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A total of 451 posts are filed under Innovation
Sangamo and Pfizer to Collaborate on Development of Gene Therapy for ALS
Rare Daily Staff Sangamo Therapeutics and Pfizer said they will collaborate to develop a potential gene therapy using zinc finger protein… Continue Reading
FDA Approves Spark’s Gene Therapy to Treat Patients with a Rare Form of Inherited Vision Loss
Rare Daily Staff The U.S. Food and Drug Administration said it approved Luxturna, Spark Therapeutics’ gene therapy to treat an inherited… Continue Reading
Study in the Journal Blood Suggest uniQure Gene Therapy Provides Durable Response in Hemophilia B Patients
Rare Daily Staff Gene therapy company uniQure said a study published in the journal Blood based clinical data from an ongoing phase 1/2… Continue Reading
Ionis Licenses Huntington’s Disease Drug to Roche After Promising Results
Rare Daily Staff Ionis Pharmaceuticals said that Roche has exercised its option to license IONIS-HTTRx following the completion of a… Continue Reading
Spark and Pfizer Gene Therapy Shines in NEJM Report of Interim Data from Study in Hemophilia B
Rare Daily Staff Interim data from a phase 1/2 study of Spark Therapeutics’ and Pfizer’s experimental gene therapy SPK-9001 for… Continue Reading
Vanderbilt Center to Integrated Face2Gene Apps into its Center for Undiagnosed Diseases
Rare Daily Staff Vanderbilt University Medical Center said it would integrate FDNA’s Face2Gene suite of applications into the Vanderbilt… Continue Reading
Ultragenyx and Kyowa Kirin Report Positive Data from Late-State X-Linked Hypophosphatemia Study
Ultragenyx Pharmaceutical, Kyowa Hakko Kirin, and Kyowa Kirin International reported positive results from a 48-week late-stage study of… Continue Reading