CRISPR-based therapy for ATTR shows promising results in Phase 1 trial

A single dose of Nexiguran Ziclumeran (nex-z), a CRISPR-based gene editing therapy, led to rapid, deep, and durable TTR reductions (average ~90%) in hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN), with most patients showing stability or improvement in neuropathy and generally mild adverse events; ongoing phase 3 studies continue to support its potential as a one-time, disease-modifying treatment.

A new twist in gene therapy? Researchers have successfully used polypurine reverse Hoogsteen hairpins (PPRHs) to silence PCSK9, reduce cholesterol levels, and bypass the cost and complexity of monoclonal antibodies and siRNA therapies. In vivo data show ~50% PCSK9 and cholesterol reduction after just one injection, without triggering liver toxicity or inflammation. Is this the next step in democratizing LDL-C control? #GeneTherapy #PCSK9 #Cholesterol #PPRH #BiotechInnovation #Hypercholesterolemia #RNAtherapy #siRNA #LDLR #Cardiovascular https://lnkd.in/e9n4WaMU

🔬 REGENXBIO Completes Enrollment in Phase 3 Trials for Wet AMD Gene Therapy Regenxbio has announced full enrollment in its pivotal Phase 3 ATMOSPHERE and ASCENT studies for ABBV-RGX-314 (sura-vec), a one-time gene therapy being evaluated for the treatment of wet age-related macular degeneration (AMD). These global, multicenter trials, comparing sura-vec to ranibizumab and aflibercept, have enrolled over 1,200 participants across more than 200 clinical sites. The primary endpoint is visual acuity change, with topline data expected in Q4 2026. 🔗 Read more here: https://lnkd.in/dMNSvxHM #ClinicalTrial #GeneTherapy #AMD #Ophthalmology #VisionHealth #Treatment #Innovation #Regenxbio

OBN shares some positive news for #WetAMD patients and REGENXBIO. The ATMOSPHERE and ASCENT Phase III #ClinicalTrials for their one-time gene therapy treatment surabgene lomparvovec (sura-vec) have completed enrollment. The possible advent of a treatment option that could reduce the burden of frequent eye-injections is great news for patients. #genetherapy

Regeneron’s DB-OTO Gene Therapy Shows Breakthrough Hearing Restoration in Hereditary Hearing Loss in Phase 1 / 2 CHORD Trial Regeneron Pharmaceuticals has announced ground-breaking results from its investigational gene therapy DB-OTO, designed to treat profound genetic hearing loss caused by mutations in the otoferlin (OTOF) gene. Trial Results are published in The New England Journal of Medicine, the data from the Phase 1/2 CHORD clinical trial show that nearly all treated pediatric patients experienced significant hearing improvements, with some achieving normal hearing levels. https://lnkd.in/dpjRBnNh #DBOTO #REGENERON #HEREDITARYHEARINGLOSS #OTOFGENE #CHORDTRIAL #FDA #FDAAPRROVAL #NEJM # #GeneTherapy ##Otoferlin #PediatricHealth #ClinicalTrials #MedicalBreakthrough 

🫀 Perseverance in Gene Therapy: When Technology Finally Catches Up The recent Nature Medicine paper on AB-1002 validates years of belief: the SERCA2a pathway wasn't wrong, we just needed better delivery tools. CUPID-2 failed because AAV1 couldn't adequately transduce the heart. The innovation here? A novel AAV2i8 capsid delivering 3-10x higher doses, plus targeting PP1 inhibition upstream rather than SERCA2a directly. Phase 1 shows no treatment-related AEs and confirmed high myocardial transduction. The lesson: Sometimes you wait for technology to match the science. Those who gave up after CUPID-2 missed the point. We didn't need a new target; we needed better delivery. Too early for efficacy claims? Of course. It's only 11 patients. But after years following this approach, seeing successful cardiac transduction feels like vindication for those who persevered. Good science sometimes just needs the right tools and patience. https://lnkd.in/eQj8UEsx #GeneTherapy #HeartFailure #Innovation

💉 A single infusion of the investigational in vivo gene-editing therapy, nexiguran ziclumeran, has shown "profound and durable" efficacy against hereditary transthyretin (ATTR) amyloidosis with polyneuropathy. 🌟According to a multinational Phase 1 trial led by University College London’s National Amyloidosis Center, the one-time treatment produced a rapid and sustained mean reduction of 92% in the misfolded TTR protein at 24 months. For the majority of the 36 patients, polyneuropathy disability scores either remained stable or improved, suggesting that this single-dose approach could offer a viable, long-term alternative to current therapies that require repeated, lifelong administration. 🔬Nexiguran ziclumeranis a lipid nanoparticle (LNP)-based therapy that delivers a CRISPR-Cas9 gene editing construct to the liver to treat ATTR. The findings, published in The New England Journal of Medicine, strongly support continued development of this therapy for the rare, fatal, and debilitating disease. #GeneEditing #Amyloidosis #ATTR #RareDisease #GeneTherapy #CRISPRCas9 #LNP #PackGene https://lnkd.in/eFAn9N4P

Today, JCR Pharmaceuticals presented new non-clinical data demonstrating the ability of its proprietary JUST-AAV capsid engineering platform to achieve efficient delivery of adeno-associated virus (AAV) gene therapy to the central nervous system (CNS) and muscle while reducing liver exposure.    The data, presented during an oral session at the European Society of Gene and Cell Therapy 32nd Annual Congress, showcase the platform’s potential to overcome longstanding challenges in treating CNS and muscle disorders.    JCR researchers reported that the JUST-AAV platform enables the precise and efficient delivery of an AAV gene therapy across the blood-brain barrier (BBB) and into the CNS, as well as to the muscle in mice and monkeys.    Read the full news release here: https://bit.ly/4h2YSI4.   #RareDisease #LysosomalStorageDisorders #JCRPharma #BloodBrainBarrier #Neurodegeneration #CNS #GeneTherapy #ESGCT2025 

Intellia halts 2 gene therapy trials after patient hospitalized Intellia Therapeutics has temporarily paused dosing and screening for its phase 3 Magnitude and Magnitude-2 trials of Nex-z following a patient hospitalization, according to an Oct. 27 news release. https://lnkd.in/g2jpCRZ8

Intellia halts 2 gene therapy trials after patient hospitalized Intellia Therapeutics has temporarily paused dosing and screening for its phase 3 Magnitude and Magnitude-2 trials of Nex-z following a patient hospitalization, according to an Oct. 27 news release. https://lnkd.in/gh_r-GpW