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Displaying 31–40 of 258 for “adeno-associated virus”
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Nov 7, 2019
AGTC Announces Development of Stargardt Disease Gene Therapy
Dual-vector delivery system designed to deliver the large ABCA4 gene
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May 6, 2019
ARVO 2019: Gene Therapy Clinical Trial for RP Caused by RLBP1 Mutations Ongoing in Sweden
In a Novartis lab study, mice with RLBP1 mutations had improved dark adaptation after receiving the gene therapy. The treatment remained effective after one year.
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Mar 8, 2018
Choroideremia Gene Therapy Moves into Phase 3 Human Study
Nightstar Therapeutics, a retinal-disease, gene-therapy development company in the UK, is advancing its emerging gene therapy for choroideremia into a Phase 3 clinical trial known as STAR. The study will enroll approximately 140 patients at 18 clinical sites in the US, Europe, Canada, and South America.
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Feb 7, 2018
AGTC Launches XLRP Gene Therapy Clinical Trial at Five Sites in U.S.
Taking place at five locations in the United States, the clinical trial will enroll approximately 15 males with X-linked retinitis pigmentosa caused by mutations in the gene RPGR and will primarily evaluate safety.
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Dec 20, 2017
History Is Made: FDA Approves Spark's Vision-Restoring Gene Therapy
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.
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May 23, 2017
Forty-Four High-Impact Retinal-Research Efforts Highlighted at FFB-Casey Innovation Summit
In its fourth year, the meeting is becoming the world’s most comprehensive overview of the promising research underway for emerging IRD treatments.
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Mar 20, 2017
First Patient Treated in XLRP Gene Therapy Clinical Trial
The Nightstar gene therapy involves injection of healthy copies of RPGR underneath the retina.
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May 13, 2015
ARVO 2015 Highlight: New Research Boosts Prospects for Saving Vision with RdCVF
After years of refinement and testing in animal models, the emerging therapy is about a year and a half from moving into a clinical trial.
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Full Transcript - 2019 Investing in Cures Summit - March 2, 2019
Full Session Transcripts of the 2019 Investing in Cures Summit.