FDA– tag –
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NurExone Accelerates U.S. Market Entry with GMP Exosome Manufacturing Partnership with Made Scientific, Targeting First Batch by H1 2027
BioPharma BoardRoom USA Overview Exo-Top Inc., a U.S. subsidiary of NurExone Biologic Inc., has signed an MOU with Made Scientific, Inc. for a GMP exosome manufacturing partnership in the U.S. This strategic alliance aims to accelerate N... -
FDA Finalizes Q&A Guidance on Cell and Gene Therapy Product Development, Clarifying Regulatory Review, CMC, and Safety Monitoring
RAPS (Regulatory Affairs Professionals Society) USA Overview The U.S. FDA has released final FAQ guidance on developing cell and gene therapy (CGT) products, providing practical answers to common regulatory questions spanning regulatory ... -
Xellsmart Secures FDA Fast Track Designation for Parkinson’s iPSC-Derived Cell Therapy, Accelerating CNS Pipeline
Longevity.Technology UK Overview Xellsmart announced its allogeneic, off-the-shelf iPSC-derived cell therapy for Parkinson's disease has received FDA Fast Track designation, expected to accelerate its development program. Specializing in... -
Cellistic CTO Highlights Japanese Conditional Approvals for Cardiac and Parkinson’s iPSC Products, Outlining Path to Commercial-Scale iPSC Cell Therapy Manufacturing
The Medicine Maker UK Overview In an interview with The Medicine Maker, Stefan Braam, CTO of Cellistic, stated that the industry's main focus is the ability to consistently provide iPSC-based cell therapies at commercial scale for large ... -
Ultragenyx’s Genglycos Secures FDA Approval as First Gene Therapy for GSDIa – Bolstering In-House Manufacturing and Long-Term Post-Marketing Monitoring
Pharmaceutical Technology USA Overview Ultragenyx Pharmaceutical has received FDA accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), the first gene therapy targeting the root cause of the ultra-rare metabolic disorder,... -
Ultragenyx’s Genglycos Receives FDA Accelerated Approval as First Gene Therapy for Glycogen Storage Disease Type Ia, Requiring 10-Year Post-Marketing Monitoring
Pharmaceutical Technology USA Overview Ultragenyx Pharmaceutical announced that Genglycos (pariglasgene brecaparvovec-opnr), the first gene therapy for Glycogen Storage Disease Type Ia (GSDIa), has received FDA accelerated approval. This... -
Allogene Therapeutics’ TALEN-Edited Lymphoma Cell Therapy cema-cel Receives FDA RMAT and Fast Track Designations, Achieves 58.3% MRD Negativity in Phase 2 Trial
PRISM MarketView USA Overview Allogene Therapeutics' TALEN-edited allogeneic cell therapy, cema-cel, has received both Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA, accelerating its regulatory pa... -
FDA Lifts Clinical Hold on Intellia Therapeutics’ Phase 3 Trial for nex-z in Transthyretin Amyloidosis
Intellia Therapeutics (via LinkedIn) USA Overview Intellia Therapeutics announced the FDA has lifted the clinical hold on its Phase 3 MAGNITUDE-2 clinical trial for nexiguran ziclumeran (nex-z), a therapeutic candidate for transthyretin ... -
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... -
Vertex Pharmaceuticals’ CASGEVY Receives FDA Label Expansion for Over 5,500 US Children with Sickle Cell and Beta-Thalassemia, Driving Soaring Sales
ScanX USA Overview Vertex Pharmaceuticals' gene therapy, CASGEVY (exagamglogene autotemcel), received FDA label expansion for patients aged 2 and older with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). This...
