Category GENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2026-04-24

FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss Under National Priority Voucher Program

Groundbreaking AAV-based gene therapy offers potential treatment for patients with OTOF gene-associated severe-to-profound and profound hearing loss The U.S. Food and Drug Administration approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy. Otarmeni is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any

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No Comments InGENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2026-04-11

Phase I trial published in Nature: China’s novel base-editing therapy brings hope of cure for thalassemia patients

In a breakthrough for gene therapy, the international academic journal Nature has published a landmark clinical study detailing the successful use of a novel base-editing drug to treat β-thalassemia. Jointly conducted by ShanghaiTech University, the First Affiliated Hospital of Guangxi Medical University, Fudan University, and CorrectSequence Therapeutics, the early-stage clinical trial of the base-editing drug CS-101 injection reports that

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No Comments InGENE EDITING GENE THERAPY RARE DISEASES RESEARCH
PIER MARIA FORNASARI
2026-02-14

Cell and gene therapy across 35 years

Cell and gene therapies, or CGT, have come a long way since they were first introduced. In the last few decades, both cell therapy — the transplantation of living cells — and gene therapy — the use of genetic material to modify cell functions — have been increasingly incorporated into clinical practice. Various challenges and advances

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No Comments InADVANCED THERAPY MEDICINAL PRODUCT CELL THERAPY GENE THERAPY RARE DISEASES REGENERATIVE MEDICINE
PIER MARIA FORNASARI
2025-12-10

FDA and EMA Approve First Gene Therapy Treatment for Wiskott-Aldrich Syndrome to Fondazione Telethon ETS

Agency exercises regulatory flexibility to address unmet need for rare, life-threatening disease The U.S. Food and Drug Administration approved Waskyra (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS). Waskyra is indicated for pediatric patients six months and older and adults with WAS who have a mutation in the WAS gene and

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PIER MARIA FORNASARI
2025-12-07

Gene Therapy with exagamglogene autotemcel (exa-cel) Leads to Improved Quality of Life in Patients with Sickle Cell Disease and Beta Thalassemia

Substantial improvements seen across ages in overall health and wellbeing Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia, according to two studies published in Blood Advances. “This is the first time we’ve been able to measure improvements in quality of

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No Comments InCRISPR GENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2025-11-26

FDA Approves Gene Therapy for Treatment of Spinal Muscular Atrophy

The U.S. Food and Drug Administration approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in the survival motor neuron 1 (SMN1) gene. Itvisma is an adeno-associated virus (AAV) vector-based gene therapy. “Today’s approval shows the power of

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PIER MARIA FORNASARI
2025-05-23

American Academy Neurology issues Evidence in Focus article on Duchenne muscular dystrophy gene therapy

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article, published May 14, 2025, online in Neurology®. AAN Evidence in Focus articles highlight the strength of the current evidence for

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PIER MARIA FORNASARI
2025-04-12

Innovative gene therapy developed to treat a rare and devastating genetic heart disease in young men

A CNIC team has designed a gene therapy-based treatment that could transform the approach to arrhythmogenic cardiomyopathy type 5 (ARVC5), a rare and fatal genetic disease that particularly affects young men. A team from the  National Center for Cardiovascular Research (CNIC) has developed an innovative gene therapy that could transform the treatment of arrhythmogenic cardiomyopathy type 5 (ARVC5),

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PIER MARIA FORNASARI
2025-02-26

Gene therapy in children with AIPL1-associated severe retinal dystrophy can improve vision

Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with AIPL1-associated severe retinal dystrophy. This is the first human trial of gene supplementation therapy targeting this condition. Retinal dystrophy caused by biallelic variants in the AIPL1 gene

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PIER MARIA FORNASARI
2024-12-08

An Universal Gene Therapy for Diamond-Blackfan Anemia Is Poised for Clinical Trials

Treatment designed to work across different genetic mutations At a glance: Efforts to develop a gene therapy for Diamond-Blackfan anemia (DBA) — a rare, life-threatening disorder in which bone marrow cannot make mature, functioning red blood cells — have been hampered by the fact that at least 30 different genetic mutations can cause the disorder.

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