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.
Kymera Therapeutics
NASDAQ:KYMRKymera Therapeutics, Inc., a biopharmaceutical company, focuses on discovering and developing novel small molecule therapeutics that selectively degrade disease-causing proteins by harnessing the body's own natural protein degradation system. It engages in developing IRAK4 program, which is in Phase II clinical trial for the treatment of immunology-inflammation diseases, including hidradenitis suppurativa, atopic dermatitis; STAT3 program for the treatment of hematologic malignancies and solid tumors, as well as autoimmune diseases and fibrosis; and MDM2 program to treat hematological malignancies and solid tumors. The company develops STAT6, a Type 2 inflammation in allergic diseases; and TYK2, a treatment for inflammatory bowel disease, psoriasis, psoriatic arthritis, and lupus. The company was incorporated in 2015 and is headquartered in Watertown, Massachusetts.
NantKwest
NASDAQ:NKNantKwest, Inc., a clinical-stage immunotherapy company, develops immunotherapeutic treatments for cancer and viral infectious diseases in the United States. The company develops activated natural killer (aNK) cell, a natural killer cell-line and genetically modified derivative capable of killing cancer and virally infected cells; and Nant cancer vaccine, a personalized therapy that utilizes its off-the-shelf natural killer cells. It is also developing high-affinity natural killer (haNK) product candidates primarily for the treatment of various cancers; and tank and t-haNK product candidates to treat solid tumors. NantKwest, Inc. has a co-development agreement with Altor BioScience, LLC to collaborate on the development of therapeutic applications combining the company's proprietary natural killer cells with Altor's N-801 and/or N-803 products; collaboration with Be The Match BioTherapies for the development of cell therapy to prevent COVID-19 deaths; and strategic alliance with Immunitybio. It also has a collaboration with CBR Systems, Inc. to develop a COVID-19 treatment leveraging newborn stem cells. The company was formerly known as Conkwest, Inc. and changed its name to NantKwest, Inc. in July 2015. NantKwest, Inc. was founded in 2002 and is headquartered in San Diego, California.
Rubius Therapeutics
NASDAQ:RUBYRubius Therapeutics, Inc., a biopharmaceutical company, focuses on the development of red cell therapeutics for the treatment of cancer and autoimmune diseases. The company was formerly known as VL26, Inc and as changed its name to Rubius Therapeutics, Inc. in 2015. Rubius Therapeutics, Inc. was incorporated in 2013 and is based in Foxborough, Massachusetts.
Sangamo Therapeutics
NASDAQ:SGMOSangamo Therapeutics, Inc., a clinical-stage genomic medicine company, focuses on translating science into medicines that transform the lives of patients and families afflicted with serious diseases in the United States. The company's clinical-stage product candidates are ST-920, a gene therapy product candidate, which is in Phase 1/2 clinical study for the treatment of Fabry disease; TX200, a chimeric antigen receptor engineered regulatory T cell (CAR-Treg) therapy product candidate that is in Phase 1/2 clinical study for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation; SB-525, a gene therapy product candidate, which is in Phase 3 clinical trial for the treatment of moderately severe to severe hemophilia A; BIVV003, a zinc finger nuclease gene-edited cell therapy product candidate that is in Phase 1/2 PRECIZN-1 clinical study for the treatment of sickle cell disease. Its preclinical development products focus on CAR-Treg cell therapies for autoimmune disorders and genome engineering for neurological diseases. Sangamo Therapeutics, Inc. has collaborative and strategic partnerships with Biogen MA, Inc.; Kite Pharma, Inc.; Pfizer Inc.; Sanofi S.A.; Novartis Institutes for BioMedical Research, Inc.; Shire International GmbH; Dow AgroSciences LLC; Sigma-Aldrich Corporation; Genentech, Inc.; Open Monoclonal Technology, Inc.; F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; and California Institute for Regenerative Medicine. The company was formerly known as Sangamo BioSciences, Inc. and changed its name to Sangamo Therapeutics, Inc. in January 2017. Sangamo Therapeutics, Inc. was incorporated in 1995 and is headquartered in Brisbane, California.