Generation Bio
NASDAQ:GBIOGeneration Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.
Gossamer Bio
NASDAQ:GOSSGossamer Bio, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing seralutinib for the treatment of pulmonary arterial hypertension (PAH) in the United States. The company is developing GB002, an inhaled, small molecule, platelet-derived growth factor receptor, or PDGFR, colony-stimulatin factor 1 receptor and c-KIT inhibitor, which is in Phase 3 clinical trial for the treatment of PAH. It has license agreements with Pulmokine, Inc. to develop and commercialize GB002 and related backup compounds. The company was formerly known as FSG, Bio, Inc. and changed its name to Gossamer Bio, Inc. in 2017. Gossamer Bio, Inc. was incorporated in 2015 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.
Travere Therapeutics
NASDAQ:RTRXTravere Therapeutics, Inc. is a biopharmaceutical company. It engages in the identification, development, commercialization, and distribution of therapies to people living with rare diseases. Its products include Chenodal, Cholbam, and Thiola. The company was founded by Martin Shkreli on February 8, 2008 and is headquartered in San Diego, CA.
Stoke Therapeutics
NASDAQ:STOKStoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.