Jane Elizabeth Machin

Genzyme Corp. engages in development and manufacture of specialty therapeutic, surgical and diagnostic product. It serves in the areas of rare genetic diseases, multiple sclerosis, cardiovascular diseases and endocrinology. The company was founded on June 8, 1981 and is headquartered in Cambridge, MA.

Scioderm, Inc. engages in the development of innovative therapies for areas of high unmet need, including orphan products. The company was founded by Robert Ryan and Robert John Coull in 2012 and is headquartered in Durham, NC.

Duke University is an academic university that offers undergraduate, graduate and professional programs. It also has publications and research services. The university was founded in 1838 and is headquartered in Durham, NC.

BioNJ, Inc. is based in Trenton, NJ. BioNJ, Inc. is a life sciences trade association with nearly 400 member companies. The company supports the discovery, development, and commercialization of therapies and cures that save and improve lives. BioNJ drives capital formation, fosters entrepreneurship, advocates for medical innovation, provides access to talent and education, and offers critical commercial resources. The company also serves as a clearinghouse for funding information and opportunities. The company was founded in 1994 by James J. Marino, Jayne C. Gershkowitz, Abraham Abuchowski. The CEO is Debbie Hart.

National Tay-Sachs & Allied Diseases Association is a non-profit organization. The firm engages in the provision of funding, research, and support services for individual affected with Tay-Sachs, Canavan, Sandhoff, GM1 gangliosidosis, and related diseases. The company was founded in 1957 and is headquartered in Boston, MA.

ARM is a Washington, DC-based multi-stakeholder advocacy organization that promotes global initiatives necessary to facilitate access to life-giving advances in regenerative medicine. The organization promotes legislative, regulatory reimbursement, investment, technical, and other initiatives to accelerate the development of safe and effective regenerative medicine technologies. ARM also works to increase public understand- ing of the field and its potential to transform human healthcare. Prior to the formation of ARM in 2009, there was a need for more coordinated and cohesive advocacy representing the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today ARM has more than 140 members and is the leading global advocacy organization in this field.

NightstaRx Ltd. develops and commercializes therapies for retinal dystrophies. It offers retinal gene therapy that provides one-time cure for choroideremia (a rare inherited cause of blindness) and other retinal dystrophies. The company was founded by Robert MacLaren, Miguel Seabra, and Matthew During in January 2014 and is headquartered in London, the United Kingdom.

Founded in 2016, Amicus Therapeutics International Holding Ltd. is a British company that functions as an investment holding company. Part of Amicus Therapeutics, Inc., the company is based in Marlow, UK.

Part of Amicus Therapeutics, Inc., Amicus Therapeutics GmbH is a German biotechnology company that develops therapies for rare and orphan diseases. The company is based in Munich, Germany. The CEOs are Lori Elizabeth Mochan, Christian Ruppenthal, Anita Jane Clifford. The company was founded in 2002.

Founded in 2019, Tungsten Bidco Ltd. is a British company that functions as an investment holding company. Part of Biogen, Inc., the company is based in Maidenhead, UK.

Gennao Bio, Inc. is a privately-held genetic medicines company that is developing first-in-class, non-viral, targeted nucleic acid therapeutics utilizing its proprietary gene monoclonal antibody (gmab) platform technology. The company is based in Pennington, NJ. The company's gmab technology uses a novel, cell-penetrating antibody to non-covalently bind to, and deliver therapeutic levels of a wide variety of nucleic acid payloads, to select cells. Gennao Bio is initially focusing on addressing significant unmet needs in oncology and rare monogenic skeletal muscle diseases. The company's non-viral delivery platform offers significant advantages over traditional gene delivery systems as it can deliver multiple types of nucleic acids, allows for repeat dosing and dose optimization, and can be easily and affordably manufactured. The company was founded in 2020 by Bruce Turner, Elias Quijano, Peter M. Glazer, Stephen P. Squinto. Christopher M. Duke has been the CEO of the company since 2021.