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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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The Foundation Fighting Blindness and Dr. H. James & Carole Free Collaborate to Combat AMD

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The Foundation Fighting Blindness has announced the launch of The Free Family AMD Research Program, which is providing funding for 10 research projects over five years for the development of age-related macular degeneration (AMD) therapies. With an anchor investment from Dr. H. James & Carole Free, along with legacy gift matching funds from the estate of James Lea, this new program will begin in July 2019.

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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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The Foundation Receives a $100,000 Research Grant from Sofia Sees Hope

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Sofia Sees Hope, a nonprofit dedicated to finding treatments and cures for people with Leber congenital amaurosis (LCA) and other inherited retinal diseases (IRDs), has made a $100,000 donation to the Foundation Fighting Blindness to support therapy development and genetic testing.
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Pixium’s PRIMA Bionic Vision System Restores Central Vision in Dry AMD Clinical Trial

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The French bioelectronics company Pixium Vision has reported that its PRIMA bionic vision system has restored some central vision in patients with advanced dry age-related macular degeneration (AMD) participating in a clinical feasibility trial. Led by Dr. Yannick Le Mer, the study took place in the Department of Pr. José Sahel, Hopital des Quinze Vingts and Fondation Ophtalmologique A. De Rothschild, in Paris, France.
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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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FDA Authorizes Clinical Trial for CRISPR/Cas9 Therapy for LCA 10

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Editas Medicine, a company developing gene-editing treatments, has received authorization from the US Food and Drug Administration to launch a clinical trial for its emerging CRISPR/Cas9 therapy for people with a mutation in the gene CEP290, which causes Leber congenital amaurosis 10 (LCA10). LCA causes severe vision loss or blindness at birth.
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ProQR Receives FDA Authorization to Launch Clinical Trial for USH2A Therapy

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ProQR, a biotech in the Netherlands developing therapies for rare diseases, has received authorization from the US Food and Drug Administration to launch a Phase 1/2 clinical trial for QR-421a, its treatment targeting mutations in exon 13 of the USH2A gene. The mutations cause Usher syndrome type 2A (combined vision and hearing loss) and non-syndromic retinitis pigmentosa (vision loss only) in approximately 16,000 people in the Western World. ProQR plans to begin enrolling patients in the QR-421a Phase 1/2 trial named STELLAR in the coming months with preliminary data expected in mid-2019.
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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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Michael Kalberer: Determined to Empower Others and Bring Hope

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Long Island native Michael Kalberer has a knack for connecting with everyone around him, despite being born with cerebral palsy (CP), a disorder that affects a person’s ability to move and maintain balance. The condition can also cause vision loss. At the age of 12, Michael noticed issues with his depth perception and visual field, which resulted in a diagnosis that he was legally blind. At 33 years old, he was finally diagnosed, specifically, with Leber congenital amaurosis (LCA).
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