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Disease
Often diagnosed in childhood or adolescence, retinitis pigmentosa (RP) is an inherited retinal disease causing progressive loss of night and peripheral vision. The condition often leads to legal and sometimes complete blindness.
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A Retinal Research Nonprofit Paves the Way for Commercializing Gene Therapies
In the PressAN EMERGING, vision-restoring gene therapy for a devastating retinal disease is poised for Food and Drug Administration (FDA) approval. If it gets the regulatory nod, it will be the first gene therapy to receive FDA approval for the eye or an inherited condition.
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jCyte Reports Results for Phase 1/2a Clinical Trial for Retinal-Cell Treatment
Research NewsSome participants reported increased light sensitivity, improved color vision, better mobility, and improved reading ability.
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History Is Made: FDA Approves Spark's Vision-Restoring Gene Therapy
Research NewsKnown as LUXTURNA™ (voretigene neparvovec), the gene therapy restored vision in a clinical trial for people between the ages of 4 and 44 with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65.
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Beacon Stories
“I try not to get my hopes up too much, but I never lose hope; I’m determined. I just want to see my son’s face, see him get married, & see my grandchildren. I encourage everyone with a retinal disease to get a genetic test & to never ever give up hope.“
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Stem-Cell Therapy Clinics Remain Inadequately Regulated, Pose Risk to Patients
Research NewsIf a clinic is charging for a stem-cell treatment or procedure for an IRD, it is probably not legit. The expense to the patient is a major red flag.
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ProQR Doses First Participant in Its LCA10 Therapy Clinical Trial
Research NewsThe company plans to report interim, six-month study results in 2018 and 12-month results in 2019.
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Research News
An advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval.
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Research News
Investigational LUXTURNA has the potential to be both the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease in the United States
First adeno-associated viral (AAV) vector investigational candidate to reach this stage of FDA review
Investigational LUXTURNA clinical program includes up to four years of efficacy data, with observation ongoing
