00:11 , Aug 18, 2018 |  BioCentury  |  Product Development

Doing more with less

In rare diseases where natural history data are scarce, Phase II studies may have to perform dual duty to both seek efficacy signals and test out new endpoints. Ovid Therapeutics Inc. and Clementia Pharmaceuticals Inc....
00:38 , Aug 9, 2018 |  BC Innovations  |  Translation in Brief

Identifying the ubiquitinators

A team at University of Dundee and Newcastle University has developed a mass spectrometry-based screening platform to identify ubiquitin ligases and compounds that modulate them. The approach uses the uptake of ubiquitin by enzymes rather...
21:35 , Aug 3, 2018 |  BC Extra  |  Preclinical News

Mechanism identified behind thalidomide-induced birth defects

Researchers at Dana-Farber Cancer Institute uncovered the mechanistic pathway by which thalidomide and two related analogs cause severe fetal harm, answering a long-held question about the three teratogenic drugs now used as cancer therapies. The insight...
20:12 , Aug 1, 2018 |  BC Innovations  |  Distillery Techniques

Assays and screens

TECHNOLOGY: Cell-free assays; high throughput screening A high throughput, mass spectrometry-based screening assay could identify E3 ligase inhibitors and interactive pairs of E2/E3 ubiquitination enzymes as potential drug targets. For the latter, the method involves combining...
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...
21:43 , Jul 12, 2018 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: June 2018

New Therapeutic Targets and Biomarkers: June 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during June 2018. Therapeutic targets are defined as any protein, gene or other molecule...
18:18 , Jun 13, 2018 |  BC Innovations  |  Distillery Therapeutics

Cardiovascular

INDICATION: Heart failure Patient sample and cell culture studies suggest inhibiting microtubule detyrosination or promoting expression of tubulin tyrosine ligase (TTL) could help treat heart failure. In left ventricular tissue samples from patients, the density of...
23:28 , Jun 7, 2018 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: May 2018

New Therapeutic Targets and Biomarkers: May 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during May 2018. Therapeutic targets are defined as any protein, gene or other molecule...
22:15 , May 11, 2018 |  BC Extra  |  Preclinical News

Inhibiting the ubiquitin ligase RNF5 to treat CF

In a paper published in Cell Chemical Biology, Italian researchers identified a pharmacological inhibitor of ring finger protein 5 (RNF5) that prevented the premature degradation of mutant F508del cystic fibrosis transmembrane conductance regulator (CFTR) protein,...