Huntington’s disease

PTC Therapeutics has shared interim data from the 12-week portion of the PIVOT-HD Phase II study of PTC518 in Huntington’s disease (HD) patients. The study demonstrated dose-dependent lowering of Huntingtin (HTT) protein levels in peripheral blood cells, reaching a mean 30% reduction in mutant HTT levels at the 10mg dose level. In addition, PTC518 exposure […]

Currently, no effective cure or treatment for Huntington’s disease exists. To accelerate Huntington’s disease research and drug discovery, researchers need access to physiologically relevant human disease models that recapitulate patient phenotypes, in combination with more advanced methods to understand the underlying mechanisms of the disease. Download this new App Note from bit.bio to learn: How […]

Currently, no effective cure or treatment for Huntington’s disease exists. To accelerate Huntington’s disease research and drug discovery, researchers need access to physiologically relevant human disease models that recapitulate patient phenotypes, in combination with more advanced methods to understand the underlying mechanisms of the disease. In this App Note you will learn: How Charles River […]

News round-up for 21-25 August by DDW Editor Reece Armstrong A treatment for Monkeypox, spider venom for drug discovery and funding for international start-ups are some of the stories in this week’s news round-up. Covid researchers launch Monkeypox study   A new study investigating a potential treatment for people who have been diagnosed with monkeypox has […]

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France.  The National Agency for Safety of Medicines and Health Products (ANSM) gave the go-ahead to Bayer AG to conduct the clinical trial through its subsidiary BrainVectis to investigate its […]

Alchemab, a biotech company harnessing the power of naturally protective antibodies, has been awarded a UK Biomedical Catalyst early award grant from Innovate UK to support its collaboration with Medicines Discovery Catapult and the development of a novel disease-modifying antibody therapy for Huntington’s disease. Using its novel approach, Alchemab has identified a panel of antibodies […]