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.
Ribopeutic Inc. has divulged nucleic acid splicing modulators (particularly, Huntingtin [HTT; HD] [mutant] and/or transcriptional activator Myb [c-Myb]) reported to be useful for the treatment of cancer and Huntington’s disease.
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.
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.
Having 35 copies of the CAG triplet in the gene that causes Huntington’s disease is not a problem. Inheriting 40 could be a sign that goes unnoticed for decades, until reaching 80. From there, the process accelerates and neural death occurs when reaching 150 repeats. Huntington’s disease neurodegeneration is not determined by what, but by how much, according to a study conducted at the Broad Institute.
The U.S. FDA has greenlit the first steps of Uniqure NV’s accelerated approval pathway for gene therapy AMT-130 to treat Huntington’s disease. The agency said data from the ongoing phase I/II studies compared to natural history external control are muscular enough to get the process going without having to dive into additional studies.
