Publisher name Regeneron Pharmaceuticals The U.S. Food and Drug Administration has granted accelerated approval to an adeno-associated virus–based gene therapy for patients with severe-to-profound hearing loss caused by OTOF gene variants. Clinical data showed improvements in hearing thresholds in a majority of participants, with some achieving levels consistent with functional or near-normal hearing, while continued approval remains contingent on confirmatory trial outcomes.

Publisher name Cytotherapy (International Society for Cell & Gene Therapy) The article discusses ongoing efforts to improve standardization and comparability across cell therapy manufacturing processes to support consistent product quality and regulatory evaluation. It highlights the importance of harmonized methodologies, reference materials, and data frameworks to enable reproducibility and facilitate broader clinical adoption of advanced therapies. 👉 Read the full article

Publisher name Cytotherapy (International Society for Cell & Gene Therapy) The article examines persistent translational challenges in advancing cell therapies from research settings into routine clinical use, including variability in starting materials, manufacturing consistency, and quality control. It emphasizes the need for standardized frameworks and fit-for-purpose development strategies to support reproducibility, regulatory confidence, and long-term clinical integrati

Published in Cytotherapy (Review Article) Asia’s CGT sector continues to expand with fast-track pathways in Japan, Korea, China and Singapore while India and Thailand advance through flexible access models and domestic innovation. Approvals for CAR-T, stem cell and gene therapies are rising and pricing differences highlight how local manufacturing and regulatory maturity shape affordability and access. Regional collaboration and shared infrastructure are identified as key lev

Published on Endpoints News The FDA has unveiled a new “plausible mechanism pathway” to speed access to personalized gene editing therapies, inspired by the Baby KJ case. The approach aims to support rare disease treatment by allowing alternative trial designs while maintaining regulatory oversight. 👉 Read the full article: FDA unveils new path to speed personalized therapies, inspired by Baby KJ Disclaimer Images used are for illustrative purposes only and do not depict act

Published on Fierce Biotech A patient in Intellia’s phase 3 trial of the CRISPR therapy nex-z has died following severe liver toxicity, prompting the FDA to keep both studies on hold. The company is investigating the liver signal and developing risk mitigation steps while monitoring all previously treated patients. 👉 Read the full article: Patient dies after receiving Intellia's CRISPR therapy Disclaimer Images used are for illustrative purposes only and do not depict actual

Published on Holland & Knight The FDA has released new draft guidance outlining expedited pathways for regenerative medicine therapies....

Published on RAPS The US FDA has released updated guidance documents covering cell and gene therapies and regenerative medicine. These...

Published on LinkedIn (via Spencer Knight) The FDA has approved Inliruqo™ (imlunestrant) from Eli Lilly, the first oral estrogen receptor...

Published on Cytotherapy A new perspective warns that restructuring advanced therapy oversight within the European Medicines Agency could...

Published via FDA & EU regulatory updates The FDA has approved TREMFYA® as the first and only IL-23 inhibitor with both subcutaneous and...

Published on: ExCellThera / Cordex Biologics The European Commission has conditionally authorized Zemcelpro (UM171 Cell Therapy), the...

Published on: LinkedIn (Spencer Knight) The European Commission has approved TEVIMBRA (tislelizumab) from BeOne Medicines, the first PD-1...

Published on: LinkedIn (Spencer Knight) The U.S. FDA has approved DAWNZERA (donidalorsen) from Ionis Pharmaceuticals, the first...

Published on: LinkedIn (Spencer Knight) In just five days, two breakthrough approvals have reshaped treatment for metabolic...

Published on: Autolus Therapeutics The European Commission has granted marketing authorization for AUCATZYL (obecabtagene autoleucel) to...

Published on: Precigen Investors The U.S. FDA has granted full approval to PAPZIMEOS (zopapogene imadenovec-drba), the first therapy for...

Published on ISCT Telegraft Blog by Francheska Juliano At the ISCT 2024 Annual Meeting in Vancouver, global experts discussed how access...

Published on Drug Discovery News Recent FDA rejections of three advanced therapies, Capricor’s CAP‑1002, Ultragenyx’s DTX401, and Rocket...

Published on LinkedIn via Spencer Knight The FDA has updated CAR-T therapy regulations for BMS’ Abecma and Breyanzi, removing REMS...