Tag Archives: lysosomal storage disorders

September, 2018

21 September
Amicus Acquires Gene Therapy Portfolio of Ten AAV Programs in Neurologic Lysosomal Storage Disorders

CRANBURY, N.J., Sept. 20, 2018 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced the signing of a definitive agreement in which Amicus Therapeuticswill receive worldwide development and commercial rights for ten gene therapy programs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. The ten programs are licensed …
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22 September
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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6 February
FDA Permits Marketing of First Newborn Screening System for Detection of Four, Rare Metabolic Disorders

The U.S. Food and Drug Administration today permitted marketing of the Seeker System for the screening of four, rare Lysosomal Storage Disorders (LSDs) in newborns. The Seeker system is designed to detect Mucopolysaccharidosis Type I(MPS I), Pompe, Gaucher and Fabry. It is the first newborn screening test permitted to be …
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8 February
FDA Clears Sangamo Biosciences’ IND Application of SB-318 for Treatment of MPS I

RICHMOND, Calif., Feb. 8, 2016 /PRNewswire/ — Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type …
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8 April
FDA Accepts ArmaGen’s IND for its Hurler Syndrome Candidate

Privately-held biotech ArmaGen, Inc. announced today that US health regulators have accepted for review its investigational New Drug (IND) application for its drug for a rare lysosomal storage disease. The company, focused on developing novel therapies to treat severe neurological disorders, said that the US Food and Drug Administration (FDA) …
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