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In First Trial of New Gene Therapy Program, Pfizer Doses First Duchenne Muscular Dystrophy Patient biospace
April 16, 2018
Pfizer Inc. has dosed its first patient in a Phase Ib clinical trial of PF-06939926 for Duchenne muscular dystrophy (DMD).
Pfizer Doses First Patient Using Investigational Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy biospace
April 13, 2018
Pfizer Inc. has initiated a Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in boys with Duchenne muscular dystrophy (DMD).
GSK Divests Rare Disease Gene Therapy Business to Orchard Therapeutics biospace
April 13, 2018
GlaxoSmithKline Chief Executive Officer Emma Walmsley continues to trim excess fat from the company.
Children’s Hospital Los Angeles Treats First Patient on the West Coast Using Gene Therapy biospace
March 22, 2018
Surgeons of The Vision Center at Children's Hospital Los Angeles completed a revolutionary gene replacement procedure on Tuesday, March 20 at 9:20 AM
Gene therapy medication brings improvements in patients with severe SMA europeanpharmaceuticalreview
February 28, 2018
Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of the spinal muscular atrophy…
Scotland’s Synpromics expands gene therapy work with UCL deal pharmatimes
January 29, 2018
Edinburgh, Scotland based Synpromics has teamed up with UCL Great Ormond Street Institute of Child Health to develop novel gene therapies for diseases affecting the haematopoietic system.
Merck to work with bluebird bio on viral vector manufacturing pharmaasia
January 04, 2018
Merck aims to accelerate its manufacturing of new treatments for a broad range of serious diseases via this collaboration.
MeiraGTx obtains rare disease designation for gene therapy A001 pharmaceutical-technology
December 12, 2017
Gene Therapy Developer MeiraGTx Receives Outstanding Pediatric Disease Title from the U.S. Food and Drug Administration (FDA)
UniQure, chasing Spark-level FIX activity, tweaks hemophilia B gene therapy ahead of 2018 pivotal tr fiercebiotech
October 20, 2017
UniQure has modified its hemophilia B gene therapy to dial up factor IX (FIX) activity to the levels achieved by Spark Therapeutics’ rival product.
Merck Brings Customers Closer to Gene Therapy Product Commercialization prnasia
October 19, 2017
Merck, a leading science and technology company, today announced that its Carlsbad, California-based manufacturing facility for the production of BioReliance® viral and gene therapy products has completed both a U.S.

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