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
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
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
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
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),
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
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.
Study results formed the basis of the therapy’s FDA approval in April 2024 Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine by researchers from the University of
Administering gene therapy in both ears of five children with DFNB9 led to additional benefits compared to previous trial, including sound source localization and hearing in noisy environments. Key Takeaways A novel gene therapy designed to target a form of inherited deafness restored hearing function in five children who were treated in both ears. The
Regeneron presented results from an ongoing Phase I/II clinical trial for its investigational gene therapy, DB-OTO, at the annual American Society of Gene and Cell Therapy (ASGCT) meeting, being held in Baltimore, Md. from May 7–11. DB-OTO, a gene therapy for genetic deafness, improved hearing in one child, treated at 11 months old to normal levels within
