BioCentury | Oct 24, 2019

Engineered hematopoietic stem and progenitor cells for mucopolysaccharidosis type I

...DISEASE CATEGORY: Endocrine/Metabolic INDICATION: Mucopolysaccharidosis Hematopoietic stem and progenitor cells (HSPCs) engineered to overexpress IDUA could...
...could treat mucopolysaccharidosis I (MPS I; Hurler syndrome), a lysosomal storage disease caused by insufficient IDUA...
...vector delivering IDUA to the CNS, in Phase I testing for MPS I. TARGET/MARKER/PATHWAY: α-L-iduronidase (IDUA...
BioCentury | Feb 15, 2019
Clinical News

Sangamo looks to next-gen therapies after reporting first version 'not enough'

...targeting the albumin locus and the gene for IDUA. In EMPOWERS, SB-318 increased in leukocyte IDUA...
...or IDUA; safety Status: Interim Phase I/II data Milestone: Additional Phase I/II data Chris Lieu SB-318 SB-913 Sangamo Therapeutics Inc. Alpha-L-iduronidase (IDUA) Iduronate...
BioCentury | Feb 8, 2019
Clinical News

Sangamo looks to next-gen therapies after reporting first version 'not enough'

...targeting the albumin locus and the gene for IDUA. In EMPOWERS, SB-318 increased in leukocyte IDUA...
...from both trials this year. IDUA - α-L-iduronidase ; IDS - iduronate 2-sulfatase Chris Lieu SB-318 SB-913 Sangamo Therapeutics Inc. Alpha-L-iduronidase (IDUA) Iduronate...
BioCentury | Apr 27, 2018
Financial News

Sangamo raises $200M in follow-on

...locus to treat hemophilia B; SB-318, which targets the albumin locus and the gene for alpha-L-iduronidase...
BioCentury | Apr 26, 2018
Financial News

Sangamo raises $200M in follow-on

...locus to treat hemophilia B; SB-318, which targets the albumin locus and the gene for alpha-L-iduronidase...
BioCentury | Mar 9, 2018
Clinical News

ArmaGen reports 52-week Phase II data for MPS I candidate

...ages two and older with severe mucopolysaccharidosis I (MPS I, Hurler syndrome) showing that AGT-181 (HIRMAb-IDUA...
...of the trial (see BioCentury, April 27, 2017 ). The enzyme replacement therapy consisting of alpha-L-iduronidase (IDUA)...
...AGT-181 (HIRMAb-IDUA) Business: Endocrine/Metabolic Molecular target: Insulin receptor (INSR) Description: Enzyme replacement therapy consisting of alpha-L-iduronidase (IDUA)...
BioCentury | Apr 27, 2017
Clinical News

AGT-181: Preliminary Ph I/II AGT-181-101 data

...Inc. , Calabasas, Calif. Product: AGT-181 (HIRMAb-IDUA) Business: Endocrine/Metabolic Molecular target: Insulin receptor (INSR) Description: Alpha-L-iduronidase (IDUA)...
...neuroimaging parameters Status: Preliminary Phase I/II data Milestone: Start pivotal trial (year end 2017) Alex Himes AGT-181 HIRMAb-IDUA ArmaGen Inc. Alpha-L-iduronidase (IDUA) Insulin...
BioCentury | Mar 2, 2017
Clinical News

SB-318 regulatory update

...finger DNA binding protein (ZFP) nucleases (ZFNs) targeting the albumin locus and the gene for alpha-L-iduronidase (IDUA)...
BioCentury | Aug 8, 2016
Clinical News

RGX-121 regulatory update

...vector that delivers the IDS gene, and RGX-111 , an AAV9 vector delivering the huiman alpha-L-iduronidase (IDUA)...
BioCentury | Jan 11, 2016
Clinical News

RGX-111 regulatory update

...I/II trial of RGX-111, an adeno-associated virus serotype 9 (AAV9) vector that delivers the human alpha-L-iduronidase (IDUA)...
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