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 Kd of 33 nM...
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 expressing...
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 pig...
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...
22:44 , Apr 10, 2018 |  BC Innovations  |  Distillery Techniques

Disease models

TECHNOLOGY: Animal models Pigs expressing mutant HTT could be useful for screening therapies to treat HD. In pigs, knock-in of an HTT mutant containing a 150-residue polyglutamine repeat recapitulated the respiratory and motor function defects, poor...
22:32 , Mar 30, 2018 |  BC Extra  |  Preclinical News

Pig model of Huntington's disease mimics human disease

In a paper published in Cell, an international team of researchers developed a knock-in pig model of Huntington's disease that recapitulates neurological and respiratory symptoms of the disease in humans. Huntington's disease is caused by protein...
17:14 , Mar 9, 2018 |  BC Week In Review  |  Clinical News

Ionis' IONIS-HTTRx reduces mutant HTT protein in Phase I/II for HD

Ionis Pharmaceuticals Inc. (NASDAQ:IONS) said IONIS-HTTRx (RG6042) significantly reduced mutant huntingtin (HTT) protein in the CSF of patients with early stage Huntington’s disease vs. placebo in a Phase I/II trial. Ionis said Roche (SIX:ROG; OTCQX:RHHBY)...
20:31 , Mar 2, 2018 |  BC Extra  |  Clinical News

Ionis' Huntington's antisense headed for pivotal trial

Ionis Pharmaceuticals Inc. (NASDAQ:IONS) said IONIS-HTTRx (RG6042) significantly reduced mutant huntingtin (HTT) protein in the CSF of patients with early stage Huntington’s disease vs. placebo in a Phase I/II trial. Ionis said Roche (SIX:ROG; OTCQX:RHHBY)...
00:56 , Feb 27, 2018 |  BC Extra  |  Financial News

CRISPR stocks gain

CRISPR companies added $458 million in market cap on Monday in apparent reaction to two articles published in the past two days. CRISPR Therapeutics AG (NASDAQ:CRSP) added $5.30 (12%) to $48.34, Intellia Therapeutics Inc. (NASDAQ:NTLA) gained...