This week, the FDA released a proposal to streamline the way they oversee the evaluation of protocols related to gene therapy. Essentially the US government has recognized that gene therapies are an important emerging class of medicines, and they’re trying to re-vamp the administrative process so that it’s shorter and less repetitive. Several NIH and FDA-associated directors have written about this publicly and explain how the process will change moving forward. This includes an article in the New England Journal of Medicine and a statement by NIH Director Dr. Francis Collins, a long-time supporter of the HD community and a member of the collaborative team that identified the HD gene in 1993, as well as a blog post by NIH Chief of Staff Dr. Carrie D. Wolinetz. It’s exciting to hear that the FDA is taking seriously the promise of gene therapy, and will work to streamline the process – we have hopes that this will speed the approval of effective new therapies. The proposal itself is very long and intricate, but anyone from the public can submit input on it until mid-October.

Milton Wexler Celebration of Life: Boston HD Conference

The biennial research conference held by the Hereditary Disease Foundation in Boston, Massachusetts, was a great success, bringing together scientists from all over the world to discuss the latest in HD and other rare disease research. Topics included huntingtin-lowering, large-scale analysis of human and model data to identify new drug targets, determining the shape of RNA and proteins and designing drugs to fit, animal models large and small, delivery of drugs to the brain, and much, much more. It was an inspiring and hopeful gathering of scientists that will boost ongoing worldwide collaborations to discover new therapies for HD.