Gene therapy https://www.bioworld.com/rss en-us A free gene therapy? Regeneron’s Otarmeni approved for hearing loss http://www.bioworld.com/articles/730604 Thu, 23 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730604-a-free-gene-therapy-regenerons-otarmeni-approved-for-hearing-loss Boy cupping ear with soundwave graphic AAV-based gene therapy encoding NeuroD1 inhibits glioma growth and extends survival http://www.bioworld.com/articles/730560 Wed, 22 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730560-aav-based-gene-therapy-encoding-neurod1-inhibits-glioma-growth-and-extends-survival Concept art for adeno-associated viral-based gene therapy. Entos and L-CMD Research Foundation collaborate http://www.bioworld.com/articles/730541 Tue, 21 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730541-entos-and-l-cmd-research-foundation-collaborate Deal illustration Pleased to own ya, Kelonia: Lilly’s $7B CAR T deal http://www.bioworld.com/articles/730467 Mon, 20 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730467-pleased-to-own-ya-kelonia-lillys-7b-car-t-deal Gold-encircled handshake Roche plots route to EMA approval for DMD gene therapy http://www.bioworld.com/articles/730404 Fri, 17 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730404-roche-plots-route-to-ema-approval-for-dmd-gene-therapy Green strand of DNA New guidance advises on assessing safety of gene editing http://www.bioworld.com/articles/730446 Wed, 15 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730446-new-guidance-advises-on-assessing-safety-of-gene-editing Gene editing illustration New guidance advises on assessing safety of gene editing http://www.bioworld.com/articles/730424 Tue, 14 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730424-new-guidance-advises-on-assessing-safety-of-gene-editing Gene editing illustration MRD word heard loud, clear in Allogene LBCL cema-cel phase II http://www.bioworld.com/articles/730278 Mon, 13 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730278-mrd-word-heard-loud-clear-in-allogene-lbcl-cema-cel-phase-ii Art concept for hematologic cancer LHON updates: Gene therapy progress; idebenone receives US CRL http://www.bioworld.com/articles/730074 Thu, 02 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730074-lhon-updates-gene-therapy-progress-idebenone-receives-us-crl Eye and DNA illustration FDA approves Rocket’s Kresladi as first gene therapy for LAD-I http://www.bioworld.com/articles/729913 Fri, 27 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729913-fda-approves-rockets-kresladi-as-first-gene-therapy-for-lad-i Transparent capsule with DNA and cells Efficient editing in skeletal muscle of dystrophic mice with SORT LNPs encapsulating Cas9 mRNA http://www.bioworld.com/articles/729881 Wed, 25 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729881-efficient-editing-in-skeletal-muscle-of-dystrophic-mice-with-sort-lnps-encapsulating-cas9-mrna Human body skeleton with DNA science background art Grant supports Tessera’s genetic approaches to HIV cure http://www.bioworld.com/articles/729794 Mon, 23 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729794-grant-supports-tesseras-genetic-approaches-to-hiv-cure DNA in drug capsules MDA: Vinay away, AA in play for Regenxbio gene therapy? http://www.bioworld.com/articles/729478 Thu, 12 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729478-mda-vinay-away-aa-in-play-for-regenxbio-gene-therapy Illustration of man holding magnifying glass to human body model showing muscle anatomy Lentiviral HSC gene therapy shows preclinical efficacy for GM1 gangliosidosis treatment http://www.bioworld.com/articles/729611 Thu, 12 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729611-lentiviral-hsc-gene-therapy-shows-preclinical-efficacy-for-gm1-gangliosidosis-treatment 3D illustration of brain neuron in lysosomal storage diseases AAV-based gene therapy for GBA1-related diseases, including Parkinson’s and Gaucher GBA1, is a ubiquitous lysosomal enzyme that breaks down lipid substrates, glucosylceramide (GL-1) and glucosylsphingosine (Lyso-GL1), into glucose and ceramide. Loss-of-function mutations in GBA1 reduce GCase activity, resulting in lipid accumulation within lysosomes and subsequent lysosomal dysfunction.]]> http://www.bioworld.com/articles/729610 Thu, 12 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729610-aav-based-gene-therapy-for-gba1-related-diseases-including-parkinsons-and-gaucher 3D rendering of adeno-associated viral vector SGT-212 restores FXN function in Friedreich’s ataxia models http://www.bioworld.com/articles/729588 Wed, 11 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729588-sgt-212-restores-fxn-function-in-friedreichs-ataxia-models Forecast brighter for Uniqure with Prasad’s departure? http://www.bioworld.com/articles/729434 Mon, 09 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729434-forecast-brighter-for-uniqure-with-prasads-departure Rare disease illustration Tenaya, Alnylam join in $1B+ cardio deal for novel genetic targets http://www.bioworld.com/articles/729477 Fri, 06 Mar 2026 09:00:00 -0500 https://www.bioworld.com/articles/729477-tenaya-alnylam-join-in-1b-cardio-deal-for-novel-genetic-targets Heart and DNA Tenaya, Alnylam join in $1B+ cardio deal for novel genetic targets http://www.bioworld.com/articles/729341 Thu, 05 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729341-tenaya-alnylam-join-in-1b-cardio-deal-for-novel-genetic-targets Heart and DNA FDA’s rare disease toolbox not fully used http://www.bioworld.com/articles/729300 Wed, 04 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729300-fdas-rare-disease-toolbox-not-fully-used Magnifying glass, clock and capsules Experts: Regulatory roadblocks stalling rare disease therapies http://www.bioworld.com/articles/729285 Tue, 03 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729285-experts-regulatory-roadblocks-stalling-rare-disease-therapies Red and white roadblock Cure Rare Disease signs ANO5-related disease partnership http://www.bioworld.com/articles/729153 Tue, 03 Mar 2026 08:00:00 -0500 https://www.bioworld.com/articles/729153-cure-rare-disease-signs-ano5-related-disease-partnership DNA, dollars illustration Uniqure sinks as FDA demands new trial for Huntington’s gene therapy http://www.bioworld.com/articles/729268 Mon, 02 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729268-uniqure-sinks-as-fda-demands-new-trial-for-huntingtons-gene-therapy Illustration of brain and DNA strands Beam Therapeutics reveals new program for PKU http://www.bioworld.com/articles/729116 Fri, 27 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/729116-beam-therapeutics-reveals-new-program-for-pku Hematologic DNA blood test US Fed Circuit protects 101 passage for some gene therapy patents http://www.bioworld.com/articles/729195 Tue, 24 Feb 2026 12:00:00 -0500 https://www.bioworld.com/articles/729195-us-fed-circuit-protects-101-passage-for-some-gene-therapy-patents Patent gears Tessera’s TSRA-196 designated orphan drug for AATD http://www.bioworld.com/articles/729019 Tue, 24 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/729019-tesseras-tsra-196-designated-orphan-drug-for-aatd DNA double helix under a magnifying glass CRISPR-Cas13d-mediated targeting of RasGRP3 shows selective anticancer effects in uveal melanoma http://www.bioworld.com/articles/728975 Mon, 23 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/728975-crispr-cas13d-mediated-targeting-of-rasgrp3-shows-selective-anticancer-effects-in-uveal-melanoma Eye and DNA Subretinal NRF2 gene therapy preserves retinal function in dry AMD models http://www.bioworld.com/articles/728955 Fri, 20 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/728955-subretinal-nrf2-gene-therapy-preserves-retinal-function-in-dry-amd-models Affinia’s AFTX-201 designated EU orphan drug http://www.bioworld.com/articles/728941 Thu, 19 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/728941-affinias-aftx-201-designated-eu-orphan-drug 3D illustration of heart cross section Apertura Gene Therapy signs FRRS1L license agreement http://www.bioworld.com/articles/728939 Thu, 19 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/728939-apertura-gene-therapy-signs-frrs1l-license-agreement Brain and DNA