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Category Archives: Genetic Therapy

SMA Gene Therapy Promising in Early Results | Medpage Today – MedPage Today

BOSTON -- Early results from a small phase I trial of a gene therapy for spinal muscular atrophy (SMA) were promising, with many children hitting milestones not typically seen in the condition, researchers said here. Continue reading

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Pulling the Plug on the First Gene Therapy Drug – PLoS Blogs (blog)

2017 is supposed to be the year that FDA finally approves a gene therapy. Continue reading

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Spark hemophilia B gene therapy clears another early test – FierceBiotech

Spark Therapeutics has posted fresh data from a phase 1/2 trial of its hemophilia B gene therapy, SPK-9001. The data suggest Sparks tapering courses of steroids successfully arrested the factor IX (FIX) declines seen in two patients who appeared to have an immune reaction to the Spark100 vector capsid. Philadelphia-based Spark first reported on the two patients in December. Continue reading

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Abeona Announces Rare Disease Gene Therapy Symposium with the New York Academy of Sciences – GlobeNewswire (press release)

April 05, 2017 07:43 ET | Source: Abeona Therapeutics Inc Company CEO to Co-Chair Symposium being held on April 11th in NYC NEW YORK and CLEVELAND, April 05, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare genetic diseases, is pleased to announce a Gene Therapy for Rare Diseases Symposium with the New York Academy of Sciences (NYAS) and the Biochemical Pharmacology Discussion Group in New York City on April 11th, 2017. Continue reading

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Angionetics Nears Key Gene Therapy Trial for Coronary Heart Disease – Xconomy

Xconomy San Diego Christopher Reinhard will tell you there is nothing unusual about the 20-plus years hes spent getting an experimental heart therapy to late-stage clinical trials. Very rarely will you get a short story on development of a drug, said Reinhard (above), who is a principal investor and the CEO of San Diego-based Angionetics. Two decades is about what you would expect for a new drug-making method, Reinhard said. Continue reading

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New Gene Therapy for Cancer Offers Hope to Those With No … – NBCNews.com

Dimas Padilla, 43, of Kissimmee, is in remission from non-Hodgkin's lymphoma after receiving an experimental cancer therapy called CAR-T. Continue reading

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