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Forma Therapeutics Holdings, Inc., a clinical-stage biopharmaceutical company, focuses on development and commercialization of novel therapeutics for treatment of rare hematologic diseases and cancers. Its core product candidates for development include FT-4202, which is Phase 1 trial for the treatment of sickle cell disease and other hemoglobinopathies; and FT-7051 for the treatment of metastatic castration-resistant prostate cancer. The company is also developing Olutasidenib, a selective inhibitor for cancers with isocitrate dehydrogenase 1 gene mutations, which is in Phase II trial for the treatment of relapsed/refractory acute myeloid leukemia and Phase I trial to treat glioma; and FT-8225, a liver-targeted fatty-acid synthase inhibitor designed to block de novo lipogenesis in the liver. Forma Therapeutics Holdings, Inc. was founded in 2007 and is headquartered in Watertown, Massachusetts. As of October 14, 2022, Forma Therapeutics Holdings, Inc. operates as a subsidiary of Novo Nordisk A/S.

Immunovant, Inc., a clinical-stage biopharmaceutical company, develops monoclonal antibodies for the treatment of autoimmune diseases. It develops batoclimab, a novel fully human monoclonal antibody that target the neonatal fragment crystallizable receptor for the treatment of myasthenia gravis, thyroid eye disease, chronic inflammatory demyelinating polyneuropathy, and Graves diseases, as well as warm autoimmune hemolytic anemia. The company is headquartered in New York, New York. Immunovant, Inc. operates as a subsidiary of Roivant Sciences Ltd.

Krystal Biotech, Inc., a commercial-stage biotechnology company, discovers, develops, and commercializes genetic medicines for patients with rare diseases in the United States. It commercializes VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB). The company also develops KB105, which is in Phase 1/2 clinical trials for treating patients with deficient autosomal recessive congenital ichthyosis; KB104 for treating netherton syndrome; KB407 that is in Phase 1 clinical trials for treating cystic fibrosis; KB707 that is in Phase 1 clinical trials for the treatment of anti-PD-1 relapsed/refractory; KB408, which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study with ophthalmic B-VEC for treating for ocular complications of deb. Krystal Biotech, Inc. was founded in 2016 and is headquartered in Pittsburgh, Pennsylvania.

Replimune Group, Inc., a clinical-stage biotechnology company, focuses on the development of oncolytic immunotherapies to treat cancer. The company's proprietary tumor-directed oncolytic immunotherapy product candidates are designed and intended to activate the immune system against cancer. Its lead product candidate is RP1, a selectively replicating version of HSV-1 that expresses GALV-GP R(-) and human GM-CSF, which is in Phase I/II clinical trials for a range of solid tumors; and that has completed Phase II clinical trials for treating cutaneous squamous cell carcinoma. The company is also developing RP2, which is in Phase I clinical trials to express an anti-CTLA-4 antibody-like protein to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 that is in Phase I clinical trial to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapies that deliver functional genes to cells with genetic defects in the United States. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's products in pipeline includes ABBV-RGX-314 for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases; and RGX-202, which is in Phase I/II clinical trial for the treatment of Duchenne muscular dystrophy. It also develops RGX-121 for the treatment of mucopolysaccharidosis type II that is in Phase III clinical trial; RGX-111 for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late infantile neuronal ceroid lipofuscinosis type II; and RGX-381 to treat the ocular manifestations of CLN2 disease. In addition, the company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. Further, it has a collaboration and license agreement with AbbVie Global Enterprises Ltd. to develop ABBV-RGX-314 outside the United States. REGENXBIO Inc. was incorporated in 2008 and is headquartered in Rockville, Maryland.