FDA– tag –
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CRISPR Gene Editing Technology Drives Clinical Adoption: FDA’s First Approval, Casgevy, for Sickle Cell Disease and Beta Thalassemia Accelerates Clinical Translation
Britannica Global Overview CRISPR, a powerful gene-editing tool with high sequence specificity, precisely removes or inserts DNA at specific genomic locations, offering superior efficiency, reliability, and cost-effectiveness over tradit... -
Hopstem’s iPSC-Derived Neural Progenitor Cell hNPC01 Receives FDA IND Clearance and Fast Track Designation for Stroke and Traumatic Brain Injury
AllSci USA Overview Hopstem Biotechnology announced that its allogeneic iPSC-derived forebrain neural progenitor cell therapy, hNPC01, received both IND clearance and Fast Track designation from the U.S. FDA for treating chronic motor de... -
FDA Flexibilizes Cell and Gene Therapy CMC Regulations, Eliminating ‘Rule of Three’ to Accelerate Development
BioProcess International USA Overview The U.S. FDA has announced a flexible Chemistry, Manufacturing, and Controls (CMC) guidance for Cell and Gene Therapy (CGT) products, effectively ending the traditional 'Rule of Three' validation req... -
Allogene’s Off-the-Shelf CAR T Cema-Cel Secures FDA RMAT and Fast Track for High-Risk LBCL First-Line Consolidation
OncLive USA Overview Allogene Therapeutics' off-the-shelf anti-CD19 CAR T-cell therapy, cemacabtagene ansegedleucel (cema-cel), received FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for first-line consoli... -
FDA Regenerative Medicine Approvals Accelerate: Record 8 Approvals in 2024, RMAT Designations Signal Continued Momentum
Pharmaceutical Technology USA Overview U.S. FDA approvals for regenerative medicine products have accelerated significantly, reaching a record eight approvals in 2024. The surge in Regenerative Medicine Advanced Therapy (RMAT) designatio... -
Allogene’s Off-the-Shelf CAR T, Cema-Cel, Earns FDA RMAT and Fast Track for High-Risk LBCL First-Line Consolidation
Allogene Therapeutics, Inc. USA Overview Allogene Therapeutics has secured FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for its investigational allogeneic CAR T-cell therapy, cemacabtagene ansegedleucel (... -
FDA Clarifies Regenerative Medicine Regulations: Strategic Use of RMAT Designation and HCT/P Classification to Accelerate Development
Global MedTech Expert USA Overview The U.S. FDA has detailed its Regenerative Medicine Advanced Therapy (RMAT) designation, offering a fast-track development framework for cell and gene therapies targeting serious or life-threatening dis... -
FDA Clears Prime Medicine’s In Vivo Prime Editor for Wilson Disease, Setting Stage for 2027 Data
BioSpace USA Overview Prime Medicine has secured FDA Investigational New Drug (IND) clearance for PM577a, an in vivo Prime Editor designed to correct the H1069Q mutation in Wilson disease. This approval initiates a global Phase 1/2 clini... -
AI and Digital Twin Challenges in GMP Manufacturing: Regulators Demand Audit Trails for Continuously Learning Models
BioPharma APAC Singapore Overview AI and digital twin technologies promise breakthroughs in GMP biomanufacturing, enabling predictive maintenance, real-time release, and high yields. However, regulatory challenges concerning the validati... -
FDA Approves Orca Bio’s Precision-Engineered Cell Therapy Tregzi (Orca-T) for Allogeneic Transplant in Adults with Hematological Malignancies
CliniExpert USA Overview The U.S. FDA has approved Orca Bio’s precision-engineered cell therapy, Tregzi™ (Orca-T®), for allogeneic transplant in adult patients with hematological malignancies. Tregzi is a personalized therapy manufacture...
