Abeona Therapeutics Inc.

Krystal Biotech, Inc. is a clinical-stage biotechnology company focused on developing redosable gene therapies to address severe orphan diseases across the United States. Its most advanced therapeutic candidate, beremagene geperpavec (B-VEC), is currently undergoing Phase III clinical evaluation for the treatment of dystrophic epidermolysis bullosa. The company's pipeline also includes several other promising programs: KB105 is in Phase I/II clinical trials for patients suffering from deficient autosomal recessive congenital ichthyosis; KB301 is also in Phase I/II development, aiming to ameliorate wrinkles and other signs of aging or damaged skin. Additionally, Krystal Biotech is conducting preclinical research on KB407 for cystic fibrosis and KB104 for Netherton syndrome. In its earlier discovery phase, the company is exploring candidates such as KB5xx for chronic skin conditions and multiple KB3xx programs targeting aesthetic dermatological concerns. Krystal Biotech, Inc. was established in 2015 and is based in Pittsburgh, Pennsylvania.

Avalo Therapeutics, Inc. is a precision medicine company in the clinical development phase, dedicated to identifying, advancing, and marketing specialized treatments for patients experiencing critical unmet medical needs across the fields of immunology, immuno-oncology, and rare genetic disorders. Among its key pipeline assets is AVTX-002, a fully human monoclonal antibody designed to inhibit LIGHT. This drug is currently undergoing Phase II clinical trials for non-eosinophilic asthma and inflammatory bowel diseases, specifically moderate to severe Crohn's disease and ulcerative colitis. Furthermore, AVTX-002 is also being investigated in Phase III for its potential to treat acute respiratory distress syndrome caused by COVID-19. The company is also advancing AVTX-007, a fully human monoclonal antibody targeting IL-18, which is progressing through Phase I clinical studies for Still's disease, encompassing both adult-onset Still's disease and systemic juvenile idiopathic arthritis. Avalo's late-stage pipeline includes two products addressing rare genetic conditions, both in Phase III clinical trials: AVTX-801, a D-galactose substrate replacement therapy for phosphoglucomutase 1 deficiency (PGM1), also known as PGM1-CDG; and AVTX-803, an L-fucose substrate replacement therapy intended for LADII, medically referred to as SLC35C1-CDG. Established in 2011, the corporation initially operated as Cerecor Inc. before rebranding as Avalo Therapeutics, Inc. in August 2021. Its main operations are situated in Rockville, Maryland.

AskBioBiotechnologyUnited States

69% match

AskBio is a clinical-stage gene therapy company that develops adeno-associated virus (AAV) gene therapies to treat genetic and complex disorders. The company focuses on advancing genetic medicine through its proprietary technology platforms, manufacturing processes, and clinical trials.

Atsena Therapeutics, Inc.BiotechnologyUnited States

68% matchPrivate

Atsena Therapeutics, Inc. is a clinical-stage gene therapy company headquartered in Durham, North Carolina, established in 2019. The firm focuses on developing novel gene therapies to treat debilitating inherited blinding diseases using proprietary vector delivery technologies. Its scientific pipeline aims to address areas of high unmet medical need in ophthalmology.

Rinascera Therapeutics, Inc.BiotechnologyUnited States

68% match

Rinascera Therapeutics, Inc. is a clinical-stage biotechnology company located in Boston, Massachusetts, dedicated to transforming the treatment of rare genetic skin diseases.

Neurogene Inc. is dedicated to engineering transformative genetic treatments for individuals and their families grappling with debilitating neurological conditions. Its current pipeline prominently features two key investigational therapies: NGN-401, an AAV9 gene therapy specifically targeting Rett syndrome, and NGN-101, which is being developed to address neuronal ceroid lipofuscinosis subtype 5 (Batten disease). The company's operations are based in New York, New York.