@adityabasu.bsky.socialFDA clears UX111: The 1st systemic AAV9 gene therapy for pediatric Sanfilippo syndrome Type A (MPS IIIA)! Crossing the blood-brain barrier via IV infusion, it cuts CSF heparan sulfate >65% and stabilizes cognition.
Read full feature: url.nextechie.in/2nADch
@moltherapy.bsky.socialTODAY at 11 AM CDT: Molecular Therapy Presents - AI-Powered Precision Medicine: From Biomarker Discovery to Therapeutic Design and Delivery
Join us for this exciting FREE webinar today: http://dlvr.it/TVcMM4
#ArtificialIntelligence #GeneTherapy #RNAtherapeutics #ASGCT
@magic-horizon.bsky.socialMeet the people behind MAGIC 🎥
Dr Sinéad Ryan from Maynooth University works alongside Prof Karen English, exploring the immune challenges linked to gene therapies for muscular dystrophies. Their work is helping build a stronger foundation for safe and effective treatments.
@genethonfr.bsky.social📢 #WMS2026 Sept. 30
🎙️ Oral Session 3 (3:30–4:00 PM)
Mario Amendola: CRISPR-based utrophin upregulation for Duchenne muscular dystrophy
Vincent Laugel: Long-term Phase 1/2 results of GNT0004 gene therapy
đź“„ 3 posters from @genethonfr.bsky.social
(5:15–6:15 PM)
@jmirlatestresearch.bsky.socialApplication of aiAtlas Version 1.2 to Simulate Variant Gene Function Restoration and Rescue Thresholds in Rare Diseases: Validation Case Study #ArtificialIntelligence #Genomics #RareDiseases #GeneTherapy #BiomedicalResearch
@dcrinews.bsky.socialThe DCRI is proud to be part of the Bespoke Gene Therapy Consortium, helping accelerate gene therapy research for rare diseases. Learn more: dcri.org/news/dcri-se...
#RareDiseaseResearch #GeneTherapy
@emg-gold.comBeacon Therapeutics’ gene therapy laru-zova has met its primary goal in a pivotal trial for X-linked retinitis pigmentosa, a rare eye disease with no approved treatments.
Read the full story: www.emjreviews.com/emj-gold/new...
@pharmaphorum.bsky.social#BeaconTherapeutics is preparing to file for approval of its #genetherapy for eye disorder #Xlinkedretinitispigmentosa (XLRP) after reporting positive results in a pivotal trial.
@coherebtmi792.bsky.socialCell Banking Outsourcing Market Trends, Size, and Growth Analysis 2026-2033
Read full blog on our website:
www.coherentmarketinsights.com/market-insig...
#CoherentMarketInsights #CellBankingOutsourcingMarket #Biopharmaceuticals #Biopharma #Biotechnology #GeneTherapy #CellTherapy
@prof-clark.bsky.socialCould functional vision be the future of retinal gene-therapy approvals? Beacon’s VISTA win in XLRP puts patient-relevant vision outcomes centre stage. #GeneTherapy #Ophthalmology buff.ly/MODKvwq
@thebabaklab.bsky.socialThe @fda.gov has approved the first gene therapy for children with Sanfilippo syndrome type A.
Link in the comments 👇
#ClinicalMondays #GeneTherapy #SanfilippoSyndrome #Pediatrics #OrphanDrug #FastTrack #BreakthroughTherapy
@wimi-bio-tech.bsky.social#PekingUniversity researchers discovered PmCas12m—a naturally nuclease-dead miniature Cas nuclease—and engineered #xCas12m (581 aa), enabling #single-AAV packaging of xCas12m-CRISPRoff for durable in vivo HBV epigenetic silencing in mice. 🧬🤖 #Epigenetics #CRISPRoff #GeneTherapy #HBV
@drtimos.bsky.social🧬 Gene & Cell Therapy | #RareDiseases 🚀
#LucidQuest delivers sharp insights to help biotechs, investors & BD teams stay ahead in fast-moving markets.
đź“© [email protected] or http://dlvr.it/TVYYdX
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights
@samillingworth.comđź’‰ One shot cut bleeding episodes 96%
Chinese teenagers with haemophilia B got one gene therapy infusion. A year on their annual bleeding rate fell from 13.9 episodes to 0.5, as their bodies made the clotting protein they'd been missing.
đź”— doi.org/10.1038/s41591-026-04636-8
#GeneTherapy #Health 🧪
@emg-gold.comUltragenyx has set a $3.95m US list price for Fayuvi, the first FDA-approved treatment for the fatal rare disease Sanfilippo syndrome Type A.
The one-time gene therapy could reach peak worldwide sales of up to $250m, according to analysts.
@elsihub.bsky.socialHow has the death of Jesse Gelsinger shaped gene therapy today?
Join Dr. Arthur L. Caplan and Dr. Alison Bateman-House for a special #ELSIconversations session.
The Death of Jesse Gelsinger: A Complicated Ethical Legacy
Oct. 2 | 12–1:30 PM ET
đź”— evt.to/gmaioguhw
@pharmaphorum.bsky.socialPatients in the #US with #SanfilipposyndrometypeA have their first #FDA-approved treatment, #UltragenyxPharma's #genetherapy #Fayuvi, which will launch with a $3.9 million price tag.
@wimi-bio-tech.bsky.socialMohankumar et al. demonstrated that #truncatinggRNAs to 15–19 nt narrows the #baseediting window to suppress bystander mutations across ABE and CBE platforms, with 17 nt guides optimizing target precision during transient delivery in primary CD34+ HSPCs. 🩸🤖 #BystanderEditing #HSPCs #GeneTherapy
@drtimos.bsky.socialWhat’s shaping CVC investments for 2025? Key trends: AI/ML, Gene Therapy, and a focus on Immune Disorders & Neurodegenerative Diseases. Curious for more? 📥 Download the full report for insights! http://dlvr.it/TVVnNN
#CVC #InvestingInInnovation #VentureCapital #GeneTherapy #AI
@dnanexus.bsky.socialOur team will be at the UC San Diego Gene Therapy Initiative Symposium, 16–17 September in La Jolla.
Schedule time to meet with us to learn how we enable pharma organizations to accelerate their time to insights for early-response results.
