23:55 , May 7, 2019 |  BC Extra  |  Clinical News

More SMA data at AAN suggest new therapies could displace Spinraza

Novartis continued to add on new data for its spinal muscular atrophy gene therapy Zolgensma at AAN while Roche and Genentech presented updated SMA data for their oral small molecule risdiplam. The dosing and potential...
20:17 , May 3, 2019 |  BC Extra  |  Preclinical News

May 3 Preclinical Quick Takes: CRISPR, Cincinnati Children's, Chromatin

Small molecule CRISPR inhibitors are here  Two groups have reported small molecule inhibitors this week that could be used to control CRISPR-based gene editing, as the U.S. Defense Advanced Research Projects Agency looks for ways...
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...
17:40 , Apr 25, 2019 |  BC Innovations  |  Translation in Brief

Risking CRISPR correction before birth

A team from CHOP and UPenn has published its second study showing CRISPR-based gene editing can treat deadly diseases in the womb but has yet to address the safety repercussions for the mother and fetus....
21:13 , Apr 17, 2019 |  BC Extra  |  Clinical News

Ionis slides after AveXis reports SMA data for Zolgensma

Ionis lost $10.61 (13%) to $72.67 on Wednesday after AveXis reported that Zolgensma improved event-free survival, motor function and milestone achievement in the Phase III STR1VE trial to treat the type 1 form of spinal...
21:32 , Mar 14, 2019 |  BC Extra  |  Preclinical News

Antibody-drug conjugate against ALK could selectively treat neuroblastoma

Researchers from CHOP and Celldex showed that an antibody-drug conjugate targeting ALK selectively kills neuroblastoma cells, suggesting the ADC could have a better safety profile than marketed drug dinutuximab. Unituxin dinutuximab from United Therapeutics Corp....
01:10 , Mar 2, 2019 |  BioCentury  |  Product Development

Roche broadens its BD scope

Roche’s proposed acquisition of Spark Therapeutics Inc. is the pharma’s first major deal since James Sabry took over partnering, and could be a glimpse of what’s to come as the pharma broadens its horizons on...
21:06 , Dec 14, 2018 |  BC Week In Review  |  Clinical News

Adcetris improves PFS by 27.4 months in first-line PTCL

Seattle Genetics Inc. (NASDAQ:SGEN) and Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) reported detailed data from the Phase III ECHELON-2 trial to treat previously untreated CD30-expressing peripheral T cell lymphoma (PTCL) showing that Adcetris brentuximab vedotin plus...
04:21 , Nov 30, 2018 |  BC Innovations  |  Targets & Mechanisms

Adhesion targets emerge at ASH 2018

The next wave of targets in blood cancers and hematology will come from adhesion molecules, innate immune cells and regulators of gene expression, according to BioCentury’s analysis of emerging targets at ASH 2018. BioCentury’s analysis...
03:21 , Nov 17, 2018 |  BioCentury  |  Product Development

Betting Combos on Biomarkers

Despite lackluster monotherapy data at SITC from the latest tranche of immunotherapy candidates, drug developers are moving into combination trials with PD-1 and PD-L1 inhibitors based on biomarkers of pharmacodynamic activity. Results from the next...