16:39 , Jul 18, 2019 |  BC Innovations  |  Distillery Techniques

CRISPR RNA editing platform converts C bases to U

TECHNIQUES CATEGORY: Drug platforms TECHNOLOGY: Gene therapy Feng Zhang's group developed a CRISPR-based RNA editing system that converts cytidine to uridine bases and could be used to edit pathogenic RNA mutations. The system, dubbed RESCUE...
01:30 , Jul 13, 2019 |  BioCentury  |  Tools & Techniques

Broadening role for external control arms in clinical trials

External control arms are moving from theory to practice as drug developers begin to use them to make internal go/no-go decisions for clinical programs and to support regulatory applications. The field is largely split between...
22:22 , Jul 12, 2019 |  BC Extra  |  Preclinical News

July 12 Preclinical Quick Takes: Feng Zhang develops latest RNA editing platform, plus tools from David Liu and NIH

Zhang's RESCUE platform converts RNA C to U bases  Feng Zhang and colleagues at Massachusetts Institute of Technology and the Broad Institute of MIT and Harvard have developed a single base RNA editing system that...
00:01 , May 11, 2019 |  BC Innovations  |  Translation in Brief

Arvinas, Palleon founders extend the reach of targeted degraders

A study from Arvinas founder Craig Crews is the second report in as many months of a technology geared to degrade extracellular proteins. The platforms open up new territory for targeted degradation therapies, on the...
01:42 , May 4, 2019 |  BioCentury  |  Product Development

How the amyloid hypothesis holds its grip

By now, two things are clear about the amyloid hypothesis of Alzheimer’s disease: one is that the amyloid peptide is somehow involved; the other is that it’s a terrible target. β amyloid has had an...
01:39 , May 4, 2019 |  BioCentury  |  Product Development

Amyloid: how did we get here and what can we learn?

Unlike any other theory in drug development, the amyloid hypothesis of Alzheimer’s disease has maintained an almost cult-like following, despite two decades littered with a long line of late-stage failures and not a single success....
01:33 , Apr 19, 2019 |  BC Innovations  |  Targets & Mechanisms

The next wave in neurodegeneration expands beyond neurons

Working in the background, while amyloid therapies have floundered, has been a group of companies betting that the immune system can do for neurodegeneration what it is has done for oncology. The first wave of...
21:44 , Mar 22, 2019 |  BC Extra  |  Clinical News

After aducanumab failure, Eisai to test another anti-amyloid mAb

With the announcement that Eisai plans to start yet another Phase III trial of an anti-amyloid candidate, Alzheimer's disease researcher Rudolph Tanzi told BioCentury he wouldn't enroll any more AD patients in trials of therapies...
03:36 , Jan 4, 2019 |  BC Innovations  |  Tools & Techniques

Making AD mice as diverse as patients

The Jackson Laboratory aims to make Alzheimer’s disease mouse models more useful for predicting which patients will respond to therapy by engineering in a high degree of genetic diversity to better reflect trial populations. The...
21:59 , Dec 6, 2018 |  BC Week In Review  |  Clinical News

Yumanity reveals Parkinson's candidate as first compound from yeast platform

Yumanity Therapeutics LLC (Cambridge, Mass.) and colleagues used the company's yeast platform to identify inhibitors of the enzyme stearoyl-CoA desaturase-1 (SCD1) that could prevent α-synuclein toxicity in Parkinson's disease. The SCD1 inhibitors are the first...