Precision gene editing could prevent vision loss from Stargardt disease
Researchers have developed a therapy to treat Stargardt disease, the most common form of inherited macular degeneration, ...
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Breakthrough Gene Editing Technique Offers Hope for Stargardt Disease Patients
Rewriting genes to preserve eyesight IOB and Beam Therapeutics develop efficient gene editing technique for Stargardt disease ...
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FDA clears investigational new drug application for Stargardt disease gene therapy
Please provide your email address to receive an email when new articles are posted on . A phase 1/2 trial will assess safety, tolerability and preliminary efficacy for VG801. The FDA also selected ...
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Alnylam scraps trial in rare eye disorder, blaming drug price reforms
Alnylam has said it will not proceed with a clinical trial of its RNAi drug vutrisiran in the rare eye disorder Stargardt disease – at least for now – tying its decision to President Joe Biden ...
GEN5d
Base Editing Addresses Inherited Macular Degeneration in Primate, Human Tissue Models
The adenine base editor corrected the most common mutation associated with Stargardt disease in the ABCA4 gene with no off-target effects.
Alkeus Pharmaceuticals to Present at the 43rd Annual J.P. Morgan Healthcare Conference
Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today ...
Institute of Molecular and Clinical Ophthalmology Basel (IOB): Breakthrough Gene Editing Technique Offers Hope for Stargardt Disease Patients
Researchers have developed a highly efficient gene editing therapy that could potentially treat Stargardt disease, the most common form of inherited macular degeneration. This groundbreaking study, pu ...
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Sisters to highlight rare eye condition at BT science exhibition
Caitlin and Caoimhe Mynes stole the hearts of viewers when they had the craic with Ryan Tubridy making lava lamps and glitter ...