Congressional Briefing Highlights Impact of Research Funding on Rare Eye Diseases: Retinitis Pigmentosa and Stargardt Disease
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Congressional Briefing Highlights Impact of Research Funding on Rare Eye Diseases: Retinitis Pigmentosa and Stargardt Disease

Written by Judith Hill, Communications and Events Director, National Alliance for Eye and Vision Research In a remarkable show of unity and commitment to eye health, the Alliance for Eye…

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Two Partners Come Together to Manufacture Gene Therapy for RPGRIP1-Related Vision Disorders
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Two Partners Come Together to Manufacture Gene Therapy for RPGRIP1-Related Vision Disorders

According to a story from World Pharma Today, Andelyn Biosciences, Inc., which is a gene therapy Contract Development and Manufacturing Organization (CDMO), has recently struck a partnership with Odylia Therapeutics,…

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The Discovery of 261 New Gene Mutations Which Could Cause Blindness May Lead to More Accurate Diagnoses and New Treatments
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The Discovery of 261 New Gene Mutations Which Could Cause Blindness May Lead to More Accurate Diagnoses and New Treatments

Ala Moshiri from the University of California is both a researcher and an eye doctor. He recently discussed his frustration surrounding the lack of available treatments for so many genetic…

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InformedDNA, Foundation Fighting Blindness Partnership Will Screen Patients for Usher Syndrome, RP Clinical Trials
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InformedDNA, Foundation Fighting Blindness Partnership Will Screen Patients for Usher Syndrome, RP Clinical Trials

ProQR Therapeutics is working to develop unique therapeutic solutions for genetic eye diseases. Currently, the company is evaluating its therapeutic candidate ultevursen within the Phase 2/3 Sirius and Celeste clinical trials; you can read…

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INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 2)
Daniel de Boer. Photo courtesy of: ProQR and FTI Consulting

INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 2)

If you would like to read the first part of this interview, please take a look at Part 1 here. In the first part of the interview, I spoke with…

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INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 1)
Daniel de Boer. Photo courtesy of: ProQR and FTI Consulting

INTERVIEW: How ProQR’s Sirius and Celeste Studies Could Change the Therapeutic Landscape for USH2A-Mediated RP and Usher Syndrome (Pt. 1)

Clinical trials, drug development, and medical research have the potential to change the therapeutic landscape for patients with a wide variety of conditions. At ProQR, founder and CEO Daniel de…

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Results Available from GS030 Study for RP in NHP
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Results Available from GS030 Study for RP in NHP

Recently, biopharmaceutical company GenSight Biologics ("GenSight") announced the publication of data from a study evaluating GS030-Drug Products (GS030-DP or "GS030") as a potential treatment option for retinitis pigmentosa (RP). Although…

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New Investigative Therapy Holds Promise for Retinitis Pigmentosa in Mouse Model

Researchers from Nanoscope, thanks to funding by the NIH, have developed a protein that can effectively restore vision in mice. This study was published in Nature Gene Therapy. The Study…

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X-Linked Retinitis Pigmentosa Investigative Gene Therapy is Looking Promising
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X-Linked Retinitis Pigmentosa Investigative Gene Therapy is Looking Promising

Janssen Pharmaceuticals has an ongoing Phase 1/2 clinical trial for the inherited retinal disease X-linked retinitis pigmentosa (XLRP). They are investigating an adeno-associated gene therapy called RPGR as a therapeutic…

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