Listen to this page using ReadSpeaker
Archive for the Diseases Category

Study Suggests Vitamin A May Benefit Children with RP

Audio version:

An FFB-funded study at Massachusetts Eye and Ear Infirmary (MEEI) suggests that vitamin A palmitate supplementation may slow the decline of cone function by nearly 50 percent in children with retinitis pigmentosa (RP). Cones are the photoreceptors that normally provide daytime vision, and the ability to read, recognize faces, and perceive colors. Cone function was evaluated in the study by a full-field electroretinogram or ERG, which measures the retina’s electrical response to light.
Continue Reading…

Vision Improvements Reported in Early Stem Cell Trial for Wet AMD

Audio version:

Two patients with advanced wet age-related macular degeneration (AMD) in a Phase I clinical trial demonstrated improved visual acuity sustained for one year after a sheet of retinal pigment epithelial (RPE) cells derived from embryonic stem cells was transplanted under their retinas. Each patient had one eye treated. Vision improvement for one patient was 29 letters or about 6 lines on an eye chart. The other had a gain of 21 letters or about 4 lines.
Continue Reading…

Choroideremia Gene Therapy Moves into Phase 3 Human Study

Audio version:

Nightstar Therapeutics, a retinal-disease, gene-therapy development company in the UK, is advancing its emerging gene therapy for choroideremia into a Phase 3 clinical trial known as STAR. The study will enroll approximately 140 patients at 18 clinical sites in the US, Europe, Canada, and South America.
Continue Reading…

Natural History Study Launches for LCA Caused by Specific Mutation in CEP290

Massachusetts Eye and Ear (MEE) is participating in a natural history study for people with Leber congenital amaurosis (LCA) type 10 caused by a mutation referred to as “c.2991+1655A>G” in intron 26 of the CEP290 gene.
Continue Reading…

FFB-CRI Investing $7.5 Million in Emerging Therapy for USH2A

Audio version:

The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has entered into a partnership with ProQR to develop a retinal therapy for people with Usher syndrome type 2A (USH2A) caused by mutations in exon 13 of the USH2A gene. FFB-CRI will be investing up to $7.5 million in milestone-based funding to advance the treatment, known as QR-421a, toward a Phase 1/2 clinical trial during 2018. ProQR plans to issue the initial data report for the clinical study in 2019.
Continue Reading…

AGTC Launches XLRP Gene Therapy Clinical Trial at Five Sites in U.S.

Audio version:
Applied Genetic Technologies Corporation (AGTC) is now recruiting for its Phase 1/2 gene therapy clinical trial for males with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. Approximately 15 patients will be enrolled in the study, which is primarily evaluating safety. Three doses of the gene therapy will be tested. The trial is taking place at five sites in the U.S.
Continue Reading…

Ophthotech Launching Human Study of Emerging Therapy for Stargardt Disease

Audio Version:

Ophthotech, a biopharmaceutical company developing therapies for eye diseases, has enrolled the first patient in its Phase 2b clinical trial of Zimura® for people with Stargardt disease caused by mutations in the gene ABCA4. The 120-participant study will be taking place at more than 30 sites. Data and knowledge gleaned from ProgStar, a natural history study for people with Stargardt disease funded by the Foundation Fighting Blindness, was used in the design of the clinical trial.
Continue Reading…

Top Retinal Research Advances for 2017

To view and listen to Ben Shaberman’s presentation of “Top Retinal Research Advances for 2017,” with full slides and audio, click here. The text to the presentation is as follows:

This is Ben Shaberman, director of science communications, at the Foundation Fighting Blindness (FFB), and I’m pleased to present a quick overview of some of the exciting research advances for inherited retinal diseases made during 2017. It has been an exciting year with several promising therapies moving into and through clinical trials.
Continue Reading…

jCyte Reports Results for Phase 1/2a Clinical Trial for Retinal-Cell Treatment

jcyteA retinal-cell treatment for people with retinitis pigmentosa (RP) has performed encouragingly in a Phase 1/2a clinical trial. Developed by jCyte, the treatment was evaluated for 12 months in 28 people at two sites in Southern California.

Side effects were minor in the safety-oriented trial. Those receiving the highest dose of the treatment had the best results. Their visual acuity, as measured using an eye chart, was nearly two lines (nine letters) better in their treated eye than in their untreated eye. Some participants reported increased light sensitivity, improved color vision, better mobility, and improved reading ability. Ultimately, 22 of the 28 participants had their second eye treated. Continue Reading…

History Is Made: FDA Approves Spark’s Vision-Restoring Gene Therapy

Spark LogoSpark Therapeutics’ vision-restoring RPE65 gene therapy has received marketing approval from the U.S. Food and Drug Administration, becoming the first gene therapy to gain regulatory approval in the U.S. for the eye or any inherited condition.

Known 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. Study participants with severe vision loss reported putting away their navigational canes, seeing stars, being able to read, and recognizing faces of loved ones. Vision restoration has persisted for at least three years. The treatment is also designed to work for people with retinitis pigmentosa (RP) caused by RPE65 mutations. Continue Reading…