Design Therapeutics
NASDAQ:DSGNDesign Therapeutics, Inc. a biopharmaceutical company, researches, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.
IDEAYA Biosciences
NASDAQ:IDYAIDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 1/2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung, bladder, gastric, and esophageal cancers; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; GSK101, a Pol Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other homologous recombination, and HRD mutations; and Werner Helicase inhibitors for tumors with high microsatellite instability. It offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Novartis International Pharmaceuticals Ltd., Amgen Inc., Gilead Sciences, Inc., Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.
Kinnate Biopharma
NASDAQ:KNTEKinnate Biopharma Inc., a clinical-stage oncology company, focuses on the discovery and development of small molecule kinase inhibitors to treat genomically defined cancers in the United States. The company develops KIN-2787, a rapidly accelerated fibrosarcoma inhibitor for the treatment of patients with lung cancer, melanoma, and other solid tumors; KIN-3248 small-molecule kinase inhibitors that target cancer-associated alterations in fibroblast growth factor receptors FGFR2 and FGFR3 genes; and small molecule research programs, including Cyclin-Dependent Kinase 12(CDK12) inhibitor in its KIN004 program. The company was incorporated in 2018 and is headquartered in San Francisco, California. Kinnate Biopharma Inc. is a former subsidiary of Fount Therapeutics, LLC.
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.
Zymeworks
NYSE:ZYMEZymeworks Inc., a clinical-stage biopharmaceutical company, discovers, develops, and commercializes biotherapeutics for the treatment of cancer. The company's lead product candidates include zanidatamab, a human epidermal growth factor receptor 2 (HER2) that is in Phase 1, Phase 2, and Phase 3 clinical trials, including certain ongoing pivotal clinical trials; and zanidatamab zovodotin, a HER2 -targeted antibody-drug conjugate that is in Phase 2 clinical trial for the treatment of advanced or metastatic HER2-expressing tumors. It also develops a pipeline of preclinical product candidates and discovery-stage programs in oncology, including immuno-oncology agents and other therapeutic areas. Zymeworks Inc. has strategic partnerships and collaborations with BeiGene, Ltd.; Celgene Corporation; Celgene Alpine Investment Co. LLC; GlaxoSmithKline Intellectual Property Development Limited; Daiichi Sankyo Co., Ltd.; Janssen Biotech, Inc.; LEO Pharma A/S; Iconic Therapeutics, Inc.; Merck Sharp & Dohme Research GmbH; and Atreca, Inc. Zymeworks Inc. was incorporated in 2003 and is based in Middletown, Delaware.