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Design 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.

Galectin Therapeutics Inc., a clinical stage biopharmaceutical company, engages in the research and development of therapies for fibrotic, cancer, and other diseases. Its lead product candidate is belapectin (GR-MD-02) galectin-3 inhibitor, a galactoarabino-rhamnogalacturonan polysaccharide polymer that is in Phase III clinical trial for the treatment of liver fibrosis associated with fatty liver disease and non-alcoholic steatohepatitis cirrhosis, as well as for the treatment of cancer. The company, through its Galectin Sciences, LLC, which is a collaborative joint venture co-owned by SBH Sciences, Inc., to research and development of small organic molecule inhibitors of galectin-3 for oral administration. The company was formerly known as Pro-Pharmaceuticals, Inc. and changed its name to Galectin Therapeutics, Inc. in May 2011. Galectin Therapeutics Inc. was founded in 2000 and is based in Norcross, Georgia.

Merrimack Pharmaceuticals, Inc. operates as a biopharmaceutical company in the United States. The company was incorporated in 1993 and is headquartered in Cambridge, Massachusetts.

Prelude Therapeutics Incorporated, a clinical-stage biopharmaceutical company, focuses on the discovery and development of novel precision cancer medicines to underserved patients. It is developing PRT1419, a myeloid cell leukemia-1 inhibitor, which is in Phase 1 clinical trial for the treatment of selected relapsed/refractory myeloid or B-cell malignancies; PRT2527, a cyclin-dependent kinase, which is in Phase 1 clinical trial for the treatment of advanced solid tumors; PRT3645, a cyclin-dependent kinase 4/6 inhibitor, which is in Phase 1 clinical trial for the treatment of advanced and metastatic solid tumors; and PRT3879, a SMARCA2 selective protein degrader, which is in Phase 2/3 clinical trial for the treatment of advanced and metastatic solid tumors with loss of SMARCA4 due to truncating mutation and/or deletion. The company's CDK9 program is a regulator of cancer-promoting transcriptional programs, including MCL1, MYC and MYB. Prelude Therapeutics Incorporated was incorporated in 2016 and is based in Wilmington, Delaware.