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Archive for the General Category

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

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

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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.
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Ophthotech Launching Human Study of Emerging Therapy for Stargardt Disease

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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.
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Clinical Trial to Launch for System Combining Optogenetics and Eyewear

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gensightThe French biotech GenSight Biologics has received regulatory authorization in the UK to launch the PIONEER Phase 1/2 clinical trial for its GS030 system — a light-sensing gene therapy (optogenetics) coupled with eyewear, which enhances visual stimulation. The system is designed to restore vision for people who are blind from retinitis pigmentosa (RP) and potentially other retinal conditions such as: Usher syndrome, Stargardt disease, and dry age-related macular degeneration.

The GS030 will be evaluated in 18 people with RP who can see no better than counting fingers. Additional trial details are available at the clinical trials Web site hosted by the National Institutes of Health.
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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.
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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…

Stem-Cell Therapy Clinics Remain Inadequately Regulated, Pose Risk to Patients

Though the U.S. Food & Drug Administration (FDA) is more tightly regulating U.S. clinics that tout stem-cell trials and treatments for inherited retinal diseases (IRDs) and a host of other conditions, many significant loopholes and enforcement limitations remain.

The bottom line is: The FDA has not yet established regulations to adequately protect patients with IRDs seeking treatments from these clinics. If 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

ProQR, a biotech company in the Netherlands, has treated its first patient in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.

The Phase 1/2 trial will enroll six children and six adults who have the mutation. Participants will receive a dose in one eye every three months for a year. The treatment is delivered through an injection into the vitreous, the gel-like substance in the middle of the eye.
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FDA Committee Unanimously Recommends Approval for Spark’s RPE65 Gene Therapy – Final Decision Due in January 2018

Ashley and Cole Carper traveled from Little Rock, AR, to tell their family’s story at the FDA hearing.

Ashley and Cole Carper traveled from Little Rock, AR, to tell their family’s story at the FDA hearing.

Spark Therapeutics has taken a major step closer to gaining marketing approval for its vision-restoring gene therapy for people with RPE65 mutations causing Leber congenital amaurosis (LCA) and retinitis pigmentosa. At the conclusion of a public hearing on October 12, 2017, an advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval. The FDA is due to make a final decision on marketing approval for the treatment, known as voretigene neparvovec, by January 12, 2018.

The event held at FDA headquarters included the presentation of trial results from Spark representatives, as well as compelling testimony from patients, family members, and industry stakeholders.

Twenty-four-year-old Katelyn Corey told hearing attendees that before receiving the treatment, her constant adaptation to dwindling vision didn’t leave time for much else in her life. But her circumstances changed dramatically in December 2013, after she received the RPE65 gene therapy in Spark’s Phase III clinical trial.
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