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FDA Authorizes Clinical Trial for CRISPR/Cas9 Therapy for LCA 10

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. Known as EDIT-101, the treatment will be evaluated in a Phase 1/2, dose-escalation clinical trial.  The company expects to enroll 10 to 20 patients. Further details and contact information for the study will be listed soon at www.clinicaltrials.gov . EDIT-101 targets a specific mutation, “c.2991+1655A>G” in intron 26, of the CEP290 gene. Editas has earned a $25 million milestone payment from Allergan as part of a drug-development alliance. “We are very pleased with this historic milestone in the treatment of inherited retinal diseases,” says Stephen Rose, PhD, chief scientific officer, Foundation Fighting Blindness. “EDIT-101 is the fi...
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Vision Improvements Reported in ProQR’s Clinical Trial for LCA10 Treatment

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ProQR, a biotech company in the Netherlands, ha s 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 su bjects in the trial demonstrated improvements in visual acuity and their ability to navigate a mobility course . The treatment was also safe for patients. As a result of the encouraging interim results, ProQR has concluded the Phase 1/2 trial and plans to move the treatment into a Phase 2/3 clinical trial. Ten people were treated in the Phase 1/2 study. Results from the interim analy sis were presented on September 5, 2018, at the Retinal Degeneration 2018 meeting in Killarney,   Ireland , by principal investigator   Artur Cideciyan , PhD , research professor of ophthalmology at the   Scheie Eye Ins...
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Cataract Surgery Leads to Longer Life in Women

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Written by Dr. David Evans Last modified on September 6, 2018 An article I wrote back in April described a National Bureau of Economic Research study that concluded cataract surgery patients have a life expectancy 1.8 years longer than people with cataracts who do not undergo surgery. Much of that piece focused on the finding that people, who have vision diminished by cataracts, are less likely to be active, and more likely to suffer accidental falls and related injuries. As a follow-on, a new study published in JAMA Ophthalmology suggests an even stronger link between cataract surgery and mortality risk. About the Study The genesis of this study was something completely different than evaluating effects of cataract vision loss. Its original purpose was to evaluate the effects of hormone therapy and dietary changes in postmenopausal women. That study was cut short once it was determined that hormone therapy increased the risk of vascular events. Although the hormone therapy aspect ...
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