Category GENE THERAPY RARE DISEASES

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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PIER MARIA FORNASARI
2024-09-26

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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PIER MARIA FORNASARI
2024-06-07

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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PIER MARIA FORNASARI
2024-05-11

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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PIER MARIA FORNASARI
2024-05-11

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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PIER MARIA FORNASARI
2024-05-07

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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PIER MARIA FORNASARI
2024-03-19

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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PIER MARIA FORNASARI
2024-01-24

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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