C4 Therapeutics
NASDAQ:CCCCC4 Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops novel therapeutic candidates to degrade disease-causing proteins for the treatment of cancer, neurodegenerative conditions, and other diseases. Its lead product candidate is CFT7455, an orally bioavailable MonoDAC degrader of protein that is in Phase 1/2 trial targeting IKZF1 and IKZF3 for multiple myeloma and non-Hodgkin lymphomas, including peripheral T-cell lymphoma and mantle cell lymphoma, currently under Phase 1/2 clinical trials. The company is also developing CFT1946, an orally bioavailable BiDAC degrader targeting V600X mutant BRAF to treat melanoma, non-small cell lung cancer (NSCLC), colorectal cancer, and other solid malignancies, currently under Phase 1/2 clinical trials; and CFT8919, an orally bioavailable, allosteric, and mutant-selective BiDAC degrader of epidermal growth factor receptor, or EGFR, with an L858R mutation in NSCLC. It has strategic collaborations with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; Biogen MA, Inc.; Betta Pharmaceuticals, Co., Ltd.; and Merck Sharp & Dohme, LLC, as well as Calico Life Sciences LLC. The company was incorporated in 2015 and is headquartered in Watertown, Massachusetts.
Inhibrx
NASDAQ:INBXInhibrx, Inc., a clinical-stage biopharmaceutical company, develops a pipeline of novel biologic therapeutic candidates. The company's therapeutic candidates include INBRX-109, a tetravalent therapeutic candidate targeting death receptor 5, which is in Phase 2 clinical trials to treat cancers, such as chondrosarcoma, mesothelioma, colorectal cancer, ewing sarcoma, and pancreatic adenocarcinoma; and INBRX-101, an alpha-1 antitrypsin (AAT)-Fc fusion protein therapeutic candidate, which is in Phase 1 clinical trials for use in the treatment of patients with AAT deficiency. It also develops INBRX-106, a hexavalent agonist of OX40 for a range of oncology indications. The company has license and collaboration agreements with 2seventy bio, Inc. and Bristol-Myers Squibb Company. Inhibrx, Inc. was incorporated in 2009 and is headquartered in La Jolla, California.
Krystal Biotech
NASDAQ:KRYSKrystal 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.
REGENXBIO
NASDAQ:RGNXREGENXBIO 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.
Sorrento Therapeutics
NASDAQ:SRNESorrento Therapeutics, Inc. engages in the research, development and manufacture of biopharmaceutical products. It focuses on therapies to treat specific stages in the evolution of cancer, from elimination, to equilibrium and escape which include biosimilars, immuno-oncology antibodies, cellular therapy, cell internalizing antibodies, and antibody drug conjugates. It operates through the Sorrento Therapeutics and Scilex segments. The company was founded by Henry H. Ji in 1989 and is headquartered in San Diego, CA.