-CRISPR, a CRISPR/Cas9-based therapy platform for the repair or elimination of cancer-causing genes inside tumor cells; and TTX-mRNA, an mRNA-based platform for the development of cancer vaccines that activate cytotoxic immune responses against tumor cells. Until now, targeting biomarkers that are the primary drivers of cancer onset, progression and recurrence have been locked behind the challenge of delivery – cytosolic delivery to engage these known targets. TransCode believes it has overcome this barrier, opening the door to a broad array of cancer therapies -- a disease with the highest unmet need that affects everyone. It is through the design and optimization of this technology that TransCode has devised an elegant solution -- TTX -- a proprietary delivery platform for the transport of targeted therapeutics to cancer cells. With their world class team and know how, TransCode has a rapidly expanding platform of drug candidates designed to target a variety of tumor indications with the goal of long-term treatment survival.

TScan Therapeutics, Inc., a preclinical-stage biopharmaceutical company, focuses on developing a pipeline of T cell receptor-engineered T cell, or TCR-T, therapies for the treatment of patients with cancer. It is developing TSC-100 and TSC-101 for the treatment of patients with hematologic malignancies to eliminate residual leukemia and prevent relapse after hematopoietic stem cell transplantation; and TSC-200, TSC-201, TSC-202, and TSC-203 for the treatment of solid tumors. The company has a collaboration and license agreement with Novartis Institutes for Biomedical Research, Inc. to discover and develop novel TCR-T therapies. TScan Therapeutics, Inc. was incorporated in 2018 and is headquartered in Waltham, Massachusetts.

Vericel is a leading provider of advanced therapies for the sports medicine and severe burn care markets. The Company combines innovations in biology with medical technologies, resulting in a highly differentiated portfolio of innovative cell therapies and specialty biologics that repair injuries and restore lives. We currently market two autologous cell therapy products and one specialty biologic product in the United States. Personal integrity, teamwork, collaboration, and innovative technology are the foundations of our work. We seek to practice transparency in our clinical trials and research, and in our relationships with each other, our patients, and the investors who support us. PRIVACY AND COMMUNITY GUIDELINES When you engage with Vericel social media channels and our content providers on social media, you also agree to abide by the following Community Guidelines (found here: https://bit.ly/3pg3UKQ), which apply in addition to the Terms and Conditions or other legal notices specific to each social media channel. Please note that third-party social media channels also have access to information you share via social media. For more details, read LinkedIn's Privacy Policy here: https://bit.ly/3GF2r5Y. For additional information about privacy at Vericel, please see our Privacy Policy (bit.ly/43ErZcx) and Terms and Conditions of Use (bit.ly/4568Cdx). If you engage with us, you consent and give Vericel permission to communicate with you and to use any information, text and media associated with the interaction or post.

Verve Therapeutics, Inc., a genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases. Its lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver. The company also engages in the development ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is based in Cambridge, Massachusetts.

We are a cell therapy company that combines a novel patient engineering approach with targeted therapies to provide a solution for patients suffering from hematological malignancies.

Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com. Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc.

Waters Corporation, a global leader in analytical instruments and software, has pioneered innovations in chromatography, mass spectrometry, and thermal analysis serving life, materials, and food sciences for more than 60 years. With approximately 8.000 employees worldwide, Waters operates in 35 countries, with 14 manufacturing facilities, and products available in 100 countries.

Welgen Inc is a Biotechnology company located in 25 Winthrop St, Worcester, Massachusetts, United States.

Xenetic Biosciences, Inc. (NASDAQ: XBIO) is a biopharmaceutical company focused on progressing XCART, a personalized CAR T platform technology engineered to target patient-specific tumor neoantigens. The Company is initially advancing cell-based therapeutics targeting the unique B-cell receptor on the surface of an individual patient’s malignant tumor cells for the treatment of B-cell lymphomas. XCART has the potential to fuel a robust pipeline of therapeutic assets targeting high-value oncology indications. Additionally, Xenetic is leveraging PolyXen™, its proprietary drug delivery platform, by partnering with biotechnology and pharmaceutical companies. PolyXen™ has demonstrated its ability to improve the half-life and other pharmacological properties of next-generation biologic drugs. The Company has an exclusive license agreement with Takeda Pharmaceuticals Co. Ltd. in the field of coagulation disorders and expects to earn royalty payments under this agreement.

La Laiterie du Berger est une entreprise familiale sénégalaise, créée en 2006. Notre mission est de collecter le lait frais des éleveurs de notre pays, pour offrir chaque jour aux consommateurs sénégalais des produits laitiers, au lait local et naturel, connus sous la marque Dolima. C’est ce qui fait que la Laiterie du Berger n’est pas une entreprise comme les autres, et que le yaourt Dolima n’est pas un yaourt comme les autres. Notre engagement s’incarne dans notre mission sociale : construire une filière sénégalaise plus forte, avec un élevage plus productif, en mesure d’approvisionner le marché local. Il s’agit aussi de plus en plus d’aller vers la nutrition, pour apporter la santé par une alimentation saine et accessible dans notre pays où cet enjeu est si grand.