gILLIAn P. BATES, FMEDSCI, PHD, FRS

Professor of Molecular Neuroscience

Co-Director Huntington’s Disease Centre University College London (UCL) Institute of Neurology

Fellow, Royal Society

Dr. Gillian Bates was a member of the HDF’s Venezuela Project that identified the causative gene for HD in 1993. She went on to develop the first mouse model of HD in 1996. She currently uses mouse models to understand the earliest events that cause HD, validate approaches to treating this disease and steer drug development programs.

In 2013 Dr. Bates demonstrated that, when carrying the HD mutation, the HD gene produces a small, truncated huntingtin protein (exon 1 HTT) along with the complete HTT protein. This exon 1 HTT is known to readily form the aggregates that are found in HD patient brain tissue and to cause brain cell dysfunction. Finding approaches that will decrease exon 1 HTT levels has become an important goal in developing treatments that will delay the onset or slow the progression of HD. Dr. Bates received the HDF’s Leslie Gehry Brenner Prize for Innovation in Science in 2012.

Neurons vulnerable in HD

C. FRAnk BEnnETT,

PHD

Chief Scientific Officer

Ionis Pharmaceuticals

2019 Breakthrough Prize in Life Sciences

Dr. C. Frank Bennett is one of the founding members of Ionis Pharmaceuticals, leading much of their preclinical antisense drug discovery and antisense technology research. He has been involved in the development of antisense oligonucleotides as therapeutic agents, including research on the application of oligonucleotides for inflammatory, neurodegenerative diseases and cancer, oligonucleotide delivery, pharmacokinetics and medicinal chemistry. He has published more than 230 papers in the field of antisense research and development and has more than 175 issued US patents.

Dr. Bennett collaborated with Adrian Krainer, of Cold Spring Harbor Laboratory, to develop Spinraza, the first effective therapy for spinal muscular atrophy (SMA). SMA is a progressive neurodegenerative disease and the number one genetic cause of death for infants. It affects the motor nerve cells in the spinal cord and impacts the muscles used for activities such as breathing, eating, crawling, and walking. Since Spinraza’s approval in 2016, more than 11,000 people have been treated with it worldwide.

Prior to joining Ionis, Dr. Bennett was Associate Senior Investigator in the Department of Molecular Pharmacology at SmithKline and French Laboratories, currently GSK. He received HDF’s 2018 Leslie Gehry Brenner Prize for Innovation in Science.