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Posts tagged clinical trial

Apellis Launches Phase 3 Clinical Trial Program for Advanced Dry AMD Treatment

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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MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial

MeiraGTx, a gene-therapy company in London and New York City, has treated its first patient in a gene-therapy clinical trial for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. The Phase I/II study is taking place at Moorfields Eye Hospital in London. The safety-oriented trial will enroll 36 participants. Three dose levels of the therapy will be evaluated.

XLRP is a leading cause of inherited, progressive retinal degeneration and vision loss. The condition usually affects males, but is also diagnosed occasionally in females. Mutations in the gene RPGR cause about 70 percent of XLRP cases. RPGR mutations affect about 15,000 people in the United States and tens of thousands more around the world.
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Twelve People Receive XLRS Gene Therapy in AGTC’s Clinical Trial

Applied Genetics Technology Corporation (AGTC) reported that its gene therapy for X-linked retinoschisis (XLRS) has performed encouragingly in a Phase I/II, safety-oriented clinical trial taking place at seven sites in the U.S.

XLRS is an inherited disease that leads to significant vision loss due to splitting of the layers of the retina. The condition affects about 35,000 males in the U.S. and Europe. XLRS is caused by mutations in the gene retinoschisin. AGTC’s gene therapy uses a human-engineered virus — and adeno-associated virus or AAV — to deliver normal copies of retinoschisin to the patient’s retina.
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SparingVision Formed to Advance Sight-Saving Protein for RP

L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, and Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance

L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance

The development of a vision-saving treatment for people with retinitis pigmentosa (RP) is getting a major boost thanks to the formation of the French biotech SparingVision to move it into a clinical trial and out to the international marketplace.

A spin-off of the Institut de la Vision, SparingVision was established to clinically develop and commercialize a protein known as rod-derived cone-viability factor (RdCVF). The emerging therapy performed well in several previous lab studies funded by the Foundation Fighting Blindness. SparingVision’s goal is to launch a clinical trial for the protein in 2019.
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Valproic Acid’s Effect Too Small in One-Year Clinical Trial

However, researchers identify a potentially powerful endpoint for evaluating emerging therapies in future studies.

Results from a clinical trial sponsored by the Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) indicate that valproic acid, a drug approved by the U.S. Food & Drug Administration for seizure disorders, did not sufficiently preserve vision in people with autosomal dominant retinitis pigmentosa (adRP). FFB-CRI launched the 90-person study in 2010, because previous lab research, and a published clinical report involving a few patients, had suggested the drug might slow vision loss in people with adRP.
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