Dr. David Segal is a professor at the University of California, Davis, in the Departments of Biochemistry and Molecular Medicine, Pharmacology, Genome Center, and MIND institute. Dr. Segal’s research focuses on engineering custom DNA-binding proteins and their application toward improving public health. This technology uses targetable nucleases and/or artificial transcription factors to make precise changes to the DNA of living cells or activate/repress specific genes of interest. Dr. Segal is a member of the FAST Integrative Research Environment Initiative (FIRE), a consortium of scientists, with precise expertise in Angelman Syndrome (AS), who work collaboratively to identify, characterize and implement new therapeutics for the treatment and ultimate cure of AS. This initiative was launched in 2013, includes five laboratories spanning four universities and is funded in full by donations made to FAST. To date, FAST’s investment in the FIRE Initiative stands at more than $3.4 million. The results of this collaboration have been remarkable and the FIRE team is now working with more than six pharmaceutical and biotechnology companies to prepare for and test potential therapeutics for human clinical trial. The focus of Dr. Segal’s research in Angelman Syndrome is to activate the paternal UBE3A gene through deactivation of the UBE3A-ATS (antisense transcript).
At the last FAST Summit, Dr. Segal discussed a compound called TAT-S1, that was found to cross the blood brain barrier after peripheral injection, and distribute widely throughout the brain, ultimately turning on UBE3A in the AS mice. This is incredibly promising, as to date getting therapeutics into the brain diffusely has proven to be very difficult. When Dr. Segal’s lab evaluated this strategy in live mice, with fluorescent imaging, the activity was only seen to last ~24 hours, so further evaluation of mechanisms for chronic therapeutic delivery are needed.
The focus of Dr. Segal’s presentation was to discuss the progress in designing epigenetic regulators for persistent UBE3A activation. He focused on 3 aspects of his research: 1) Epigenetics, 2) In situ production of therapeutics, 3) The benefit of using rat models in AS.
Since Dr. Segal’s presentation at the last FAST Summit, two pharmaceutical companies have expressed interest in his approach to treating AS.
To read about how this FAST-supported research has led to $1.1 Million in additional funding, click here…