Recent News: ARPA-H Awards Up to $160M for Custom Genetic Medicines on July 13, 2026 ARPA-H Clinical Trials curative medicines Gene Modification genetic medicines GIVE program Hereditary Diseases in vivo Infrequent Personalized Precision - temporal Programs - Publication Format Thrive +
FDA Approvals Outrun Staff Cuts: 23 Novel Drugs Mark Best First Half in Three Years on July 06, 2026 Communicable Diseases Drug Approval First Half Hereditary Diseases Infrequent Metabolic Diseases Neoplasms New Staff United States Food and Drug Administration +
BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test on May 19, 2026 BioMarin BMN Clinical Benefit Deficiency ENPP1 gene growth Z-score Hereditary Diseases Infrequent inorganic pyrophosphate Plasma prepulse inhibition activity Rare Diseases RGI-C scores Rickets Severities +
FDA Launches New Submission Program for Rare Disease Gene Therapies via CDER and CBER on September 04, 2025 Accelerated Approved CBER CDER gene therapy Hereditary Diseases Infrequent RDEP regulatory guidance submission program United States Food and Drug Administration +
BioMarin's R&D Chief Warns of Innovation Slowdown Amid NIH Funding Cuts on February 22, 2025 BioMarin biomedical innovation Drug Development Hereditary Diseases NIH Mouse research partnerships +