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Design Therapeutics Inc is a clinical-stage biopharmaceutical company engaged in the research and development of GeneTACTM molecules, which are a novel class of small-molecule gene-targeted chimera therapeutic candidates designed to be disease-modifying by addressing the underlying cause of diseases caused by inherited nucleotide repeat expansion mutations. The Company's product candidate is in Friedreich ataxia (FA), its second product candidate is in Fuchs endothelial corneal dystrophy (FECD), and it is also advancing its GeneTACTM program to address other serious nucleotide repeat-driven monogenic diseases.
Eledon Pharmaceuticals Inc is a clinical-stage biotechnology company. The firm is focused on transplantation and autoimmune diseases. It targets the CD40L pathway to develop potential treatments for patients living with an autoimmune disease, patients requiring an organ or cell-based transplant, and patients living with ALS. The company's compound in development is tegoprubart, an IgG1, an anti-CD40L antibody with high affinity for the CD40 Ligand, a well-validated biological target that has broad therapeutic potential.