CRISPR Gene Editing Promises to Cure 10,000 diseases. Now in Its First Human Clinical Trial. Will it Eventually Deliver?

  Clustered Regularly Interspaced Short Palindromic Repeats is more commonly known as CRISPR. As reported recently in Science News, the CRISPR/Cas9 “molecular scissors” is slated to make its highly anticipated debut…

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The World’s First Editing of DNA in Humans Using CRISPR to Treat Leber Congenital Amaurosis

  Allergan plc, a leading global pharmaceutical company, and its partner, Editas Medicine, Inc. have been given the green light for a clinical trial designed to treat patients with severe…

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As Gene Therapy Becomes a Reality, Spark Therapeutics Looks at Challenges That Still Remain
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As Gene Therapy Becomes a Reality, Spark Therapeutics Looks at Challenges That Still Remain

According to a story from BioPharma Dive, the advent of gene therapy development at Spark Therapeutics has been five years in the making. At this juncture, it has been nearly…

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New Potential Gene Therapy for Retinal Degeneration Granted ODD

Ocugen Pharmaceuticals has just received their first ever orphan drug designation by the FDA for a gene therapy treatment. It's called OCU400 and it's being investigated as a treatment for…

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Single Use Gene Therapy Approved in the EU for Leber’s Congenital Amaurosis
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Single Use Gene Therapy Approved in the EU for Leber’s Congenital Amaurosis

According to a story from pm360online.com, the drug developer Novartis has recently announced that the company's gene therapy Luxturna has gained approval in the EU as a treatment for Leber's…

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This Company is Working on Treatment for Rare Retinal Diseases
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This Company is Working on Treatment for Rare Retinal Diseases

According to a story from Eye on the Cure, the biopharmaceutical company Ophthotech is hard at work researching and developing gene therapy solutions for a variety of rare retinal conditions.…

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