Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating ...
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REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss (Revised)
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study.
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...
Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in ...
LONDON, GREATER LONDON, UNITED KINGDOM, October 15, 2025 /EINPresswire.com/ -- "Get 20% Off All Global Market Reports With Code ONLINE20 – Stay Ahead Of Trade Shifts, Macroeconomic Trends, And ...
FDA accepts Rocket Pharmaceuticals' resubmission for Kresladi gene therapy in severe LAD-I, with a decision expected by March ...
In a small pivotal trial of an investigational gene therapy, most cases of congenital profound deafness from genetic variants ...
Affinia Therapeutics (“Affinia”), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies for devastating cardiovascular ...
In a note to investors on Sunday, analysts at Leerink Partners called these findings “compelling,” adding that they indicate ...
Abeona Therapeutics Inc. (Nasdaq: ABEO), a commercial-stage biopharmaceutical company developing cell and gene therapies for ...
The dose-escalation, placebo-controlled, randomised, double-blind LION-CS101 study is designed to assess the safety of ...
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