PTC Therapeutics Inc. CEO Matthew Klein said the firm “achieved all we set out to [achieve] in phase II” with PTC-518 in Huntington’s disease (HD), but it wasn’t enough to excite Wall Street, as some questioned whether the level of associations between trial findings and efficacy would appease the U.S. FDA. Shares (NASDAQ:PTCT) ended May 5 at $40.65, down $9.30, or 18.6%. Warren, N.J.-based PTC rolled out the most recent data from the Pivot-HD effort in stage 2 and stage 3 HD with PTC-518, an oral, centrally as well as peripherally distributed huntingtin (HTT) pre-mRNA splicing modifier.

BDNF is the brain’s most abundant neurotrophic factor, playing a key role in neuronal survival and synaptic plasticity through the activation of the transcription factor CREB, which is essential for driving beneficial effects in neurons. CREB is downregulated in Parkinson’s disease, Huntington’s disease, frontotemporal dementia, Alzheimer’s disease and other neurodegenerative conditions.

Polyglutamine (polyQ) diseases, a group of dominantly inherited CNS disorders, are caused by an abnormal expansion of cytosine-adenine-guanine repeats (usually over 35-40 repeats). PolyQ diseases, including spinocerebellar ataxia and Huntington’s disease, cause brain neurodegeneration, leading to progressive motor and often cognitive signs.

Researchers from Life Edit Therapeutics Inc. recently reported preclinical data on the application of their gene editing technology Life Edit CRISPR system to Huntington’s disease (HD).

Trimtech Therapeutics closed a £25 million (US$31 million) oversubscribed seed funding round to advance its targeted protein degradation treatments for neurodegenerative and inflammatory diseases.

Trimtech Therapeutics closed a £25 million (US$31 million) oversubscribed seed funding round to advance its targeted protein degradation treatments for neurodegenerative and inflammatory diseases.

Harness Therapeutics Ltd. has raised fresh financing to further develop its technology for upregulating the translation of mRNA into proteins, and in particular to take on a previously undruggable target in Huntington’s disease.

Atalanta Therapeutics Inc. has completed a $97 million series B financing to support its investigational RNA interference (RNAi) therapies for KCNT1-related epilepsy and Huntington’s disease toward IND filings this year and through clinical proof of concept.