Gene and Cell Therapy Network

At the #VLGeneCell 2025 the experts in the field will continue exploring the unique challenges related to CMC, analytical, process development, automation, manufacturing, facility design, aseptic processing, supply chain, and other, that developers of gene and cell therapies must resolve. #VLGeneCell 🔸4th Gene & Cell Therapies: CMC, CGT & Vector Manufacturing 🔸 📆4-5 June 2025 📍Munich, Germany 🔗https://lnkd.in/dHnVHZma Discover strategies for meeting CGTP safety and efficiency criteria, regulatory and GMP manufacturing standards, and optimizing vector production processes.  For speaking and sponsorship opportunities contact 📩 Milana Dreo #atmps #analytical #bioprocessing #biotherapeutics #cart #cellular #cellengineering #celltherapy #immunotherapy #genetherapy #gmp #gene #CQAs #gtmps #cell #QC #quality #laboratory #manufacturing #regulatory #safety #automation #facility #aseptic #asepticprocessing #vector #viralvectors #cgtp #cgt #techtransfer #cleanroom #AAV #adenovirus #cdmo #lentivectors #cgts #reagents #biopharma #cmo #cmdo #workshop #exhibition #conference

This is the best overview I've seen in a while. But one particular thing stands out for me: Often, we think of AAV and LNP as competing technologies. (And in many circumstances, they are) But I'm intrigued by applications that put them together. You end up getting the advantages of both: - Higher cargo capacity - Long-term transgene expression - Transient expression of a genome editing tool - Low dose + single administration - Better manufacturing costs Interested to see where this application goes in the future. Any thoughts on this? Drop them in the comments. Image source: https://lnkd.in/eauP8y-n

CAR-T manufacturing needs to improve. Otherwise, it'll never get to most patients. This paper outlines what needs to change. Problems Assessed - Complex and resource-intensive processes that cause many delays - Variability in processes, influenced by patient-specific factors - High costs throughout the process Strategies Evaluated: - Comparison of viral and non-viral methods for CAR transgene integration - Analysis of accelerated manufacturing strategies to reduce CAR-T production time - Investigation of automation and on-site manufacturing to standardize CAR-T cell production - Ways to bypass cryopreservation Main Findings: - CAR-T cell production times can be easily be brought under 48 hours - Non-viral methods not only reduce the risks associated with viral vectors, but could pave the way for more personalized gene-editing options - Automation may allow localized CAR-T manufacturing, potentially supporting non-cryopreserved, same-day treatment. Finally, in vivo manufacturing (though in early stages), could bypass costly ex vivo manufacturing all together. It remains to be seen. Kudos to the authors, fantastic work! Any thoughts on where this field is going? Drop them in the comments.

How negative gene therapy clinical trial has led to a positive outcome- approval of Sarepta’s ELEVIDYS by FDA https://lnkd.in/e2_YJg_t #genetherapy #fda

Big achievement for BlueRock Therapeutics and the cell therapy community. #CellTherapy #ParkinsonsDisease

𝐁𝐫𝐞𝐚𝐤𝐢𝐧𝐠 𝐍𝐞𝐰𝐬: Stem Cells Cure Type 1 Diabetes 👇 A 25-year-old woman with type 1 diabetes has started producing her own insulin less than three months after receiving a transplant of reprogrammed stem cells. This marks a groundbreaking achievement as she is the first patient with this disease to be treated using cells extracted from her own body. 𝐖𝐡𝐚𝐭’𝐬 𝐫𝐞𝐦𝐚𝐫𝐤𝐚𝐛𝐥𝐞? - Autonomous Insulin Production: The woman can now produce insulin naturally. - Incredible Lifestyle Change: “I can eat sugar now,” she said, expressing newfound freedom in her diet after the transplant. - Long-lasting Results: Over a year post-transplant, her insulin production remains stable. Full study, here: https://lnkd.in/eky2ZdGW #stemcells #celltherapy #biotech #CGTweekly

“We’re largely seeing investments shifting to things that are de-risked,” said Jon Norris, a managing director at HSBC Innovation Banking. “Cell and gene therapy is becoming an area we know better, because there are products that have advanced through development, but it still doesn't compare to the number of approvals and drugs commercially available in small molecules or biologics.” #gene #cell #gct #genetherapy #celltherapy

WATCH #OTAT Town Hall recording on clinical development of hashtag #GeneTherapy products for rare diseases to hear #FDA experts: https://lnkd.in/d3ww9X7Y https://lnkd.in/dvTZZP3U

Gene therapy restores vision in first-ever trial for rare, inherited #blindness #raredesease #genetherapy #gene https://lnkd.in/d-BQf5pm

#Nanobots to kill cancer cells in a mouse model https://lnkd.in/gwdTbVfi #Cell #Science #Innovation #Cancer #Technology #NanoTechnology #Robots #Robotics