Spinal Delivery of Gene Therapy Promising for Neurodegenerative Disease
Gene therapy delivered for the first time via lumbar puncture showed promise of benefit in slowing progression of giant axonal neuropathy (GAN) in a phase I clinical trial. The slope of change in motor function went from -7.17 percentage points per year before treatment to -0.54 percentage points with the lowest dose to an improvement […]
Pediatric Neuron Disease Gets Its First Gene Therapy
The FDA approved atidarsagene autotemcel (arsa-cel; Lenmeldy), the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD), the agency announced on Monday. “This is the first FDA-approved treatment option for children who have this rare genetic disease,” Peter Marks, MD, PhD, director of […]
FDA Gives Nod to Gene Therapy for Hemophilia A
The FDA approved valoctocogene roxaparvovec (Roctavian) as the first gene therapy for the treatment of adults with severe hemophilia A, drug developer Biomarin announced on Thursday. Patients with severe disease are eligible for the one-time, single-dose treatment if they don’t have antibodies to adeno-associated virus serotype 5 (AAV5), as detected by an FDA-approved test. FDA […]
Rare Skin Blistering Condition Gets First Drug Approved
The FDA granted the first drug approval for dystrophic epidermolysis bullosa (DEB), a topical gene therapy that treats the blistering wounds it causes, the agency announced Friday. The herpes-simplex virus type 1 vector-based gene therapy, Vyjuvek, was approved for patients ages 6 months and older with mutation in the COL7A1 (collagen type VII alpha 1 […]
Duchenne Muscular Dystrophy Treatment Narrowly Captures Support of FDA Advisors
FDA advisors narrowly supported accelerated approval for Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy (DMD) on Friday. In a 8-to-6 vote, the agency’s Cellular, Tissue, and Gene Therapies Advisory Committee said the overall benefits and risks supported SRP-9001 (delandistrogene moxeparvovec) for ambulatory Duchenne patients, using expression of Sarepta’s micro-dystrophin as a surrogate endpoint. […]
FDA Approves First Gene Therapy for Non-Muscle-Invasive Bladder Cancer
The FDA has approved nadofaragene firadenovec (Adstiladrin) as the first gene therapy for the treatment of high-risk bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC), the agency announced on Friday. An adenoviral vector-based product, nadofaragene firadenovec, is specifically indicated for adult patients with carcinoma in situ (CIS) with or without papillary tumors. Approval was based on […]
Topical Gene Therapy Heals Blistering Skin Disease Lesions
A novel topical gene therapy healed most lesions among patients with dystrophic epidermolysis bullosa, a rare genetic blistering skin disease, the GEM-3 trial showed. Topical administration of beremagene geperpavec (B-VEC) led to complete healing of 67% of treated wounds as compared with 22% of those exposed to placebo at 6 months (P=0.002), reported M. Peter […]
FDA OKs First Gene Therapy for Hemophilia B
The FDA on Tuesday approved the first gene therapy for treating hemophilia B, a genetic bleeding disorder resulting from missing or insufficient levels of factor IX. Etranacogene dezaparvovec (Hemgenix) is indicated for adults with the condition who currently use factor IX prophylaxis therapy for blood clotting, those who have or have had life-threatening hemorrhage, or […]
Pattern of gene activity for ADHD
Researchers at the National Institutes of Health (NIH) have successfully identified differences in gene activity in the brains of people with attention deficit hyperactivity disorder (ADHD). The study, led by scientists at the National Human Genome Research Institute (NHGRI), part of the NIH, found that individuals diagnosed with ADHD had differences in genes that code […]
Gene mutation discovered that causes language impairment, ADHD and myasthenia
Two studies have revealed that certain disorders of the CAPRIN1 gene have significant consequences for people. First, the research team showed that insufficient production of the protein CAPRIN1 in the brain can lead to developmental differences, including autism spectrum disorders, attention deficit hyperactivity disorder (ADHD), and language disorders. Furthermore, the scientists identified a specific mutation […]