American Society of Gene & Cell Therapy’s Post

A novel gene therapy technique demonstrates potential for treating Duchenne muscular dystrophy. https://lnkd.in/dXHJBC8T

Breakthrough Gene Therapy for Glaucoma Shows Promise in Vision Restoration https://lnkd.in/eNpdkPk3

Advancements in gene therapy are transforming the future of vision restoration! 👁️ Groundbreaking gene therapy for vision: A recent study shows promising results using the AAV5-based gene therapy ATSN-101 to treat Leber congenital amaurosis 1 (LCA1), an inherited retinal disease causing early blindness. ✨ Remarkable vision improvements: Patients in the trial experienced vision improvements of up to 10,000-fold, enabling some to see in low light after decades of blindness. ⏳ Lasting impact: Vision improvements were observed within a month and persisted for over 12 months, with ongoing patient monitoring. 🌟 Advancing hope for infant blindness: This success adds to UPenn's previous gene therapy trials, offering hope for treating 20% of cases of infantile blindness caused by inherited retinal diseases. #GeneTherapy #Innovation #Biotech #MedicalBreakthrough ➡️ Read this article for more details: https://lnkd.in/eFBHtgJZ

𝐎𝐧𝐞 𝐦𝐨𝐫𝐞 𝐢𝐧𝐜𝐫𝐞𝐝𝐢𝐛𝐥𝐞 𝐚𝐝𝐯𝐚𝐧𝐜𝐞𝐦𝐞𝐧𝐭 𝐢𝐧 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲, 𝐭𝐡𝐢𝐬 𝐭𝐢𝐦𝐞 𝐟𝐨𝐫 𝐕𝐢𝐬𝐢𝐨𝐧 𝐋𝐨𝐬𝐬! 🌟 Researchers from the 𝐏𝐞𝐫𝐞𝐥𝐦𝐚𝐧 𝐒𝐜𝐡𝐨𝐨𝐥 𝐨𝐟 𝐌𝐞𝐝𝐢𝐜𝐢𝐧𝐞 𝐚𝐭 𝐭𝐡𝐞 𝐔𝐧𝐢𝐯𝐞𝐫𝐬𝐢𝐭𝐲 𝐨𝐟 𝐏𝐞𝐧𝐧𝐬𝐲𝐥𝐯𝐚𝐧𝐢𝐚 have successfully used 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 to dramatically improve vision in patients with 𝐋𝐞𝐛𝐞𝐫 𝐜𝐨𝐧𝐠𝐞𝐧𝐢𝐭𝐚𝐥 𝐚𝐦𝐚𝐮𝐫𝐨𝐬𝐢𝐬 (𝐋𝐂𝐀1) — 𝐚 𝐫𝐚𝐫𝐞 𝐠𝐞𝐧𝐞𝐭𝐢𝐜 𝐜𝐨𝐧𝐝𝐢𝐭𝐢𝐨𝐧 𝐜𝐚𝐮𝐬𝐢𝐧𝐠 𝐞𝐚𝐫𝐥𝐲 𝐜𝐡𝐢𝐥𝐝𝐡𝐨𝐨𝐝 𝐛𝐥𝐢𝐧𝐝𝐧𝐞𝐬𝐬. 👁️ Some patients experienced 𝐯𝐢𝐬𝐢𝐨𝐧 𝐢𝐦𝐩𝐫𝐨𝐯𝐞𝐦𝐞𝐧𝐭𝐬 𝐨𝐟 𝐮𝐩 𝐭𝐨 10,000-𝐟𝐨𝐥𝐝! One participant even described seeing outdoors at midnight by the light of a bonfire 🔥— something unimaginable before. 🙌 This breakthrough highlights 𝐭𝐡𝐞 𝐩𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥 𝐨𝐟 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 𝐭𝐨 𝐭𝐫𝐚𝐧𝐬𝐟𝐨𝐫𝐦 𝐥𝐢𝐯𝐞𝐬, especially in rare conditions where treatment options are limited. The results of this clinical trial, recently published in 𝐓𝐡𝐞 𝐋𝐚𝐧𝐜𝐞𝐭, bring hope to the estimated 100,000 𝐩𝐞𝐨𝐩𝐥𝐞 𝐰𝐨𝐫𝐥𝐝𝐰𝐢𝐝𝐞 𝐥𝐢𝐯𝐢𝐧𝐠 𝐰𝐢𝐭𝐡 𝐋𝐂𝐀1. With further trials planned, we could be on the path toward offering a new future of sight for many!🔬 Read more here:

#spinalcordinjury #genetherapy #repair The potential of gene therapies for spinal cord injury repair: a systematic review and meta-analysis of pre-clinical studies https://lnkd.in/gCMFTxkA Catriona Cunningham University of Aberdeen Currently, there is no cure for traumatic spinal cord injury but one therapeutic approach showing promise is gene therapy. In this systematic review and meta-analysis, we aim to assess the efficacy of gene therapies in pre-clinical models of spinal cord injury and the risk of bias.  

🚀 Exciting News in Parkinson's Treatment 🌟 MeiraGTx is pioneering advancements in Parkinson's disease treatment with promising results from their midphase trial of AAV-GAD gene therapy. The therapy, designed to improve motor function and quality of life, demonstrated significant results and is now eyeing a Phase 3 trial. In a study involving 14 patients, those on a high dose of AAV-GAD improved by 18 points on motor function at 26 weeks, a substantial leap compared to the lower doses and sham treatments. Additionally, quality of life saw an 8-point boost. These results highlight the potential for gene therapy to reprogram brain circuits and address motor dysfunction caused by Parkinson's. ✅ No serious adverse events related to AAV-GAD ✅ Significant and meaningful changes in key efficacy endpoints ✅ High-dose group showed marked improvements in motor function and quality of life As MeiraGTx discusses Phase 3 trials with regulators globally, insights from these studies bring hope to many. Having an effective one-time treatment could be a game-changer for Parkinson's patients.🔬 #GeneTherapy #ParkinsonsDisease #ClinicalTrials

The advancements in neovascular AMD therapy have been remarkable. Moving beyond just intravitreal anti-VEGF biologics to targeting additional pathways. The port delivery system allows more sustained administration is another step towards better compliance. The potential addition of several TKIs which target multiple VEGF and related receptors combining with advanced formulation to allow longer term delivery will be another significant step forward, no matter it is delivered intravitrally or suprachoroidally. Gene therapy, particularly when utilized as a biofactory, opens up new possibilities for treatment and can be delivered through various methods including intravitreally, suprachoroidally, and subretinally as summaried nicely by Lucas and Tom. These innovations hold promise for improving outcomes and expanding options for patients with neovascular AMD.

Embracing Innovation: The Future of Gene Therapy The Princess Royal, officially opened the Gene Therapy Innovation and Manufacturing Centre (GTIMC) at the University of Sheffield on October 2, 2024. The recent opening of the Pioneering Gene Therapy Innovation and Manufacturing Centre marks a significant milestone in medical science. As healthcare leaders, we stand on the cusp of a new era, where gene therapy holds the potential to revolutionize the treatment of genetic disorders and chronic diseases. These innovations offer hope to millions, enabling us to tackle conditions previously deemed untreatable. The advanced therapies like gene therapy will undoubtedly accelerate research, enhance manufacturing capabilities, and ensure faster access to life-changing treatments. The possibilities extend far beyond current capabilities, with potential applications in oncology, neurology, and personalized medicine. With gene therapy at the forefront of medical advancement, we must cultivate an environment that fosters innovation while remaining patient-focused. The opening of this centre is not just a step forward in treatment development—it’s a reminder that healthcare leaders must be visionary, adaptable, and ready to embrace the future. I look forward to seeing how such innovations will shape the future of care in our hospitals and across the industry as a whole. Reference: https://lnkd.in/dhD_qNs2

Gene therapy helps restore hearing in children. “Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results” https://lnkd.in/gPZkxCBt

Gene therapy restores hearing in children with hereditary deafness A novel gene therapy for hearing loss was administered to six children in China in a clinical trial. Each child had an inherited deafness caused by mutations in the OTOF gene, called DFNB9. The researchers report in a new study, after 26 weeks, five children demonstrated hearing recovery and dramatic improvements in speech perception and the restored ability to conduct normal conversation. With its first patient treated in December 2022, this research represents the first human clinical trial to administer gene therapy for treating this form of hearing loss, with the most patients treated and longest follow-up conducted to date. https://lnkd.in/eJDzPgrp