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...
20:10 , Aug 1, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Huntington’s disease (HD) Mouse studies identified a PPP1R15B inhibitor that could help treat HD. Chemical synthesis and in vitro testing of guanabenz analogs yielded a compound that bound PPP1R15B with a K d of...
15:42 , Jul 25, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Huntington's disease (HD) Cell culture and mouse studies suggest N6-furfuryladenine could help treat HD by promoting HTT phosphorylation. Screening of commercially available natural products in phosphorylation assays in a mouse striatal neuron cell line...
16:29 , May 4, 2018 |  BC Week In Review  |  Clinical News

uniQure reports preclinical data for Huntington's disease candidate

uniQure N.V. (NASDAQ:QURE) reported preclinical data showing that AMT-130 decreased mutant huntingtin (HTT) protein in animal models of Huntington's disease. Data were presented at the American Academy of Neurology meeting in Los Angeles. In a...
16:17 , May 4, 2018 |  BC Week In Review  |  Financial News

uniQure raises $128.5M follow-on

Gene therapy company uniQure N.V. (NASDAQ:QURE) raised $128.5 million on May 3 in a follow-on through the sale of 4.5 million shares at $28.50. Leerink Partners, Evercore ISI, Wells Fargo Securities, Chardan, H.C. Wainwright &...
17:21 , May 3, 2018 |  BC Extra  |  Financial News

uniQure raises $128.5M follow-on

Gene therapy company uniQure N.V. (NASDAQ:QURE) raised $128.5 million in a follow-on Thursday through the sale of 4.5 million shares at $28.50. Leerink Partners, Evercore ISI, Wells Fargo Securities, Chardan, H.C. Wainwright & Co. and...