Category GENE THERAPY RARE DISEASES

Single-Dose Gene Therapy is Potentially Life-Changing for Adults with Hemophilia B

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

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New Gene Therapy Trial Shows Restored Hearing and Speech in Children Born Deaf, Treated in Both Ears

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

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Regeneron Presents Update on Gene Therapy for Genetic Deafness at ASGCT

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

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Fred Hutch scientist Dr. Adair partners with colleagues around the globe to make gene therapies more effective and more widely available

Fred Hutch Cancer Center scientist Jennifer E. Adair, PhD, is on a mission to foster worldwide collaboration on potentially curative gene therapies. Holder of the Fleischauer Family Endowed Chair in Gene Therapy Translation, Adair just co-authored two articles published today in Science Translational Medicine as part of a special series on global access to these therapies that she

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Gene Therapy Treatment Increasing Body’s Signal for New Blood Vessel Growth Shows Promise

EXACT Trial Demonstrated Improvements in Exercise Duration, Ischemia, and Decreased Symptoms for Patients with Advanced CAD   Final 12-month data from the EXACT trial demonstrates safety and efficacy results for a vascular endothelial growth factor (VEGF) gene therapy treatment for patients who have advanced coronary artery disease (CAD). The late-breaking results were presented today at

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FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

The U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).  Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system. It is caused by a

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Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at the Association for Research in Otolaryngology 2024 MidWinter Meeting

Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant, the first to receive gene therapy in the United States for a genetic form of hearing loss AK-OTOF is a gene therapy being developed for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF)

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FDA Approves First CRISPR-based Gene Therapies to Treat Patients with Sickle Cell Disease

The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an

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