21:53 , May 29, 2019 |  BC Extra  |  Company News

Amicus bolsters rare disease gene therapy pipeline with expanded UPenn deal

Amicus has added three more rare disease gene therapy programs to its pipeline through an expansion of its deal with the University of Pennsylvania, with the option to add at least a dozen more. The...
23:04 , May 8, 2019 |  BC Extra  |  Preclinical News

May 8 Preclinical Quick Takes: Moderna, WntRx, schizophrenia, heart attack

Moderna debuts candidate for rare glycogen storage disease  Moderna Inc. (NASDAQ:MRNA) revealed a new preclinical mRNA candidate to treat glycogen storage disease type Ia -- mRNA-3745, which encodes G6PC. The company presented data at the...
23:01 , May 2, 2019 |  BC Week In Review  |  Clinical News

Modest differences in outcomes for doses of Audentes myopathy gene therapy

Audentes presented updated data from the Phase I/II ASPIRO trial of AT132 to treat X-linked myotubular myopathy (XLMTM) showing that the trial's low and high doses had comparable safety and efficacy profiles. Chairman and CEO...
17:33 , May 1, 2019 |  BC Extra  |  Tools & Techniques

CHOP group achieves temporal control of gene editing with small molecules

As companies home in on ways to temporally control CRISPR/Cas9 gene editing, one academic group thinks small molecule stabilizers could be a solution. Beverly Davidson, a professor of pathology and laboratory medicine at The Children's...
15:32 , May 25, 2018 |  BC Week In Review  |  Clinical News

Audentes reports updated Phase I/II myopathy data for AT132

Audentes Therapeutics Inc. (NASDAQ:BOLD) reported updated interim data from the first cohort of the open-label, dose-escalation, U.S. Phase I/II ASPIRO trial testing AT132 in patients with X-linked myotubular myopathy (XLMTM). The presentation at the American...
15:32 , May 25, 2018 |  BC Week In Review  |  Clinical News

Abeona reports updated data for MPS IIIA gene therapy

Abeona Therapeutics Inc. (NASDAQ:ABEO) said ABO-102 led to significant dose- and time-dependent reductions in CSF and urine heparan sulfate levels through 18 months in a Phase I/II trial to treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo...
15:42 , May 24, 2018 |  BC Innovations  |  Emerging Company Profile

Hitching a (Gene)Ride

LogicBio Therapeutics Inc.’s GeneRide platform could reduce the oncogenic potential of gene therapies by co-opting the endogenous albumin promoter to express therapeutic transgenes. GeneRide comprises a DNA cassette encoding a therapeutic transgene flanked by homology...
23:26 , May 18, 2018 |  BC Extra  |  Clinical News

Abeona reports updated data for MPS IIIA gene therapy

Abeona Therapeutics Inc. (NASDAQ:ABEO) said ABO-102 led to significant dose- and time-dependent reductions in CSF and urine heparan sulfate levels through 18 months in a Phase I/II trial to treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo...
22:10 , Mar 22, 2018 |  BC Innovations  |  Translation in Brief

Switching scleroderma

While existing treatment options for localized scleroderma target inflammation, Fibrocell Science Inc. (NASDAQ:FCSC) and partner Intrexon Corp. (NYSE:XON) plan to enter the clinic next half with FCX-013, a therapy that could reverse fibrosis in patients...
16:53 , Mar 1, 2018 |  BC Innovations  |  Translation in Brief

Intellia’s delivery address

Intellia Therapeutics Inc. (NASDAQ:NTLA) published data supporting its lead in vivo gene editing program and providing proof of concept for its delivery technology this week. Now, the company wants to see just how far the...