Tag Archives: MPS I

October, 2018

17 October
FDA Grants Immusoft’s Investigational MPS I Treatment Rare Pediatric Disease Designation

SEATTLE–(BUSINESS WIRE)–Immusoft Corporation, a Seattle-Wash.-based cell therapy company, announced today that the U.S. Food and Drug Administration (FDA) has granted it Rare Pediatric Disease Designation (RPDD) for Iduronicrin genleukocel-T, Immusoft’s Sleeping Beauty transposon-engineered autologous plasmablasts for the expression and delivery of alpha-L-iduronidase (IDUA) to treat Mucopolysaccharidosis type I (MPS I). …
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13 June
FDA Grants Fast Track Designation to REGENXBIO’s RGX-111 Gene Therapy for Treatment of MPS I

ROCKVILLE, Md., June 12, 2018 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RGX-111. RGX-111 is a novel, one-time investigational …
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27 February
Sangamo Therapeutics Receives Rare Pediatric Disease Designation from the FDA for its MPS I Candidate

RICHMOND, Calif., Feb. 27, 2017 /PRNewswire/ — Sangamo Therapeutics, Inc. (Nasdaq: SGMO), the leader in therapeutic genome editing, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for SB-318, the Company’s in vivo genome editing product candidate for the treatment of Mucopolysaccharidosis Type …
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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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29 July
Penn Study Verifies Human Gene Therapy in Model of Rare Metabolic Disorder

PHILADELPHIA — Researchers are closer to finding a better way to treat children with a rare metabolic disorder called MPS I. It is caused by a deficiency of the key enzyme IDUA needed to break down complex sugars in cells. MPS I eventually leads to the abnormal accumulation of sugar …
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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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