BioMarin Pharmaceuticals released positive results from its global 3-year phase 3 study of valoctocogene roxaparvovec (Roctavian™) for patients with hemophilia A. With 134 participants, GENEr8-1 is the largest phase 3 study on gene therapy for hemophilia to date. “We continue to learn more about the durability, safety and efficacy of valoctocogene roxaparvovec,” said Steven Pipe,…
Pfizer has announced positive results from its phase 3 BENAGENE-2 study assessing the noninferiority and superiority of fidanacogene elaparvovec gene therapy for moderately severe to severe hemophilia B. The results, announced via news release, showed the treatment met the primary superiority endpoint in terms of annualized bleeding rate (ABR) of total bleeds after infusion of…
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Philip J. Brooks, PhD, acting director of the Division of Rare Diseases Research Innovation at the National Institutes of Health’s National Center for Advancing Translational Sciences.
Lentiviral vector-mediated hematopoietic stem and progenitor cell (HSPC) gene therapy may lead to increased acid alpha-glucosidase (GAA) levels in animal models, demonstrating its therapeutic potential in Pompe disease (PD), according to a recently published study in Methods and Clinical Development. Currently, enzyme replacement therapy (ERT) constitutes the standard of care for patients with PD, which has…
Gene therapy represents the next horizon in the therapeutic landscape of the subtypes of Pompe disease.
Researchers reported that patients with hemophilia B who received a single administration of etranacogene dezaparvovec would likely have enough factor IX (FIX) levels to remain free from the need for prophylactic FIX replacement products for more than 2 decades, according to a study published in Current Medical Research and Opinion. “Gene therapy offers the potential…
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Deanna Tucker, PharmD, a medical science liaison team leader at Sarepta Therapeutics, on the company’s efforts to develop a gene therapy for Duchenne muscular dystrophy. Read the full story here: Sarepta Simultaneously Pursues Exon Skipping, PPMO, and Gene Therapies for DMD
Spinal muscular atrophy (SMA) is inherited in an autosomal recessive manner and is caused by abnormalities in the survival motor neuron 1 (SMN1) gene. The overwhelming majority of patients with SMA have a homozygous deletion of the gene; only 5% of cases have a heterozygous deletion and a point mutation. “The SMN protein is critical…
Michael Kelly, MD, chief scientific officer at CureDuchenne, says a gene therapy for DMD could win FDA approval as early as 2023.
