Viruses and the brain JAN25 2015 I am certain most of you would have heard that some of the therapies being developed for HD and other neurodegenerative indications involve the use of viruses to deliver a gene therapy agent. This is true in the case of HD, we are working…

Last week, CHDI hosted a stem cell meeting in Princeton, NJ, chaired by Thomas Vogt (CHDI’s new VP for Discovery & Systems Biology), and myself. We hosted several eminent scientists working on the use of stem cells for understanding HD pathogenesis, and identifying new mechanisms for potential therapeutic treatments. The…

Human genetics research sheds new hope in modulating the progression of HD MAR23 2014 It is not unfair to say that almost the entire field of therapeutic development for the treatment of HD is founded on the learnings that stem from the cloning of the gene causative of HD, the…

Pfizer’s PDE10 inhibitor enters clinical trials for HD JUN16 2013 This is a good year for HD, and for all working to develop medicines to treat all those affected. For the first time in CHDI’s existence, a clinical trial with a new drug entity will start this month. The collaboration…

As I have described before, the age of molecular therapies for the treatment of HD is around the corner. But a critical component of the drug development process is still lacking: the identification of Htt-dependent biomarkers. Why is this important? Imagine what will happen over the next 1-3 years. Several…

Huntingtin targeting therapeutics are entering the clinical realm… after many years of work, 4 big players in the biotech/pharmaceutical industry are planning clinical trials in HD. Recently you might have heard about Roche, the Swiss pharmaceutical giant, acquiring the rights to develop ISIS’s lead antisense molecule to lower HTT expression…

A recent manuscript by a Swedish group (Ji et al., The Lancet, 2012) has revealed that the incidence of cancers amongst individuals bearing mutant expanded polyglutamine genes (and particularly Huntingtin expansions) is significantly lower than in the general population, even after adjusting for age. This study is a continuation of…