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WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has recommended lifting the voluntary clinical hold on Elevidys for ambulatory patients with Duchenne Muscular Dystrophy (DMD), following a thorough …
FDA Clears Path for Resumed Use of Elevidys Gene Therapy in Ambulatory DMD Patients Read More
BERWYN, PA — Specialty pharmacy and healthcare services firm AscellaHealth is spotlighting its role as the HUB partner for Abeona Therapeutics (Nasdaq: ABEO) in the launch and commercialization of ZEVASKYN™, …
AscellaHealth Drives Patient-Centric Launch Support for Groundbreaking Gene Therapy ZEVASKYN Read More WASHINGTON, D.C. — Federal health regulators are investigating the death of an 8-year-old boy who received Elevidys, a gene therapy developed by Sarepta Therapeutics for Duchenne muscular dystrophy, a rare …
FDA Probes Death of Child Linked to Gene Therapy for Duchenne Muscular Dystrophy Read More
WASHINGTON, D.C. — The U.S. Food and Drug Administration on Friday placed Sarepta Therapeutics’ gene therapy trials for limb girdle muscular dystrophy on clinical hold following the deaths of three patients …
FDA Shuts Down Sarepta Gene Therapy Trials After Three Deaths Linked to Liver Failure Read More
WASHINGTON, D.C. — The Centers for Medicare & Medicaid Services (CMS) has unveiled a groundbreaking initiative aimed at expanding access to potentially curative gene therapies for individuals with sickle cell …
Medicaid to Expand Access to Life-Changing Gene Therapy for Sickle Cell Patients in 33 States Read More
MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced that the first patient has been dosed in its Phase 2/3 GARDian3 clinical trial evaluating OCU410ST, a gene therapy candidate designed …
Ocugen Doses First Patient in Pivotal Trial for Stargardt Gene Therapy Read More
KING OF PRUSSIA, PA — XyloCor Therapeutics has dosed the first patient in its Phase 2b EXACT-2 trial evaluating XC001 (encoberminogene rezmadenovec), a novel gene therapy designed to help patients …
XyloCor Doses First Patient in Pivotal Gene Therapy Trial for Refractory Angina Read More BERWYN, PA — AscellaHealth has released its Q2 2025 Specialty & Rare Pipeline Digest™, offering stakeholders a detailed look at the rapidly evolving landscape of specialty and rare disease therapies. The …
AscellaHealth’s Latest Digest Tracks Surge in Specialty and Rare Disease Drug Approvals Read More
PHILADELPHIA, PA — Passage Bio, Inc. (NASDAQ: PASG) has announced encouraging updates from the ongoing Phase 1/2 upliFT-D clinical trial for PBFT02, a one-time gene therapy for frontotemporal dementia (FTD) …
Passage Bio Reports Progress in Gene Therapy for Frontotemporal Dementia Read More
PHILADELPHIA, PA — Latus Bio, Inc. has announced a significant advancement in the field of gene therapy with the publication of its latest research in Nature Communications. The study, led …
Latus Bio Unveils Breakthrough in Gene Therapy for Neurodegenerative Diseases Read More