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The funding will be used for a novel Adeno-Associated Virus (AAV) gene therapy, GA-002, aimed at curing GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM).
Lead Product(s): GA-002
Therapeutic Area: Rare Diseases and Disorders Product Name: GA-002
Highest Development Status: IND Enabling Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Solve GNE
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement August 14, 2023