List of Adeno-associated viral vector (AAV) Companies in Massachusetts - 44

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.

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).

Our mission is to transform the lives of people living with rare diseases and devastating conditions through the development and delivery of innovative medicines, as well as through supportive technologies and healthcare services. By continuing to deepen our understanding of rare disease, which began with our pioneering work in complement biology, we are able to innovate and evolve into new areas where there is great unmet need and opportunity to help patients and families fully live their best lives. Our culture is rooted in integrity, inclusiveness, and our dedication to joining and supporting the communities in which we live and work. We invest in and value people who believe in the importance of our purpose and understand what it takes to deliver on it. Alexion has over 3,000 talented colleagues dedicated to serving people living with rare diseases in more than 50 countries around the world. Our global headquarters are based in Boston, Massachusetts and our EMEA headquarters are in Zürich, Switzerland. We also have a Research Center of Excellence in New Haven, Connecticut, global supply chain and operations headquarters in Ireland, as well as local and regional operations in countries around the world. At Alexion, our passion drives us to continuously innovate and create meaningful value in all we do. In doing so, we change lives for the better – ours, people living with rare diseases, and the communities we serve. Every day.

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.

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.

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.

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

Broad Institute of MIT and Harvard was launched in 2004 to improve human health by using genomics to advance our understanding of the biology and treatment of human disease, and to help lay the groundwork for a new generation of therapies.COVID-19: EUA (03/2021) for CRSP SARS-CoV-2 Real-time Reverse Transcriptase-PCR Diagnostic Assay

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.

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.

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.

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.

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

Flagship Pioneering is a biotechnology company that invents and builds platform companies, each with the potential for multiple transformative human health and sustainability products. Since its launch in 2000, Flagship has originated and fostered more than 100 scientific ventures. The current Flagship ecosystem comprises over 40 companies, including Denali Therapeutics, Foghorn Therapeutics, Generate:Biomedicines, Inari, Indigo Agriculture, and Tessera Therapeutics.

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.

Harvard Bioscience is a global developer, manufacturer and marketer of a broad range of specialized products, primarily apparatus and scientific instruments used to advance life science research at pharmaceutical and biotechnology companies, universities and government laboratories worldwide. We sell our products to thousands of researchers in over 100 countries through our full-line catalog (and various other specialty catalogs), our websites, and through distributors, including GE Healthcare, Thermo Fisher Scientific Inc., and VWR. We have sales and manufacturing operations in the United States, the United Kingdom, Germany, and Spain and sales facilities in France and Canada.

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.

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).

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/ .

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.

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,

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.

Northeastern University is a private research university with its main campus in Boston, and multiple satellite campuses.

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.

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.

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.

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

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

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.

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.

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

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.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. We discovered and developed the first medicines to treat the underlying cause of cystic fibrosis (CF), a rare, life-threatening genetic disease. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases. Founded in 1989 in Cambridge, Massachusetts, our corporate headquarters is now located in Boston’s Innovation District, and our international headquarters is in London, United Kingdom. We currently employ approximately 3,500 people in the United States, Europe, Canada, Australia and Latin America with nearly two-thirds of our staff dedicated to research and development. Vertex is consistently recognized as one of the industry’s top places to work by Science Magazine, The Boston Globe, Boston Business Journal and the San Diego Business Journal. Our research and medicines have also received esteemed recognitions, including the Robert J. Beall Therapeutics Development Award, the French Prix Galien and the British Pharmacological Society awards.

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.

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