


44 Thriving Biotech Startups (2021)




Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord




EMA Orphan Drug Designation for Forge Biologics’ gene therapy for Krabbe disease


FDA Fast Track, ODD, and RPDD Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease


FDA Clearance of Investigational New Drug for Patients with Krabbe Disease


United Leukodystrophy Foundation


RESKUE Trial for Patients with Krabbe Disease

