The @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
@thebabaklab.bsky.social
@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.
📩 [email protected] or http://dlvr.it/TVYYdX
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights
@samillingworth.com💉 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.
@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
🔗 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.
@thebabaklab.bsky.socialThe @fda.gov has approved the first therapy for glycogen storage disease type Ia (GSDIa).
Genglycos (pariglasgene brecaparvovec-opnr) received accelerated approval for patients aged 8 years and older.
Link in the comments 👇
@longevitytech.bsky.socialA SIRT6 gene therapy trial in senior beagles hit its primary endpoint, lowering biological age and offering a test bed for human medicine.
Link as first comment.
@drtimos.bsky.social🚀 Big buzz at #ASGCT25: #GeneEditing & #CRISPR headlines, 20% data-driven talk, 85% positive vibes on breakthrough therapies for #DMD & #rarediseases!🧬 Download our full #socialmedia report! http://dlvr.it/TVSzLc #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT
@wimi-bio-tech.bsky.socialFudan University researchers developed #etBE, an engineered Transformer #BaseEditor with up to 35-fold higher C-to-T editing efficiency that achieved 35.13% in vivo liver editing and a 33% LDL-C reduction in mice without off-target trade-offs. 🧬🤖 #FudanUniversity #GeneTherapy #PCSK9
@sigtrans-sttt.bsky.social#Biologics are broadening therapeutic strategies for cardiovascular disease.
This review highlights advances in #CardiacRegeneration, reverse remodeling, #GeneTherapy, vascular function and lipid modulation.
@wimi-bio-tech.bsky.socialTU Munich researchers used RFdiffusion AI to create synthetic transfer vehicles (STV-C8) that outperform LNPs by >1,000-fold in RNA delivery and achieve in vivo CRISPR editing in pigs. 🧬🤖 #Nature2026 #RNADelivery #RFdiffusion #CRISPR #GeneTherapy #STVC8
@pharmaphorum.bsky.socialRight after reporting encouraging data with its #Dravetsyndrome #genetherapy, #EncodedTherapeutics has raised an impressive $275 million to take the programme into a pivotal trial.
@cmri.bsky.socialA child's growing liver has long worked against #genetherapy. #CMRI researchers found a way to repair the faulty gene at its source instead of adding a copy that gets diluted over time. Early results are promising. Full publication here: https://doi.org/10.1016/j.ymthe.2026.06.044
@umasschan.bsky.socialA multidisciplinary UMass Chan research team has received $14.7 million to develop new models and gene therapies for alpha-1 antitrypsin deficiency, a genetic defect which causes severe loss of lung function: https://bit.ly/4cEbJj7
#GeneTherapy @xueumass.bsky.social @rti-umasschan.bsky.social