Biologics License Application– tag –
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FDA Expands Regulatory Flexibility for Cell and Gene Therapies, Streamlining CMC Requirements to Accelerate Early Clinical Development
Medicinova, Inc. (Facebook) USA Overview The US FDA has announced an expansion of regulatory flexibility for Cell and Gene Therapy (CGT) products, particularly adapting Chemistry, Manufacturing, and Controls (CMC) requirements across the... -
KOLON Biotech Expands Comprehensive CDMO Services in Korea, Specializing in Cell, Gene, and Exosome Therapies
MTEC South Korea Overview KOLON Biotech in South Korea is bolstering its capabilities as a specialized Contract Development and Manufacturing Organization (CDMO) for advanced cell, gene, and exosome therapies. The company offers end-to-e... -
Intellia Therapeutics’ Gene-Editing Therapy lonvo-z Achieves 87% Reduction in HAE Attacks and 62% Attack-Free Rate in Phase 3 Trial
The Motley Fool USA Overview Intellia Therapeutics announced its in vivo gene-editing therapy, lonvoguran ziclumeran (lonvo-z), successfully met all primary and secondary endpoints in the Phase 3 HAELO trial for hereditary angioedema (HA... -
Intellia Therapeutics Advances CRISPR Gene Editing: Lonvo-z Achieves Phase 3 Success for HAE, Targeting Mid-2027 U.S. Launch; Nex-z Phase 3 Enrollment Resumes for ATTR
Intellia Therapeutics, Inc. (Press Release) USA Overview Intellia Therapeutics reported positive Phase 3 results for lonvo-z in hereditary angioedema (HAE), with a Biologics License Application (BLA) submission to the FDA planned for H2 ... -
FDA Finalizes Flexible CMC Framework for Cell and Gene Therapy Approvals, Accelerating Personalized Medicine Regulatory Pathways
BioProcess International USA Overview The U.S. FDA finalized a flexible Chemistry, Manufacturing, and Controls (CMC) framework for cell and gene therapy (CGT) product approvals, releasing a guidance document on May 5, 2026. This framewor... -
Vertex Pharmaceuticals Secures FDA IND Approval for iPSC-Derived Type 1 Diabetes Therapies, Boosts 2026 Revenue Forecast
Vertex Pharmaceuticals USA Overview Vertex Pharmaceuticals announced robust Q2 2026 financial results and significantly raised its full-year revenue guidance, propelled by key pipeline advancements. The company secured FDA Investigationa... -
Intellia Therapeutics Reports Positive Phase 3 HAELO Data for lonvo-z in HAE and Resumes Nex-z ATTR Phase 3 Enrollment
Intellia Therapeutics USA Overview Intellia Therapeutics announced positive Phase 3 HAELO clinical data for lonvo-z in hereditary angioedema (HAE), published in the New England Journal of Medicine. The company anticipates FDA Biologics L... -
FDA Introduces Flexible CMC Guidance for Cell and Gene Therapy Products, Ending ‘Rule of Three’ for Commercial Validation Batches
BioProcess International USA Overview The FDA published and immediately implemented Level 2 guidance, 'CMC Flexibilities for Developing Human Cellular and Gene Therapy Products for a Biologics License Application' on May 5, 2026. This la... -
FDA Details Regenerative Medicine Advanced Therapy (RMAT) Designation Framework, Offering Fast Track and Breakthrough Therapy Benefits
Global MedTech Expert USA Overview Global MedTech Expert details the FDA's regulatory framework for 351/361 HCT/P classification and Regenerative Medicine Advanced Therapy (RMAT) designation. RMAT offers significant benefits, including F... -
Catalent and Nanoscope Expand Supply Partnership for Commercialization of Optogenetic Gene Therapy MCO-010 for Retinal Degenerative Diseases
LucidQuest Ventures USA Overview Catalent and Nanoscope Therapeutics have expanded their partnership to support the late-stage development and commercial supply of MCO-010, an optogenetic gene therapy candidate for retinal degenerative d...
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