
The therapy has demonstrated benefit in multiple models of dry AMD.
The therapy has demonstrated benefit in multiple models of dry AMD.
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Researchers used novel functional MRI approaches to better measure the effects of gene therapy in children in current clinical trials.
While focused on the retina, the researchers showed the platform works for the identification of AAVs that target tissues in various organs.
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Matthew J. Frigault, MD, discussed safety and efficacy findings on CART-ddBCMA.
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The move comes after the EMA recommended an additional clinical trial of sepofarsen before applying for marketing authorization.
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Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Data is expected in the first quarter of 2023 for both Stargardt disease and retinitis pigmentosa.
The long-term RESTORE study included data from 55 participants in previous phase 3 trials.
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After submitting an amended IND, Adverum’s gene therapy is set to be evaluated in the phase 2 LUNA trial.
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Foundation Fighting Blindness is helping to support the development of gene therapies for retinal diseases.
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Review top news and interview highlights from the week ending July 1, 2022.
ADVM-022 was also granted PRIME designation by the EMA on June 24, 2022.