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Optimizing Autologous Stem Cell Backup Mobilization with GCSF and Plerixafor in Sickle Cell Disease: Implications for Gene Therapy
Hematology-Oncologygene therapysickle cell disease

Optimizing Autologous Stem Cell Backup Mobilization with GCSF and Plerixafor in Sickle Cell Disease: Implications for Gene Therapy

By MedXY|Sep 18, 2026

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.

Sustained Genetic Silencing of BCL11A Using shmiR Vector in Sickle Cell Disease: Long-Term Safety and Efficacy
Hematology-Oncologygene therapy
BCL11A

Sustained Genetic Silencing of BCL11A Using shmiR Vector in Sickle Cell Disease: Long-Term Safety and Efficacy

By MedXY|Aug 24, 2026

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

AAVrh.10hFXN Gene Therapy for Friedreich Ataxia Cardiomyopathy: Safety and Preliminary Efficacy in a Nonrandomized Clinical Trial
CardiologyAAVrh.10hFXNcardiomyopathy

AAVrh.10hFXN Gene Therapy for Friedreich Ataxia Cardiomyopathy: Safety and Preliminary Efficacy in a Nonrandomized Clinical Trial

By MedXY|Jul 1, 2026

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

Optimized AAV5-RPGR Gene Therapy Rescues Photoreceptor Integrity and Function in X-Linked Retinitis Pigmentosa Mouse Model
newsAAV5gene therapy

Optimized AAV5-RPGR Gene Therapy Rescues Photoreceptor Integrity and Function in X-Linked Retinitis Pigmentosa Mouse Model

By MedXY|Jun 13, 2026

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.

Cardiomyopathy Gene Therapy Reaches an Inflection Point: From AAV Delivery to Precision Genome Editing
CardiologyAAVcardiomyopathy

Cardiomyopathy Gene Therapy Reaches an Inflection Point: From AAV Delivery to Precision Genome Editing

By MedXY|May 12, 2026

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.

Evolution and Future Frontiers of PCSK9 Inhibition: From Genetic Discovery to Genomic Medicine
Clinical Updatescardiovascular outcomesgene therapy

Evolution and Future Frontiers of PCSK9 Inhibition: From Genetic Discovery to Genomic Medicine

By MedXY|Mar 25, 2026

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

Treatment Evolution in 5q-Spinal Muscular Atrophy: Real-World Evidence and Clinical Insights from the SMArtCARE Registry
Clinical Updatesgene therapyreal‑world evidence

Treatment Evolution in 5q-Spinal Muscular Atrophy: Real-World Evidence and Clinical Insights from the SMArtCARE Registry

By MedXY|Mar 8, 2026

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.

Fordadistrogene Movaparvovec Fails to Improve Functional Outcomes in Duchenne Muscular Dystrophy: Insights from the Phase 3 CIFFREO Trial
NeurologyBệnh Teo Cơ Bắp DuchenneCIFFREO Trial

Fordadistrogene Movaparvovec Fails to Improve Functional Outcomes in Duchenne Muscular Dystrophy: Insights from the Phase 3 CIFFREO Trial

By MedXY|Mar 8, 2026

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

CIFFREO Trial Results: Fordadistrogene Movaparvovec Fails Primary Efficacy Endpoint in Duchenne Muscular Dystrophy
NeurologyCIFFREO Trialgene therapy

CIFFREO Trial Results: Fordadistrogene Movaparvovec Fails Primary Efficacy Endpoint in Duchenne Muscular Dystrophy

By MedXY|Feb 24, 2026

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

AAV9 Gene Therapy Shows Clinical Promise in Stabilizing Type II GM1 Gangliosidosis
Neurologygene therapyneurology

AAV9 Gene Therapy Shows Clinical Promise in Stabilizing Type II GM1 Gangliosidosis

By MedXY|Feb 23, 2026

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

Breaking the Cystine Barrier: Long-term Safety and Efficacy of CTNS-RD-04 Gene Therapy in Cystinosis
Hematology-Oncologygene therapyhematopoietic stem cell transplantation

Breaking the Cystine Barrier: Long-term Safety and Efficacy of CTNS-RD-04 Gene Therapy in Cystinosis

By MedXY|Feb 22, 2026

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.

Anatomically Directed HGF Gene Therapy Significantly Accelerates Ulcer Healing in Chronic Limb-Threatening Ischemia: Insights from the LEGenD-1 Trial
CardiologyChronic Limb-Threatening Ischemiagene therapy

Anatomically Directed HGF Gene Therapy Significantly Accelerates Ulcer Healing in Chronic Limb-Threatening Ischemia: Insights from the LEGenD-1 Trial

By MedXY|Feb 4, 2026

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

Beyond the CNS and Skeleton: HSPC-Gene Therapy (OTL-203) Demonstrates Superior Multi-System Efficacy in Hurler Syndrome
Hematology-Oncologygene therapyhematopoietic stem cell transplantation

Beyond the CNS and Skeleton: HSPC-Gene Therapy (OTL-203) Demonstrates Superior Multi-System Efficacy in Hurler Syndrome

By MedXY|Jan 20, 2026

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

Breaking the Cycle of Chronic Wounds: Prademagene Zamikeracel Sets New Standard for RDEB Treatment
Dermatologydermatologygene therapy

Breaking the Cycle of Chronic Wounds: Prademagene Zamikeracel Sets New Standard for RDEB Treatment

By MedXY|Jan 7, 2026

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

Hematology-Oncologyclinical trialsgene therapy

Five-Year Durability of Etranacogene Dezaparvovec: Redefining the Long-term Management of Hemophilia B

By MedXY|Dec 19, 2025

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

Non‑conditioned Autologous Gene Therapy Reverses Bone Marrow Failure in Fanconi Anaemia‑A: FANCOLEN‑1 Phase 1/2 and Long‑Term Outcomes
Hematology-OncologyFanconi anaemiagene therapy

Non‑conditioned Autologous Gene Therapy Reverses Bone Marrow Failure in Fanconi Anaemia‑A: FANCOLEN‑1 Phase 1/2 and Long‑Term Outcomes

By MedXY|Nov 11, 2025

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

AAV‑OTOF Gene Therapy Restores Substantial Hearing in DFNB9 Across Ages — Rapid, Age‑dependent Benefits in a First-in‑Human Trial
newsAAVgene therapy

AAV‑OTOF Gene Therapy Restores Substantial Hearing in DFNB9 Across Ages — Rapid, Age‑dependent Benefits in a First-in‑Human Trial

By MedXY|Nov 4, 2025

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

OTOF Gene Therapy Rivaled—and in Some Domains Outperformed—Cochlear Implants in Restoring Hearing and Speech in Congenital Deafness
newsgene therapyOTOF

OTOF Gene Therapy Rivaled—and in Some Domains Outperformed—Cochlear Implants in Restoring Hearing and Speech in Congenital Deafness

By MedXY|Nov 4, 2025

A 2022–2024 nonblinded cohort study found OTOF gene therapy produced rapid, durable gains in auditory thresholds, speech perception, mismatch-negativity responses, and music perception versus cochlear implantation in children with genetical

DB-OTO Restores and Sustains Hearing in Otoferlin-Deficient Mice: A Hair-Cell–Targeted Dual-AAV Strategy Advances to Clinical Testing
newsgene therapyOTOF

DB-OTO Restores and Sustains Hearing in Otoferlin-Deficient Mice: A Hair-Cell–Targeted Dual-AAV Strategy Advances to Clinical Testing

By MedXY|Nov 4, 2025

DB-OTO, a hair-cell-specific dual-AAV gene therapy encoding full-length human otoferlin, achieved dose-dependent and sustained auditory restoration in OTOF-deficient mice, supporting an ongoing Phase I/II pediatric trial.

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
Allergy & Immunologygene therapylong-term outcomes

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency

By MedXY|Oct 25, 2025

This study demonstrates the long-term effectiveness and safety of autologous gene therapy for treating ADA deficiency, showing sustained immune function and minimal adverse effects over a median follow-up of 7.5 years.

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