Esteve Pharmaceuticals S.A. operates as a global pharmaceutical company. It provides Ophthalmology and other Pathologies therapies. The company is headquartered in Barcelona, Spain.

The Board of Regents of The University of Texas System, a subsidiary of The University of Texas at Austin, is an American company located in Austin, TX. The firm oversees college activities.

Institut National de la Sant? et de la Recherche M?dicale provides medical services. The firm specializes in biomedical research and public health research. The company is headquartered in Paris, Cedex, France.

Sanofi Genzyme is the specialty care global business unit of Sanofi, focused on rare diseases, multiple sclerosis, immunology, and oncology. They help people with debilitating and complex conditions that are often difficult to diagnose and treat. They are dedicated to discovering and advancing new therapies, providing hope to patients and their families around the world. Genzyme became part of Sanofi in 2011. While continuing to focus on rare diseases, they also established ourselves as a leader in the field of multiple sclerosis. In 2016, their focus expanded to include Sanofi’s programs in oncology and immunology. Their approach is shaped by a long history of developing highly specialized treatments and forging close relationships with physician and patient communities. Founded as Genzyme in Boston in 1981, they rapidly grew to become one of the world’s leading biotechnology companies, recognized for their pioneering development of treatments for rare genetic disorders and their innovative contributions to medical science and biomanufacturing.

The University of Iowa Research Foundation (UIRF) - a 501C3 corporation - commercializes University of Iowa developed technologies and inventions through licensing and new venture formation, and manages the subsequent revenue stream.

Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases and a robust intellectual property portfolio with issued composition of matter patents in the United States for its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines.

Spirovant Sciences, Inc. is a gene therapy company, which engages in the development of gene therapies for cystic fibrosis and other lung diseases. It offers AAV platform which consists of a highly optimized transgene payload, a novel AAV capsid, and an augmenter; and lentiviral platform, a pseudotyped with an envelope protein which has demonstrated capability to transduce human airway epithelia from apical surfaces. Its pipeline include SPIRO-2101, SPIRO-2102, and SPIRO 2110. The company was founded by Joan Lau and Eric Yuen and is headquartered in Philadelphia, PA.

Oxford University Innovation is the University of Oxford's technology transfer company and manages the University's intellectual property portfolio, working with University researchers on identifying, protecting and marketing technologies through licensing, spin-out company formation and material sales. Oxford University Innovation also manages Oxford University Consulting, which arranges consulting services providing clients access to the expertise of the University's academics. Oxford University’s overarching Strategic Plan begins, “The University of Oxford aims to lead the world in research and education” immediately followed by, “They seek to do this in ways which benefit society on a national and a global scale.” Oxford University Innovation helps staff and students to apply their expertise and research for wider social and economic benefit. Their role is to help University staff and students bring the benefits of their research and expertise to create impact in wider society. They support Oxford’s researchers, staff and students, offering commercial skills and a range of specialist resources in order to maximise research impact. Income is shared with those involved according to the University’s Regulations.

Oxford Genetics is a UK-based biotechnology company specializing in the production of versatile cloning plasmids for research in academic and biotechnology institutions. They also provide custom cloning and DNA synthesis. All of their products are free of intellectual property restrictions.

GenSight Biologics develops gene therapy-based treatments for retinal degenerative diseases. GenSight Biologics is a privately owned biopharmaceutical company, dedicated to the development and commercialization of gene therapy-based treatments of retinal degenerative diseases. Based on recent results obtained by the teams of its scientific founders, the company develops innovative approaches to prevent retinal degeneration in selected pathological conditions and to restore vision in patients suffering from very low vision or blindness.

Lonza Houston, Inc. produces viral-based therapeutics. The company was founded in 1995 and is headquartered in Houston, TX.

Vivet Therapeutics is dedicated to developing innovative gene therapy treatments for orphan diseases. Vivet Therapeutics is focused on optimizing gene therapy through a partnership with the Fundacion para la Investigacion Medica Aplicada (FIMA) at the Centro de Investigación Medica Aplicada (CIMA, Universidad de Navarra) to develop new AAV vectors specifically targeting the liver and generating new technologies to optimize gene delivery and long term expression.

UAB Research Foundation (doing business as UABRF), a subsidiary of The University of Alabama System, is a company headquartered in Birmingham, AL. The firm engages in identify, assess, and market commercially viable technology developed at The University of Alabama at Birmingham.

The Research Institute at Nationwide Children's Hospital is one of America's ten largest free-standing pediatric research centers and works to enhance the health of children by engaging in research.

Generation Bio is an innovative genetic medicines company focused on creating a new class of gene therapy to provide durable, redosable treatments for patients suffering from both rare and prevalent diseases. The company’s non-viral platform incorporates a proprietary high-capacity DNA construct called closed-ended DNA, or ceDNA; a novel cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The company is designing therapies to provide targeted delivery of genetic payloads that include large and multiple genes across a broad array of tissues, and to be redosable for individualized and extended treatment throughout a patient’s life. The platform is designed to expand access to treatments for rare diseases and to address prevalent diseases through efficient, scalable manufacturing.

Viral Vector Technology as key enabler for innovative therapeutics. Viral vectors are a tool commonly used by molecular biologists to deliver genetic material into cells. This technique enables research of gene function and innovative therapeutics designed to correct defective genes and cancer. These technologies also assist in the discovery of new drug, food and cosmetic compounds. SIRION is a global leader in viral vector technology for gene modification of mammalian cells. Along with custom services, the company stands ready to outlicense its unique technologies. An initial target currently being pursued by one of SIRION's partners is a gene therapeutic for cervical cancer. The company started business in 2007 and since then has been changing the paradigm of viral vector supplies. Specifically, all commonly applied vector types are now available within a matter of weeks - adenovirus, adeno-associated virus (AAV) and lentivirus - and a full range of virus related services is at your fingertips spanning from particle production to virus driven cell modelling, all at highest titers and in quantities required for preclinical, clinical and animal testing. The company is easily accessible with presence in Munich, Tokyo, New Hampshire and Tel Aviv. Commercial arrangements range from fee-for-service over exclusive rights on single clones all the way to milestone & licensing arrangements. The business model proves successful: an annual 70% growth since 2007 and experienced in more than 500 projects for both the academic and industry sector. The company is privately held and has over the years raised funds from 8 investors both private and government. In order to support further growth and to benefit from technology development in this exciting industry, the company welcomes new investors. Please contact Management for further information about the company and its future plans.

Akouos, Inc. is a precision genetic medicine company, which engages in the development of precision genetic medicines to restore and preserve hearing in genetically-defined patient populations. The firm's lead product candidate, AK-OTOF, is a gene therapy for the treatment of hearing loss due to mutations in the OTOF gene. It develops targeted adeno-associated viral vector based gene therapies for sensorineural hearing loss, which results from dysfunction or damage to sensory cells and nerve fibers of the inner ear. The company was founded by Emmanuel J. Simons, William F. Sewell, Richard H. Smith, Luk H. Vandenberghe, and Michael J. McKenna on March 7, 2016 and is headquartered in Boston, MA.

Gene therapy is defined as “the administration of a gene or cells with introduced genes into the human body to treat diseases”, according to the Ministry of Education, Culture, Sports, Science and Technology and the Ministry of Health, Labor and Welfare in Japan. Gene therapy research is progressing globally. In Japan, clinical studies have already been conducted for some diseases. The age of gene therapy is about to begin. In our research, we have found a unique gene therapy technique that is expected to have great therapeutic effects: an innovative approach using the highly safe adeno-associated virus (AAV) as a vector to carry therapeutic genes. Based on extensive research, we are convinced that we can provide world-leading gene therapies for refractory diseases for which effective therapies have not been established yet. Primarily focusing on Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and Alzheimer’s disease, we established the Gene Therapy Research Institution Co., Ltd. (GTRI) to realize this innovative research in practice, in the fastest way from basic research to clinical studies with the goal of popularizing gene therapy. Our gene therapy approaches are expected to have long-term effects via single administration; therefore, we believe that they will reduce medical expenses in our super-aged society and ensure their global competitiveness against other approaches. GTRI aims to be the world-leading gene therapy provider based in Japan and makes every effort to pursue safer virus vectors and more effective transgene technology. Our mission is to use globally available knowledge and technology for practical applications and specifically to provide these therapies to patients suffering from refractory diseases worldwide.

Behringwerke AG, a subsidiary of Sanofi, is a German company located in Marburg. The firm produces specialty chemicals and pharmaceutical products.