For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

The five-year trial saw patients who received a single injection of Lumevoq sustaining clinical recovery in a rare condition ...

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Currently, patients are treated with injections of asfotase alfa ... "We believe the next evolution in treating HPP will be a gene therapy in which a single injected dose will provide a lifelong ...

A one-time injection of Lumevoq demonstrated sustained visual acuity improvements with a favorable safety profile at 5 years ...

This article examines viral and non-viral vectors in gene therapy, highlighting their mechanisms, advantages, and limitations ...

Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a ...

GenSight Biologics announces efficacy and safety results for Lumevoq gene therapy at the conclusion of the REFLECT phase III clinical study: Paris Thursday, February 13, 2025, 17: ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated ...

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including the additional benefit observed in participants receiving a bilateral intravitreal injection of the gene therapy," said Prof. Patrick Yu-Wai-Man, MD, PhD, Professor of Ophthalmology and ...