19:51 , Apr 27, 2018 |  BC Week In Review  |  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...
17:27 , Apr 26, 2018 |  BC Extra  |  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...
18:57 , Mar 9, 2018 |  BC Week In Review  |  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...
21:09 , Apr 27, 2017 |  BC Week In Review  |  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...
20:19 , Mar 2, 2017 |  BC Week In Review  |  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...
07:00 , Aug 8, 2016 |  BC Week In Review  |  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...
08:00 , Jan 11, 2016 |  BC Week In Review  |  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...
01:33 , Dec 31, 2015 |  BC Extra  |  Top Story

RegenxBio MPS therapy gets rare pediatric disease designation

RegenxBio Inc. (NASDAQ:RGNX) said FDA granted rare pediatric disease designation to RGX-111, a gene therapy in preclinical testing to treat mucopolysaccharidosis I (MPS I, Hurler syndrome). The designation is among the criteria that would make...
08:00 , Nov 16, 2015 |  BC Week In Review  |  Clinical News

HIRMAb-IDUA regulatory update

FDA granted rare pediatric disease designation to AGT-181 from ArmaGen to treat mucopolysaccharidosis I (MPS I, Hurler syndrome). AGT-181 is in Phase I/IIa testing for MPS I. The product is alpha-L-iduronidase (IDUA) fused to...
07:00 , Sep 21, 2015 |  BC Week In Review  |  Clinical News

HIRMAb-IDUA: Phase I/IIa started

ArmaGen began an open-label, U.S. Phase I/IIa trial to evaluate 1, 3 and 6-9 mg/kg IV AGT-181 once weekly for 8 weeks in about 9 adult patients. ArmaGen Inc., Calabasas, Calif.   Product: HIRMAb-IDUA (...