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Rare Diseases

FDA Committee Unanimously Recommends Approval of Spark’s Gene Therapy for Rare Eye Disease

PHILADELPHIA, Oct. 12, 2017 (GLOBE NEWSWIRE) — Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended (16-0) approval of LUXTURNA™ (voretigene neparvovec), an investigational, potential one-time gene therapy, …

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FDA Accepts Prometic’s BLA for its Rare Disease Drug Ryplazim

LAVAL, QC, Oct. 13, 2017 /PRNewswire/ – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic) today announced that the U.S Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for its plasminogen replacement therapy (RyplazimTM) having  granted a priority review status and set a Prescription Drug …

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Biogen’s Spinraza Data Show Initiating Treatment Earlier May Improve Motor Function in SMA

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen (NASDAQ: BIIB) presented new data demonstrating that earlier initiation of treatment with SPINRAZA® (nusinersen) may improve motor function outcomes in infants and children with spinal muscular atrophy (SMA). Results continued to reinforce the favorable efficacy and safety profile of SPINRAZA. The data were shared at the 22nd International Annual Congress …

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Bluebird Gene Therapy Halts Progression of Cerebral Adrenoleukodystrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, today announced that interim data from an initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™ investigational …

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Catabasis’ Edasalonexent Demonstrates Positive Mid-Stage Results in Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported new positive efficacy results showing sustained disease-modifying effects in the MoveDMD trial open-label extension following 24 and 36 weeks of treatment with edasalonexent. Across all key assessments of muscle function, improvements were observed in the rate of decline after …

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Zogenix’s Treatment for Dravet Syndrome Demonstrates Positive Results in Late-Stage Study

EMERYVILLE, Calif., Sept. 29, 2017 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ:ZGNX), a pharmaceutical company developing therapies for the treatment of rare central nervous system (CNS) disorders, today reported positive top-line results from its first Phase 3 trial (Study 1) for its investigational drug, ZX008 (low-dose fenfluramine hydrochloride), for the treatment of Dravet syndrome. The …

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Fibrocell Reports Interim Results of Phase 1/2 Trial of Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

EXTON, Pa., Sept. 26, 2017 (GLOBE NEWSWIRE) — Fibrocell Science, Inc. (NASDAQ:FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today reported interim results in its Phase 1/2 clinical trial of FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Three …

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Avrobio Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease

CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Pompe disease. This pre-clinical program becomes AVROBIO’s third gene therapy for Lysosomal Storage Disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program …

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FDA Grants Orphan Drug Designation to PhaseRx’s Rare Liver Disease Drug

SEATTLE, Sept. 20, 2017 /PRNewswire/ — PhaseRx, Inc. (NASDAQ: PZRX), a biopharmaceutical company developing mRNA treatments for life-threatening inherited liver diseases in children, today announced that its second drug development candidate, PRX-ASL, for the treatment of argininosuccinate lyase deficiency (ASLD), has received orphan drug designation by the U.S. Food and …

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Alnylam’s Rare Disease Drug Meets Endpoints in Late-Stage hATTR Amyloidosis Study

CAMBRIDGE, Mass. & PARIS–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR amyloidosis with polyneuropathy, met its primary efficacy endpoint …

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