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Posts tagged clinical trial

First Patient Receives ProQR’s Emerging USH2A Therapy in Clinical Trial

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ProQR, a developer of RNA therapies in the Netherlands, announced that the first clinical-trial participant has received its emerging treatment, which targets retinitis pigmentosa and Usher syndrome caused by mutations in exon 13 of the USH2A gene. The Phase 1/2 clinical trial is taking place at Retina Foundation of the Southwest in Dallas and the University of Michigan in Ann Arbor. Known as QR-421a, the treatment is intended to slow or potentially reverse vision loss.
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Encouraging Vision Improvements Reported in ReNeuron’s Cell-Therapy Clinical Trial

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ReNeuron, a cellular therapy developer in the UK, has reported vision improvements in the treated eyes of the first three retinitis pigmentosa (RP) patients in the Phase II part of the Phase I/II clinical trial for its proprietary human retinal progenitor cells (hRPC). The Phase I portion of the trial, completed last year, primarily assessed safety in subjects with minimal remaining vision.
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AGTC Announces Topline Interim Six-Month Data of XLRS Gene Therapy from Ongoing Phase 1/2 Clinical Trial

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Applied Genetic Technologies Corporation (AGTC), a leading developer of gene therapies, has reported the six-month follow-up data in all patients treated with its gene therapy for X-linked retinoschisis (XLRS) in its Phase 1/2 clinical trial. The company reported that no consistently significant clinical activity from the treatment was observed after six months. AGTC will continue to analyze all of the XLRS trial data and complete all visits specified in the clinical protocol. However, if the data at additional follow-up data visits are consistent with the current six-month findings, the company has stated that they will not further develop the product.
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Acucela Enrolling Patients in Phase 3 Trial for Stargardt Disease Treatment

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The Seattle biotech Acucela is now enrolling participants in its Phase 3 clinical trial for emixustat hydrochloride, an emerging oral drug for slowing vision loss in people with Stargardt disease, an inherited form of a macular degeneration. The progressive retinal condition affects 30,000 people in the US.
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Apellis Launches Phase 3 Clinical Trial Program for Advanced Dry AMD Treatment

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The biopharmaceutical company Apellis has treated the first patient in its Phase 3 clinical trial program for APL-2, a compound designed to slow the progression of advanced dry age-related macular degeneration (AMD) also known as geographic atrophy (GA).
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Vision Improvements Reported in ProQR’s Clinical Trial for LCA10 Treatment

ProQR, a biotech company in the Netherlands, has reported vision improvements for patients 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 company reported that 60 percent of subjects in the trial demonstrated improvements in visual acuity and their ability to navigate a mobility course. The treatment was also safe for patients.
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French Gene Therapy Company Advancing Three Programs for Retinal Diseases

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Horama, a French biotech developing gene therapies for rare eye diseases, was established in 2014 as a spin-off of INSERM, France’s public scientific and technology institute. Today, the company has three gene-therapy development programs underway for rare inherited retinal diseases, targeting people with mutations in the genes PDE6B, RPE65, and RLBP1.
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Best Disease Gene Therapy Advances Toward Clinical Trial

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BEST1-Untreated cBest patho MCT1-E66

Untreated retina

BEST1-treated MCT1-E66

Retina treated with gene therapy

Using gene therapy, FFB-funded researchers at the University of Pennsylvania School of Veterinary Medicine (Penn Vet) and Perelman School of Medicine have reversed the disease process in a canine model of Best disease, an inherited form of macular degeneration that can lead to severe vision loss in humans. The therapeutic effect of the treatment has been sustained for as long as five years. Results of the study led by Karina Guziewicz, PhD, and Artur Cideciyan, PhD, were published online in the journal Proceedings of the National Academy of Sciences (PNAS).
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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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