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Applied Genetic Technologies Corporation, a clinical-stage biotechnology company, develops transformational genetic therapies for patients suffering from rare and debilitating diseases. Its advanced product candidates include three ophthalmology development programs across two targets, including X-linked retinitis pigmentosa, which is in the Phase 1/2 clinical trials; and achromatopsia that is in Phase 1/2 clinical trials. It is also developing an optogenetic product candidate to treat advanced retinal disease. In addition, the company has initiated one preclinical program in otology; preclinical program in dry age-related macular degeneration; and two preclinical programs in targeting central nervous system disorders, including frontotemporal dementia and amyotrophic lateral sclerosis. It has collaboration agreements with University of Florida; Bionic Sight, Inc.; and Otonomy, Inc. The company was incorporated in 1999 and is headquartered in Alachua, Florida.

Microbot Medical Inc. operates as a pre-clinical medical device company in the United Kingdom. It primarily engages in the research, design, and development of next generation robotic endoluminal surgery devices targeting the minimally invasive surgery space. The company also offers LIBERTY, an endovascular robotic surgical system which allows physicians to conduct a catheter-based procedure from outside the catheterization laboratory, and avoid radiation exposure, physical strain, and the risk of cross contamination for use in cardiovascular, peripheral, and neurovascular spaces. In addition, it provides NovaCross, an intellectual property and technology in the field of intraluminal revascularization devices with anchoring mechanism and integrated microcatheter. The company also has a strategic collaboration agreement with Stryker Corporation for technology co-development. Microbot Medical Inc. was founded in 2010 and is based in Braintree, Massachusetts.

Renovacor, Inc., a biotechnology company, focuses on delivering various precision therapies to enhance the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. It primarily focuses on the treatment of BCL2-associated athanogene 3 (BAG3) mutation-associated dilated cardiomyopathy (DCM). The company's lead product candidate is REN-001, a recombinant adeno-associated virus 9-based gene therapy designed to deliver a functional BAG3 gene to augment BAG3 protein levels in cardiomyocytes, and slow or halt progression of BAG3 DCM. It is also developing a pipeline of BAG3-associated gene therapies for diseases with high unmet medical need associated with mutations in the BAG3 gene and mechanistically linked to BAG3's expression and function. The company was founded in 2013 and is based in Greenwich, Connecticut.

Protara Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in advancing transformative therapies for the treatment of cancer and rare diseases. The company's lead program is TARA-002, an investigational cell therapy, which is in Phase II clinical trial for the treatment of non-muscle invasive bladder cancer and lymphatic malformations. It is also developing intravenous choline chloride, an investigational phospholipid substrate replacement therapy that is in Phase II clinical trial for patients receiving parenteral nutrition. The company is headquartered in New York, New York.

X4 Pharmaceuticals, Inc., a late-stage clinical biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases. Its lead product candidate is mavorixafor, a small molecule inhibitor of the chemokine receptor C-X-C chemokine receptor type 4 (CXCR4), which is in Phase III clinical trial for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome; and Phase II clinical trial to treat congenital, idiopathic, or cyclic neutropenia. The company is also developing X4P-003, a CXCR4 antagonist for the treatment of CXCR4 dependent disorders and primary immunodeficiencies; and X4P-002, a CXCR4 antagonist for the treatment of blood-brain barrier diseases. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications. The company is headquartered in Boston, Massachusetts.