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Two hemophilia A patients developed persistent high-titer FVIII inhibitors following AAV-mediated gene therapy with a bioengineered FVIII transgene despite prior extensive FVIII exposure.
An open-label trial demonstrates the safety and potential efficacy of ChrimsonR-based optogenetic therapy combined with specialized goggles to enhance light sensitivity in patients with advanced retinitis pigmentosa, offering promising aven
This study demonstrates that one-stage assay (OSA) factor VIII activity better reflects the functional enhancement of AAV-derived FVIII in hemophilia A gene therapy, offering superior prediction of bleeding phenotype compared to chromogenic
A retrospective study demonstrates the safety and efficacy of GCSF and plerixafor for CD34+ stem cell mobilization as autologous backup in sickle cell patients undergoing alternate donor HSCT, with insights relevant for gene therapy.
This article reviews the first-in-human study demonstrating durable and safe posttranscriptional silencing of BCL11A in sickle cell disease patients using a shmiR vector, resulting in stable fetal hemoglobin induction and clinical benefit o
This review synthesizes evidence from a pioneering nonrandomized trial demonstrating that intravenous AAVrh.10hFXN gene therapy is safe and may improve cardiac outcomes in Friedreich ataxia cardiomyopathy, highlighting translational advance
An optimized AAV5 vector delivering a stabilized human RPGR ORF15 gene significantly restores photoreceptor structure and function in RPGR-deficient mice, demonstrating a promising gene therapy for X-linked retinitis pigmentosa.
Gene therapy for cardiomyopathy is moving from concept to clinic, but durable benefit will depend on solving delivery, immunogenicity, cargo, and safety barriers in the failing human heart.
This review synthesizes the trajectory of PCSK9 inhibitors, from pivotal monoclonal antibody trials to emerging RNA interference and gene-editing technologies, emphasizing their role in achieving ultra-low LDL-C levels and reducing major ad
This review analyzes the shifting landscape of SMA therapy using data from 2,140 patients, highlighting the transition between nusinersen, risdiplam, and onasemnogene abeparvovec and the factors driving treatment switches.
The Phase 3 CIFFREO trial found that the gene therapy fordadistrogene movaparvovec did not significantly improve motor function in boys with Duchenne muscular dystrophy compared to placebo, leading to the discontinuation of its clinical dev
The Phase 3 CIFFREO trial for the gene therapy fordadistrogene movaparvovec failed to meet its primary efficacy endpoint in boys with Duchenne muscular dystrophy, showing no significant functional improvement over placebo and leading to the
A Phase 1-2 trial of AAV9-GLB1 gene therapy in children with Type II GM1 gangliosidosis demonstrates significant biochemical improvements and stabilization of neurodevelopmental decline, marking a pivotal step toward treating this fatal lys
A phase 1-2 trial shows that autologous hematopoietic stem-cell gene therapy (CTNS-RD-04) safely reduces lysosomal cystine levels in patients with cystinosis, potentially offering a definitive alternative to lifelong cysteamine therapy.
The LEGenD-1 trial demonstrates that intramuscular HGF gene therapy (AMG0001) significantly reduces healing time for neuroischemic ulcers in patients with moderate chronic limb-threatening ischemia, offering a potential nonsurgical therapeu
A clinical study comparing OTL-203 gene therapy to allogeneic HSCT for Hurler syndrome reveals significant improvements in corneal clarity, hearing preservation, and cardiac stability, marking a potential shift in the standard of care for m
The Phase 3 VIITAL trial demonstrates that prademagene zamikeracel, an autologous gene-modified cellular sheet, significantly improves healing and reduces pain in patients with recessive dystrophic epidermolysis bullosa (RDEB), offering a t
The final 5-year analysis of the HOPE-B trial confirms that etranacogene dezaparvovec provides stable, long-term factor IX expression and clinical protection for patients with hemophilia B, effectively eliminating the need for routine proph
The FANCOLEN‑1 trial shows that infusion of autologous FANCA‑corrected CD34+ cells without cytotoxic conditioning produced sustained engraftment and clinical improvement in a majority of treated patients with Fanconi anaemia‑A, with an acce
A single‑arm trial of AAV‑OTOF (Anc80L65) in 10 patients (1.5–23.9 years) with autosomal recessive deafness 9 showed good tolerability and rapid, clinically meaningful hearing gains, with optimal outcomes in children aged 5–8 years; longer
