BC Innovations | Oct 24, 2019
Distillery Therapeutics

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 treat mucopolysaccharidosis I (MPS I; Hurler syndrome), a lysosomal storage disease caused by insufficient IDUA activity. The enzyme deficiency...
BC Week In Review | Feb 15, 2019
Clinical News

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

Sangamo Therapeutics Inc. (NASDAQ:SGMO) reported interim data from two Phase I/II trials evaluating its in vivo genome editing zinc finger nuclease (ZFN) therapies SB-913 to treat mucopolysaccharidosis II (MPS II, Hunter syndrome) and SB-318 to...
BC Extra | Feb 8, 2019
Clinical News

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

Sangamo Therapeutics Inc. (NASDAQ:SGMO) lost $3.71 (31%) to $8.31 Thursday after the company reported interim data from two Phase I/II trials evaluating its in vivo genome editing zinc finger nuclease (ZFN) therapies SB-913 to treat...
BC Week In Review | Apr 27, 2018
Financial News

Sangamo raises $200M in follow-on

Sangamo Therapeutics Inc. (NASDAQ:SGMO) raised $200 million on April 25 through the sale of 12.3 million shares at $16.25 in a follow-on underwritten by BofA Merrill Lynch, J.P. Morgan and Cowen. The price is a...
BC Extra | Apr 26, 2018
Financial News

Sangamo raises $200M in follow-on

Sangamo Therapeutics Inc. (NASDAQ:SGMO) raised $200 million through the sale of 12.3 million shares at $16.25 in a follow-on underwritten by BofA Merrill Lynch, J.P. Morgan and Cowen. The price is a 9% discount to...
BC Week In Review | Mar 9, 2018
Clinical News

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

ArmaGen Inc. (Calabasas, Calif.) reported data from stage 2 of the Phase II portion of a Phase I/II trial in patients ages two and older with severe mucopolysaccharidosis I (MPS I, Hurler syndrome) showing that...
BC Week In Review | Apr 27, 2017
Clinical News

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

In February, ArmaGen reported preliminary data from 5 children ages ≥2 with Hurler syndrome who had previously received enzyme replacement therapy (ERT) in stage 2 of the open-label, dose-escalation, Brazilian Phase I/II AGT-181-101 trial showing...
BC Week In Review | Mar 2, 2017
Clinical News

SB-318 regulatory update

FDA granted rare pediatric disease designation to SB-318 to treat mucopolysaccharidosis I (MPS I, Hurler syndrome). The product is an adeno-associated virus (AAV) vector containing zinc finger DNA binding protein (ZFP) nucleases (ZFNs) targeting the...
BC Week In Review | Aug 8, 2016
Clinical News

RGX-121 regulatory update

FDA granted Rare Pediatric Disease designation to RegenxBio’s RGX-121 to treat mucopolysaccharidosis type II (MPS II Hunter’s syndrome). RegenxBio said it intends to submit INDs for both RGX-121, an adeno-associated virus serotype 9 (AAV9) vector...
BC Week In Review | Jan 11, 2016
Clinical News

RGX-111 regulatory update

FDA granted rare pediatric disease designation to RGX-111 from RegenxBio to treat mucopolysaccharidosis I (MPS I, Hurler syndrome). The designation is among the criteria that would make RegenxBio eligible to receive a Priority Review voucher...
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