AlloVir
NASDAQ:ALVRAllovir, Inc., a clinical-stage cell therapy company, engages in the research and development of allogeneic, off-the-shelf multi-virus specific T cell (VST) therapies to prevent and treat devastating viral-associated diseases. The company's lead product is posoleucel, an allogeneic, off-the-shelf VST therapy, to treat BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, human herpesvirus 6, and JC virus. Its preclinical and clinical development product candidates include ALVR106 for the respiratory syncytial virus, influenza, parainfluenza virus, and human metapneumovirus; ALVR109 to treat SARS-CoV-2 and COVID-19; ALVR107 for treating hepatitis B; and ALVR108. The company was formerly known as ViraCyte, Inc. and changed its name to Allovir, Inc. in May 2019. The company was incorporated in 2013 and is based in Waltham, Massachusetts.
BioCryst Pharmaceuticals
NASDAQ:BCRXBioCryst Pharmaceuticals, Inc., a biotechnology company, develops oral small-molecule and protein therapeutics to treat rare diseases. The company markets peramivir injection, an intravenous neuraminidase inhibitor for the treatment of acute uncomplicated influenza under the RAPIVAB, RAPIACTA, and PERAMIFLU names; and ORLADEYO, an oral serine protease inhibitor to treat hereditary angioedema. It is also developing BCX10013, an oral factor D inhibitor for complement-mediated diseases. The company has collaborations and in-license relationships with the Torii Pharmaceutical Co., Ltd.; Seqirus UK Limited; Shionogi & Co., Ltd.; Green Cross Corporation; National Institute of Allergy and Infectious Diseases; Biomedical Advanced Research and Development Authority; the U.S. Department of Health and Human Services; and The University of Alabama at Birmingham, as well as Albert Einstein College of Medicine of Yeshiva University and Industrial Research, Ltd. BioCryst Pharmaceuticals, Inc. was founded in 1986 and is headquartered in Durham, North Carolina.
bluebird bio
NASDAQ:BLUEbluebird bio, Inc., a biotechnology company, researches, develops, and commercializes gene therapies for severe genetic diseases. Its product candidates for severe genetic diseases include ZYNTEGLO (betibeglogene autotemcel) for the treatment of transfusion-dependent ß-thalassemia; lovotibeglogene autotemcel for the treatment of sickle cell disease (SCD); and SKYSONA (elivaldogene autotemcel) to treat cerebral adrenoleukodystrophy. The company's clinical development programs include HGB-205, HGB-206, and HGB-210 to evaluate the safety and efficacy of lovo-cel in the treatment of patients with SCD; and HGB-204, HGB-205, HGB-207, and HGB-212 to evaluate the safety and efficacy of beti-cel in the treatment of patients with ß-thalassemia. It has license agreement with Orchard Therapeutics Limited. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was incorporated in 1992 and is headquartered in Somerville, Massachusetts.
Scholar Rock
NASDAQ:SRRKScholar Rock Holding Corporation, a biopharmaceutical company, focuses on the discovery, development, and delivery of medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company develops Apitegromab, an inhibitor of the activation of myostatin that is in Phase 3 clinical trial for the treatment of spinal muscular atrophy; and SRK-181, which has completed Phase 1 clinical trials for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies. It is developing a pipeline of product candidates to deliver novel therapies to treat a range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, cancer, fibrosis, and iron-restricted anemia. Scholar Rock Holding Corporation was founded in 2012 and is headquartered in Cambridge, Massachusetts.
Vericel
NASDAQ:VCELVericel Corporation, a commercial-stage biopharmaceutical company, engages in the research, development, manufacture, and distribution of cellular therapies for sports medicine and severe burn care markets in North America. The company markets autologous cell therapy products comprising MACI, an autologous cultured chondrocytes on porcine collagen membrane for the repair of symptomatic, and single or multiple full-thickness cartilage defects of the knee; Epicel, a permanent skin replacement humanitarian use device for the treatment of adult and pediatric patients with deep-dermal or full-thickness burns; and NexoBrid, a biological orphan product for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns. The company was formerly known as Aastrom Biosciences, Inc. Vericel Corporation was incorporated in 1989 and is headquartered in Cambridge, Massachusetts.