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FDA Eases CMC Rules for Cell and Gene Therapies to Speed Development

The move aligns oversight with the realities of small‑batch, personalized products to address serious unmet needs.

BioMarin to Acquire Amicus for $4.8 Billion in Cash

HHS Adds Duchenne Muscular Dystrophy and MLD to Newborn Screening Recommendations

Kyverna CAR-T Delivers Positive Phase 2 Topline in Stiff Person Syndrome as Shares Jump

China Approves Sanofi’s Qfitlia for Hemophilia A Prophylaxis and Cablivi for aTTP

Capricor’s HOPE-3 Trial Reports Cardiac and Muscle Gains in Duchenne Muscular Dystrophy

AI Model popEVE Delivers Proteome-Wide Severity Scores for Rare-Disease Diagnosis

FDA Approves Koselugo for Adults With NF1-Related Plexiform Neurofibromas

Protara Reports Strong Interim Phase 2 Results for TARA-002 in Pediatric Lymphatic Malformations

Agios’ Sickle Cell Pill Lifts Hemoglobin but Misses Pain-Crisis Goal in Phase 3

Moderna Scraps CMV Vaccine Program After Phase 3 Failure

Sanofi’s Efdoralprin Alfa Shows Superior Biomarker Response to Plasma Therapy in Phase 2 AATD Trial

FDA Signals Openness to Accelerated Path for Lexeo’s Friedreich’s Ataxia Cardiac Gene Therapy

Utah’s First Pediatric Paired Liver Exchange Saves Two Children

FDA Opens Door to Reconsider Capricor’s Duchenne Cell Therapy Based on HOPE-3 Data

Regeneron’s Garetosmab Slashes New Bone Lesions in FOP Phase 3, U.S. Filing Planned This Year

Nature Study Flags ABHD18 Inhibition as a Preclinical Path to Treating Barth Syndrome

FDA Approves Ionis’ Dawnzera to Prevent Hereditary Angioedema Attacks

European Commission Approves Ogsiveo as First Therapy for Desmoid Tumors

Prime Editing Reverses Rare Childhood Brain Disorder by Correcting Multiple Mutations in Mice