gene therapy – Page 2

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

By MedXY 11/04/2025

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 genetically driven congenital deafness.

news Otorhinolaryngology Pediatrics

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

By MedXY 11/04/2025

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.

Allergy & Immunology Hematology-Oncology news

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

By MedXY 10/25/2025

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.

news Otorhinolaryngology Pediatrics

DB-OTO Gene Therapy: A Breakthrough Treatment for OTOF-Related Inherited Deafness in Children

By MedXY 10/24/2025

Regeneron's DB-OTO gene therapy shows promising results in restoring natural hearing in children with profound hearing loss caused by OTOF gene mutations, with 75% achieving significant hearing improvement after a single treatment.

Hematology-Oncology news Ophthalmology

Advances in Targeted and Gene Therapies: Promising Data on KRAS G12D Inhibitors, DR5 Agonists, and Ocular Gene Therapy

By MedXY 10/24/2025

Recent clinical trials report encouraging tumor shrinkage with KRAS G12D inhibitor VS-7375, significant progression-free survival benefit in soft tissue sarcoma with ozekibart, and a novel gene therapy acquisition targeting wet AMD.

Allergy & Immunology Clinical Updates Dermatology Pediatrics Specialties

Prademagene Zamikeracel: A Breakthrough Gene Therapy for Chronic Wounds in Recessive Dystrophic Epidermolysis Bullosa (RDEB)

By MedXY 08/02/2025

A phase 3 trial demonstrates prademagene zamikeracel significantly accelerates healing and reduces pain in large, chronic RDEB wounds, offering a promising gene therapy with a favorable safety profile.

Clinical Updates Hematology-Oncology news Specialties

Haematopoietic Gene Therapy Without Conditioning for Fanconi Anaemia-A: Results and Clinical Implications from the FANCOLEN-1 Trials

By MedXY 08/02/2025

Gene therapy using autologous, gene-corrected stem cells without conditioning offers sustained engraftment and reverses bone marrow failure in Fanconi anaemia-A, with a favorable safety profile.

Clinical Updates Hematology-Oncology news Specialties

Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results

By MedXY 08/01/2025

A single AAV gene therapy infusion for severe hemophilia B led to sustained factor IX expression, substantial reduction in bleeding and factor use, and no late safety concerns over 13 years.