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....
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...
13:22 , May 31, 2017 |  BC Innovations  |  Distillery Techniques

Disease models

TECHNOLOGY: Cell models A human cell-based model of Angelman syndrome could be used to screen therapies and identify therapeutic targets. Induced pluripotent stem (iPS) cell-derived neurons from two patients harboring deletions of the maternal chromosomal...
07:00 , Jun 9, 2016 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Ubiquitin protein ligase E3A (UBE3A; E6AP)

Cancer INDICATION: Breast cancer Patient sample and mouse studies suggest promoting UBE3A expression could help treat metastatic breast cancer. In samples from 1,992 patients, low tumor levels of UBE3A mRNA correlated with poor survival, and...
07:00 , Jun 15, 2015 |  BC Week In Review  |  Company News

University of South Florida, Agilis Biotherapeutics deal

The university granted Agilis exclusive, worldwide rights to develop and commercialize UBE3a gene therapies to treat Angelman syndrome. The university will receive an undisclosed upfront payment, plus undisclosed milestones and royalties. University of South Florida...
07:00 , Apr 20, 2015 |  BioCentury  |  Strategy

Metamorphoses of Ovid

Neurology company Ovid Therapeutics Inc. is already tapping into the expertise of its new CEO, veteran dealmaker Jeremy Levin. The biotech is in-licensing molecules that have reached Phase III testing but are not approved in...
08:00 , Jan 8, 2015 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Ubiquitin protein ligase E3A (UBE3A; E6AP)

Neurology INDICATION: Neurology In vitro and mouse studies suggest an antisense oligonucleotide (ASO) against UBE3A-ATS - a long noncoding RNA UBE3A antisense transcript - could help treat Angelman syndrome. The disease is caused by maternal...
08:00 , Dec 11, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Various

Indication Target/marker/pathway Summary Licensing status Publication and contact information Various Various E3 ubiquitin ligase; SMAD specific E3 ubiquitin protein ligase 2 (SMURF2); NEDD4 family E3 ubiquitin-protein ligase (RSP5) In vitro studies identified compounds that could...
08:00 , Jan 26, 2012 |  BC Innovations  |  Cover Story

Macrocycles by the trillions

Japanese researchers have developed a method for creating large libraries of N-methylated peptide macrocycles, 1 a class of molecules that are sized between small molecules and biologics and have the potential to combine the pharmacological...