21:17 , Apr 23, 2019 |  BC Extra  |  Company News

Fulcrum gets rights to GSK's shelved p38 kinase inhibitor

Following the failure of late-stage trials conducted by GSK in acute coronary syndrome and chronic obstructive pulmonary disease, Fulcrum will seek to revive the pharma's losmapimod for a rare genetic disease with an exclusive, worldwide...
19:42 , Feb 1, 2019 |  BC Week In Review  |  Company News

Voyager gets $165M up front from Neurocrine for gene therapies

Voyager Therapeutics Inc. (NASDAQ:VYGR) will receive $165 million up front from Neurocrine Biosciences Inc. (NASDAQ:NBIX) in exchange for a license to four of Voyager's neurology gene therapy programs. Voyager is eligible for $1.7 billion in...
20:41 , Jan 29, 2019 |  BC Extra  |  Company News

Voyager gets $165M up front from Neurocrine for gene therapies

Voyager Therapeutics Inc. (NASDAQ:VYGR) gained $3.08 (38%) to $11.12 on Tuesday after it said it will receive $165 million up front from Neurocrine Biosciences Inc. (NASDAQ:NBIX) in exchange for a license to four of Voyager's...
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...
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...
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...
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)...
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...