Biologics License Application– tag –
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Prime Medicine’s Autologous Prime Editing Hematopoietic Stem Cell Therapy PM359 for p47phox-Deficient Chronic Granulomatous Disease Receives FDA RMAT Designation
BiopharmaWatch (Prime Medicine Press Release) USA Overview Prime Medicine announced its investigational autologous prime editing hematopoietic stem cell therapy, PM359, for the treatment of p47phox-deficient Chronic Granulomatous Disease... -
Intellia Therapeutics’ In Vivo CRISPR Therapy Lonvo-z Achieves 87% Attack Rate Reduction in HAE Phase 3 Trial, Initiates Rolling BLA Filing with FDA
Intellectia.AI USA Overview Intellia Therapeutics' in vivo CRISPR therapy, lonvo-z, demonstrated an 87% reduction in hereditary angioedema (HAE) attack frequency in its Phase 3 HAELO trial. Following these breakthrough results, the compa... -
Cell Therapy Weekly: uniQure Plans BLA Submission for Huntington’s Gene Therapy AMT-130; Ernexa Prepares IND for iMSC, Autolus Reports Early Phase I Data for SLE CAR-T
Cell & Gene Therapy Insights USA Overview uniQure plans to submit a Biologics License Application (BLA) for AMT-130, a gene therapy for Huntington's disease, in Q3 2026, based on three-year Phase I/II data. Ernexa Therapeutics has comple... -
ProBio and Curocell Achieve BLA Approval for Next-Gen CD19-Targeted CAR-T Therapy, Initiating Commercial Production in South Korea
ProBio CDMO South Korea Overview ProBio CDMO and Curocell have secured Biologics License Application (BLA) approval for anbalcabtagene autoleucel (Anbal-cel; CRC01), their next-generation CD19-targeted CAR-T cell therapy for relapsed or ... -
FDA Finalizes Guidance on CMC Flexibility for Cell and Gene Therapies, Expediting Access to Advanced Treatments
Regulatory Focus USA Overview The U.S. FDA has issued final guidance outlining flexible Chemistry, Manufacturing, and Controls (CMC) requirements for cell and gene therapy (CGT) products, recognizing their inherent complexities compared ...
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