Gene therapy https://www.bioworld.com/rss en-us The domino effect of cutting mRNA vaccine research http://www.bioworld.com/articles/724135 Mon, 15 Sep 2025 09:00:00 -0400 https://www.bioworld.com/articles/724135-the-domino-effect-of-cutting-mrna-vaccine-research Vial and syringe in row of dominoes Latus Bio’s LTS-101 is a potential therapeutic approach for Batten disease http://www.bioworld.com/articles/720557 Mon, 26 May 2025 09:00:00 -0400 https://www.bioworld.com/articles/720557-latus-bios-lts-101-is-a-potential-therapeutic-approach-for-batten-disease DNA data illustration CMV history cited in DMD liver death on Sarepta’s Elevidys http://www.bioworld.com/articles/718365 Tue, 18 Mar 2025 11:00:00 -0400 https://www.bioworld.com/articles/718365-cmv-history-cited-in-dmd-liver-death-on-sareptas-elevidys Stock table, gold arrow pointing down Series A to advance Hervolution’s HERV-targeted immunotherapies http://www.bioworld.com/articles/715689 Tue, 24 Dec 2024 09:00:00 -0500 https://www.bioworld.com/articles/715689-series-a-to-advance-hervolutions-herv-targeted-immunotherapies Dollar sign in light bulb on yellow background Series A financing at Somagenetix to advance gene therapy for chronic granulomatous disease http://www.bioworld.com/articles/713267 Wed, 09 Oct 2024 09:00:00 -0400 https://www.bioworld.com/articles/713267-series-a-financing-at-somagenetix-to-advance-gene-therapy-for-chronic-granulomatous-disease ATR12-351 demonstrates functional LEKTI delivery throughout epidermis in Netherton syndrome models SPINK5), which encodes lympho-epithelial Kazal-type-related inhibitor (LEKTI).]]> http://www.bioworld.com/articles/709020 Wed, 29 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/709020-atr12-351-demonstrates-functional-lekti-delivery-throughout-epidermis-in-netherton-syndrome-models Podocyte-specific gene therapy for nephrotic syndrome http://www.bioworld.com/articles/708844 Fri, 24 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708844-podocyte-specific-gene-therapy-for-nephrotic-syndrome Illustration showing parts of a kidney nephron AAV9-CTNNB1 gene therapy restores β-catenin expression and function in CTNNB1 syndrome CTNNB1, which plays a critical role in neuronal development, synapse formation and brain maturation.]]> http://www.bioworld.com/articles/708830 Thu, 23 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708830-aav9-ctnnb1-gene-therapy-restores-catenin-expression-and-function-in-ctnnb1-syndrome Optimized nonviral LNP system for hFVIII, P-FVIII-101, normalizes clotting efficacy in models of hemophilia A http://www.bioworld.com/articles/708682 Wed, 22 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708682-optimized-nonviral-lnp-system-for-hfviii-p-fviii-101-normalizes-clotting-efficacy-in-models-of-hemophilia-a Adolore Biotherapeutics makes progress with nonopioid analgesic gene therapies for chronic pain http://www.bioworld.com/articles/708681 Wed, 22 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708681-adolore-biotherapeutics-makes-progress-with-nonopioid-analgesic-gene-therapies-for-chronic-pain Knee pain illustration P-KLKB1-101 demonstrates highly specific on-target KLKB1 editing in preclinical models http://www.bioworld.com/articles/708650 Tue, 21 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708650-p-klkb1-101-demonstrates-highly-specific-on-target-klkb1-editing-in-preclinical-models New subretinal gene therapy in rodent models of geographic atrophy http://www.bioworld.com/articles/708535 Thu, 16 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708535-new-subretinal-gene-therapy-in-rodent-models-of-geographic-atrophy Optogenetics illustration Decades of studies on gene and cell therapies lead to ASGCT hits http://www.bioworld.com/articles/708527 Thu, 16 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708527-decades-of-studies-on-gene-and-cell-therapies-lead-to-asgct-hits Art concept for gene therapy research ACDN-01 results in robust, durable and well-tolerated ABCA4 exon editing in preclinical models http://www.bioworld.com/articles/708509 Wed, 15 May 2024 00:00:00 -0400 https://www.bioworld.com/articles/708509-acdn-01-results-in-robust-durable-and-well-tolerated-abca4-exon-editing-in-preclinical-models ASGCT: ‘From darkness to light’ in ocular gene therapy http://www.bioworld.com/articles/708607 Tue, 14 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708607-asgct-from-darkness-to-light-in-ocular-gene-therapy Vial and syringe with DNA PR-009, a new AAV-hTREM2 gene therapy to restore microglial function in patients with ALSP-CSF1R CSF1R gene have been linked to adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).]]> http://www.bioworld.com/articles/708482 Tue, 14 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708482-pr-009-a-new-aav-htrem2-gene-therapy-to-restore-microglial-function-in-patients-with-alsp-csf1r Preclinical safety and efficacy of intein-based dual-AAV gene therapy for Stargardt disease ABCA4 gene with no approved treatment on the market.]]> http://www.bioworld.com/articles/708416 Fri, 10 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708416-preclinical-safety-and-efficacy-of-intein-based-dual-aav-gene-therapy-for-stargardt-disease Eye and DNA Crispr Therapeutics announces new programs targeting refractory hypertension and acute hepatic porphyria http://www.bioworld.com/articles/708414 Fri, 10 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708414-crispr-therapeutics-announces-new-programs-targeting-refractory-hypertension-and-acute-hepatic-porphyria CRISPR Cas9 illustration CRISPR. Credit: Ernesto del Aguila III, National Human Genome Research Institute, NIH ASGCT: ‘From darkness to light’ in ocular gene therapy http://www.bioworld.com/articles/708411 Fri, 10 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708411-asgct-from-darkness-to-light-in-ocular-gene-therapy Vial and syringe with DNA ASGCT: In utero interventions can prevent organ damage after birth http://www.bioworld.com/articles/708377 Thu, 09 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708377-asgct-in-utero-interventions-can-prevent-organ-damage-after-birth Concept art for prenatal genetic testing and whole genome sequencing. Credit: Ernesto del Aguila III, National Human Genome Research Institute, NIH Soluble Fas ligand prevents disease progression in glaucoma model http://www.bioworld.com/articles/708169 Mon, 06 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708169-soluble-fas-ligand-prevents-disease-progression-in-glaucoma-model Latus Bio launches with focus on gene therapies for CNS disorders http://www.bioworld.com/articles/708135 Fri, 03 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708135-latus-bio-launches-with-focus-on-gene-therapies-for-cns-disorders Central nervous system Poseida Therapeutics advances nonviral liver-directed gene therapies http://www.bioworld.com/articles/707690 Thu, 18 Apr 2024 09:00:00 -0400 https://www.bioworld.com/articles/707690-poseida-therapeutics-advances-nonviral-liver-directed-gene-therapies Liver Circio establishes in vivo proof of concept for Circvec circular RNA platform http://www.bioworld.com/articles/707687 Thu, 18 Apr 2024 09:00:00 -0400 https://www.bioworld.com/articles/707687-circio-establishes-in-vivo-proof-of-concept-for-circvec-circular-rna-platform mRNA on digital background Saliogen nominates development candidate for Stargardt disease http://www.bioworld.com/articles/707684 Thu, 18 Apr 2024 09:00:00 -0400 https://www.bioworld.com/articles/707684-saliogen-nominates-development-candidate-for-stargardt-disease Medical vector illustration showing cross section of an eyeball with close up of the macula NHLBI grant supports Geneventiv’s development of universal gene therapy for hemophilia http://www.bioworld.com/articles/707646 Wed, 17 Apr 2024 09:00:00 -0400 https://www.bioworld.com/articles/707646-nhlbi-grant-supports-geneventivs-development-of-universal-gene-therapy-for-hemophilia Red blood cells, DNA Voyager and Neurocrine nominate development candidate in GBA1 gene therapy program for Parkinson’s disease GBA1 gene therapy program for the treatment of Parkinson’s disease and other GBA1-mediated diseases under its collaboration with Neurocrine Biosciences Inc.]]> http://www.bioworld.com/articles/707624 Tue, 16 Apr 2024 09:00:00 -0400 https://www.bioworld.com/articles/707624-voyager-and-neurocrine-nominate-development-candidate-in-gba1-gene-therapy-program-for-parkinsons-disease DNA, drug development concept art. Credit: Ernesto del Aguila III, National Human Genome Research Institute, NIH Precision Biosciences presents promising results for PBGENE-PMM gene therapeutic http://www.bioworld.com/articles/707112 Tue, 02 Apr 2024 09:00:00 -0400 https://www.bioworld.com/articles/707112-precision-biosciences-presents-promising-results-for-pbgene-pmm-gene-therapeutic Atamyo’s gene therapy for LGMD2C/R5 cleared to enter clinic in Europe http://www.bioworld.com/articles/706998 Wed, 27 Mar 2024 09:00:00 -0400 https://www.bioworld.com/articles/706998-atamyos-gene-therapy-for-lgmd2c-r5-cleared-to-enter-clinic-in-europe DNA in drug capsules, digital background Samsung Ventures makes investment in Brickbio http://www.bioworld.com/articles/706677 Tue, 19 Mar 2024 10:00:00 -0400 https://www.bioworld.com/articles/706677-samsung-ventures-makes-investment-in-brickbio Scientist, microscope and dropper