2019 FAST Summit on Angelman Syndrome - ASO Therapy for AS - Dr. Frank Bennett, Ionis Pharmaceutical

Foundation for Angelman Syndrome Therapeutics presents "The development of an antisense oligonucleotide therapy for Angelman syndrome.
" 2019 FAST Science Summit - Frank Bennett, Ph.D., Ionis Pharmaceuticals
. Information presented at the 2019 FAST Summit and Gala, December 6, 2019.

The development of an antisense oligonucleotide therapy for Angelman syndrome.
Identifying an antisense drug designed to un-silence the paternal UBE3A gene, resulting
in increased production of the missing UBE3A protein in neurons, as a potential therapy for
Angelman syndrome. Antisense oligonucleotides (ASOs), are a drug discovery platform
designed to bind to RNA, the intermediate molecule that translates information from DNA to
proteins, complementing small molecules and protein based drugs.

BIOGRAPHY
C. Frank Bennett, Ph.D., Senior Vice President, Research
Dr. Bennett is Senior Vice President of Research at Ionis Pharmaceuticals and one of the founding members of the
company. He is responsible for preclinical antisense drug discovery and antisense technology research. Dr. Bennett
is also the franchise leader for neurological programs at Ionis. 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.
Dr. Bennet is a co-recipient of the 2019 Breakthrough Prize in Life Sciences for his contributions to the discovery and
development of SPINRAZA® (nusinersen) and the 2018 Hereditary Disease Foundation’s (HDF) Leslie Gehry Brenner
Prize for Innovation in Science for his leadership and continued commitment to developing antisense therapies for
Huntington’s disease (HD).
Dr. Bennett has published more than 200 papers in the field of antisense research and development and has more
than 175 issued U.S. patents.
Prior to joining Ionis, Dr. Bennett was associate senior investigator in the Department of Molecular Pharmacology at
SmithKline and French Laboratories, currently, GlaxoSmithKline.
He received his Ph.D. in Pharmacology from Baylor College of Medicine, Houston, Texas and his B.S. degree in
Pharmacy from the University of New Mexico, Albuquerque, New Mexico. He performed his postdoctoral research in
the Department of Molecular Pharmacology at SmithKline and French Laboratories. Dr. Bennett serves on the Advisory
Board for the Experimental Therapeutics Centre in Singapore and the Hereditary Disease Foundation.

[kingofcatskingofcats]

What ever happened to wvs mice sanctions