Avidity Biosciences
NASDAQ:RNAAvidity Biosciences, Inc., a biopharmaceutical company, engages in the delivery of RNA therapeutics. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat diseases previously untreatable with RNA therapeutics. The company's lead product candidate AOC 1001 for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease that is in phase 1/2 clinical trial. Its other products in pipeline include AOC 1044 for the treatment of duchenne muscular dystrophy, which is under phase 1/2 clinical development trial; and AOC 1020 to treat facioscapulohumeral muscular dystrophy that is in phase 1/2 clinical trial. The company is developing products for rare skeletal muscle and rare cardiac diseases. Avidity Biosciences, Inc. was incorporated in 2012 and is headquartered in San Diego, California.
Repare Therapeutics
NASDAQ:RPTXRepare Therapeutics Inc., a clinical-stage precision oncology company, engages in the discovery and development of therapeutics by using its synthetic lethality approach in Canada and the United States. It uses its SNIPRx, a proprietary, genome-wide, and CRISPR-enabled platform, to discover, validate, and build a pipeline of SL-based therapeutics that focuses on genomic instability, including DNA damage repair. The company's lead product candidate is Camonsertib (RP-3500), an oral small molecule inhibitor under Phase ½ development for the treatment of solid tumors with specific DNA damage repair-related genomic alterations. It is also developing Lunresertib (RP-6306), a PKMYT1 Inhibitor, which is under Phase 1 clinical trial for tumors with genetic alterations characterized by CCNE1 amplification; RP-1664, an oral PLK4 inhibitor, under Phase 1 clinical trial designed to harness the synthetic lethal relationship with TRIM37 amplification or overexpression in solid tumors; and RP-3467, a polymerase theta adenosinetriphosphatase (ATPase) inhibitor, a SL target associated with BRCA mutations and other genomic alterations. The company has license and collaboration agreement with Hoffmann-La Roche Inc. and F. Hoffmann-La Roche Ltd; Bristol-Myers Squibb Company; New York University; and Ono Pharmaceutical Co., as well as a clinical study and collaboration agreement with Debiopharm to explore the synthetic lethal combination of PKMYT1 and WEE1 inhibition in cancer. Repare Therapeutics Inc. was incorporated in 2016 and is headquartered in Montréal, Canada.
SIGA Technologies
NASDAQ:SIGASIGA Technologies, Inc., a commercial-stage pharmaceutical company, focuses on the health security related markets in the United States. Its lead product is TPOXX, an oral formulation antiviral drug for the treatment of human smallpox disease caused by variola virus. The company was incorporated in 1995 and is headquartered in New York, New York.
Tango Therapeutics
NASDAQ:TNGXTango Therapeutics, Inc., a biotechnology company, discovers and develops drugs for the treatment of cancer. Its lead program is TNG908, a synthetic lethal small molecule inhibitor of protein arginine methyltransferase 5 that is being developed as a treatment for cancers with methylthioadenosine phosphorylase deletions. The company develops TNG462, an oral small molecule methylthioadenosine-cooperative inhibitor for the treatment for cancers with methylthioadenosine phosphorylase deletions; TNG260, a co-repressor of repressor element-1 silencing transcription -selective inhibitor; TNG348, an ubiquitin-specific protease 1 inhibitor to treat patients with BRCA1 or BRCA2-mutant cancers; and Target 3 for STK11-mutant cancers. It has a strategic collaboration with Gilead Sciences, Inc. for the discovery, development, and commercialization of a pipeline of therapies for patients with cancer. Tango Therapeutics, Inc. was founded in 2017 and is headquartered in Boston, Massachusetts.