01:50 , Sep 14, 2018 |  BC Innovations  |  Targets & Mechanisms

Repeat offenders

Dozens of intractable DNA repeat-driven diseases could boil down to a common problem: disrupted boundaries between chromatin domains, according to a study from the University of Pennsylvania. The results present new opportunities for diseases like...
17:50 , Jul 20, 2018 |  BC Week In Review  |  Company News

PTC acquiring CNS gene therapy play Agilis

PTC Therapeutics Inc. (NASDAQ:PTCT) will acquire gene therapy company Agilis Biotherapeutics Inc. (Cambridge, Mass.), giving the rare disease company four products in development for CNS disorders. PTC will pay Agilis shareholders about $200 million up front,...
22:43 , Jul 19, 2018 |  BC Extra  |  Company News

PTC acquiring CNS gene therapy play Agilis

PTC Therapeutics Inc. (NASDAQ:PTCT) will acquire gene therapy company Agilis Biotherapeutics Inc. (Cambridge, Mass.), giving the rare disease company four products in development for CNS disorders. PTC will pay Agilis shareholders about $200 million up...
16:45 , Feb 12, 2018 |  BC Extra  |  Preclinical News

HDAC3 inhibition promotes peripheral remyelination

A study published in Nature Medicine identified histone deacetylase 3 (HDAC3) as a target for boosting remyelination in peripheral neuropathies. Following demyelination in a mouse model of sciatic nerve injury, researchers from Cincinnati Children’s Hospital Medical...
03:28 , Jan 19, 2018 |  BC Innovations  |  Tools & Techniques

Synthetic expressions

Bringing synthetic biology to transcriptional regulation could catalyze a new generation of gene therapies that are more tunable, disease-specific and easy to deliver than the products dominating the field. The new constructs include synthetic promoters that...
22:06 , Jan 9, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Ataxia Patient sample and cell culture studies suggest a synthetic transcription elongation factor that induces FXN expression could help treat Friedreich’s ataxia. The synthetic molecule consists of a small molecule bromodomain containing 4 (BRD4) ligand...
21:47 , Nov 30, 2017 |  BC Innovations  |  Targets & Mechanisms

All-purpose HSCs

While most eyes are on hematopoietic stem cells for their ability to treat blood cancers, a UCSD group has built a body of work showing the immune cell precursors can treat diseases with no relation...
20:46 , Nov 10, 2017 |  BC Week In Review  |  Financial News

Voyager raises $54M follow-on

On Nov. 7, gene therapy company Voyager Therapeutics Inc. (NASDAQ:VYGR) raised $54 million through the sale of 4.5 million shares at $12 in a follow-on underwritten by Morgan Stanley, Cowen, Stifel, Wells Fargo and Nomura....
21:47 , Nov 8, 2017 |  BC Extra  |  Financial News

Voyager raises $54M in follow-on

Voyager Therapeutics Inc. (NASDAQ:VYGR) raised $54 million late Tuesday through the sale of 4.5 million shares at $12 in a follow-on underwritten by Morgan Stanley, Cowen, Stifel, Wells Fargo Securities and Nomura. The price is...
04:52 , Nov 3, 2017 |  BC Week In Review  |  Company News

Sanofi declines option for Voyager's PD gene therapy

The Genzyme unit of Sanofi (Euronext:SAN; NYSE:SNY) declined to exercise its exclusive option to obtain ex-U.S. rights to VY-AADC-01 from Voyager Therapeutics Inc. (NASDAQ:VYGR) to treat Parkinson’s disease. According to Voyager, Genzyme decided to opt out...