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.
Seres Therapeutics
NASDAQ:MCRBSeres Therapeutics, Inc., a microbiome therapeutics company, develop microbiome therapeutics to treat the modulation of the colonic microbiome. It develops a novel class of biological drugs that are designed to treat by modulating the microbiome to restore health by repairing the function of a disrupted microbiome to a non-disease state. The company's lead product candidate is VOWST, an oral microbiome therapeutic that has completed Phase III clinical trial for the treatment of recurrent Clostridioides difficile infection. Its product pipeline also includes SER-155, an investigational oral fermented microbiome therapeutic which is in Phase 1b clinical trials for the treatment of gastrointestinal infections, bacteremia, and graft versus host disease in immunocompromised patients including patients receiving allogeneic hematopoietic stem cell transplantation. In addition, the company engages in the development of SER-287 which is in Phase 2b and SER-301 that is in Phase 1b to treat ulcerative colitis. Further, it has license Agreement with NHSc Rx License GmbH for the therapeutic products based on the microbiome technology, which includes VOWST product candidate, which is developed for the treatment of CDI and recurrent CDI; and collaboration license agreement with Société des Produits Nestlé S.A. (Nestlé) for the development and commercialization of certain product candidates for the treatment and management of CDI and inflammatory bowel disease including UC and Crohn's disease. The company was formerly known as Seres Health, Inc. and changed its name to Seres Therapeutics, Inc. in May 2015. Seres Therapeutics, Inc. was incorporated in 2010 and is headquartered in Cambridge, Massachusetts.
PMV Pharmaceuticals
NASDAQ:PMVPPMV Pharmaceuticals, Inc., a precision oncology company, engages in the discovery and development of small molecule and tumor-agnostic therapies for p53 mutations in cancer. It's lead product candidate is PC14586, a small molecule that corrects mutant p53 protein containing the Y220C mutation and restores wild-type p53 function. The company was formerly known as PJ Pharmaceuticals, Inc. and changed its name to PMV Pharmaceuticals, Inc. in July 2013. PMV Pharmaceuticals, Inc. was incorporated in 2013 and is based in Princeton, New Jersey.
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.
Revance Therapeutics
NASDAQ:RVNCRevance Therapeutics, Inc., a biotechnology company, engages in the development, manufacture, and commercialization of neuromodulators for various aesthetic and therapeutic indications in the United States and internationally. The company's lead drug candidate is DAXXIFY (DaxibotulinumtoxinA-lanm) for injection for the treatment of glabellar lines and cervical dystonia; has completed phase II clinical trials to treat upper facial lines, moderate or severe dynamic forehead lines, and moderate or severe lateral canthal lines; and has completed Phase II clinical trials for the treatment of adult upper limb spasticity and plantar fasciitis. It is also developing OnabotulinumtoxinA, a biosimilar to BOTOX that is in preclinical stage. The company also offers Resilient Hyaluronic Acid (RHA) dermal filler for the correction of moderate to severe dynamic facial wrinkles; and RHA Redensity, a dermal filler for the treatment of moderate to severe dynamic perioral rhytids. It has a collaboration and license agreement with Viatris Inc. to develop, manufacture, and commercialize onabotulinumtoxinA. The company was formerly known as Essentia Biosystems, Inc. and changed its name to Revance Therapeutics, Inc. in April 2005. Revance Therapeutics, Inc. was incorporated in 1999 and is headquartered in Nashville, Tennessee.