🔬 Rare Diseases Update: CRISPR Therapy, SCA, ENPP1 Deficiency, Gene Therapy & More
This week’s roundup features transformative gene therapies, pivotal acquisitions, fresh orphan drug designations, and key regulatory shifts—each reshaping the path forward for patients with ultra-rare conditions. From the first infant treated with personalized CRISPR to crucial manufacturing alliances in gene therapy, the rare disease ecosystem is accelerating.
🚨 Don’t miss these major developments:
💰 BioMarin acquires Inozyme for $270M, adding late-stage enzyme therapy INZ-701 for ENPP1 Deficiency
🧬 First-ever infant receives personalized CRISPR therapy for a rare urea cycle disorder—developed in just 6 months
🏛️ EU Parliament launches a broad health agenda tackling rare diseases, AMR, and cross-border trials
⌛ FDA extends PDUFA date for troriluzole, a potential first-in-class treatment for Spinocerebellar Ataxia
🧪 Cure Rare Disease’s CRD-002 receives Orphan Drug Designation for Spinocerebellar Ataxia Type 3
🏭 Viralgen joins ClinDevNet to boost AAV gene therapy manufacturing for rare conditions
👶 Only 5 percent of pediatric rare diseases projected to have approved treatments by 2033—urgent innovation needed
❓ FDA’s conditional approval proposal sparks debate over safety, reimbursement, and legislative feasibility
🧠 Govorestat demonstrates long-term clinical benefit in CMT-SORD, with NDA submission planned for 2025
🌍 EMA grants Orphan Drug Designation to QRX003 for Netherton Syndrome, with 10-year exclusivity if approved
📢 Stay Ahead in Rare Disease Research!
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