00:28 , May 24, 2019 |  BC Innovations  |  Emerging Company Profile

Anima opens new target space in translational control

Anima Biotech’s answer to undruggable targets is to go after the machinery that turns their mRNA into protein, adding a new approach to the gene regulation toolkit. The strategy should give Anima a leg up...
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....
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...
21:16 , Apr 12, 2019 |  BC Week In Review  |  Clinical News

Wave down on delay for Huntington disease readouts

Wave lost $7.84 (19%) to $33.91 on April 10 after it said top-line data from two Phase Ib/IIa trials of its antisense oligonucleotide therapies for Huntington disease will be delayed until year end due to...
23:41 , Feb 7, 2019 |  BC Innovations  |  Finance

Pharma ventures earlier

Facing the squeeze from inside and out, at least three corporate venture arms are revamping operations to increase their foothold in earlier and riskier investment opportunities. From one side, the move reflects the growing reliance...
17:59 , Oct 25, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Huntington’s disease (HD) Cell culture and fruit fly studies suggest promoting USP12 expression could help treat HD. In rat primary neurons expressing an HD-related mutant HTT fragment and induced pluripotent stem (iPS) cell-derived neurons...
18:19 , Oct 18, 2018 |  BC Innovations  |  Distillery Techniques

Biomarkers

TECHNOLOGY: Plasma markers; tissue markers CSF levels of mutant HTT, and CSF or plasma levels of NEFL , could help diagnose HD and differentiate between manifest and premanifest disease. In 20 premanifest HD patients, 40...
18:05 , Oct 12, 2018 |  BC Week In Review  |  Company News

Roche gets option to Ionis' antisense dry AMD candidate

Roche (SIX:ROG; OTCQX:RHHBY) will pay Ionis Pharmaceuticals Inc. (NASDAQ:IONS) $75 million up front for an option to develop and commercialize IONIS-FB-LRx, a ligand-conjugated antisense (LICA) oligonucleotide targeting complement factor B (CFB). The partners plan to...
19:12 , Oct 10, 2018 |  BC Extra  |  Company News

Roche gets option to Ionis' antisense dry AMD candidate

Roche (SIX:ROG; OTCQX:RHHBY) will pay Ionis Pharmaceuticals Inc. (NASDAQ:IONS) $75 million up front for an option to develop and commercialize IONIS-FB-LRx, a ligand-conjugated antisense (LICA) oligonucleotide targeting complement factor B (CFB). The partners plan to...
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...