NEW YORK – Yale School of Medicine on Tuesday said it received roughly $40 million in grant funding from the National Institutes of Health to support development of CRISPR-based gene-editing therapies for Angelman and HIST1H1E syndromes, two genetic brain diseases.
The research to develop gene-editing technology capable of reaching the human brain will be led by genetics, pediatrics, neuroscience, and neurosurgery professors at Yale School of Medicine with a co-principal investigator at Rush University and participation from the Foundation for Angelman Syndrome Therapeutics and the HIST1H1E foundation.
The first $26.5 million grant will fund an initial phase of the project focused on preclinical and toxicology studies in animal models and human brain organoids. If that phase is successful, the NIH will provide an additional $13 million to fund clinical trials.
"Over the past few years, we have been working on development of this technology," said Jiangbing Zhou, a professor of neurosurgery and biomedical engineering at Yale School of Medicine and a co-leader on the project, in a statement. "I am thrilled about the opportunity to bring it to the clinical bedside."
The gene-editing technology, known as the Stimuli-responsive Traceless Engineering Platform, or STEP, is administered to patients through a spinal tap, so that ribonucleoproteins (RNPs) can penetrate the brain and edit neuronal cells. After entering the cells, RNPs degrade, which researchers say limits the risk of off-target effects.
The therapies for Angelman and HIST1H1E syndromes are a proof of concept for the STEP technology, which researchers hope will be applicable to other neurogenetic disorders.