On November 9, 2021 EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, reported that it has entered into a research collaboration with the University of Wisconsin–Madison as part of the company’s effort of translating proprietary LEAPER RNA editing technology into in vivo therapies (Press release, EdiGene, NOV 9, 2021, View Source [SID1234594945]).
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Under the partnership, EdiGene’s U.S.-based R&D Center will work with the David Gamm Laboratory at the University of Wisconsin-Madison to evaluate the pharmacological property of LEAPER RNA base editing candidates targeting specific genetic diseases.
"EdiGene’s U.S. R&D Center has built up its capabilities with an exceptional team of talented scientists who are advancing the company’s pipeline both within our own labs and through external partnerships," said Bo Zhang, Ph.D., Head of EdiGene’s U.S. subsidiary. "Dr. Gamm’s knowledge and insights are a tremendous asset in this effort."
"This collaboration allows us to leverage our expertise and experience to help translate EdiGene’s RNA base editing technology into transformative medicines that can benefit patients with genetic diseases who today have limited or no therapeutic options," said David Gamm, MD, Ph.D., Principal Investigator of the research, Professor of Ophthalmology and Visual Sciences, and Director of the McPherson Eye Research Institute at the University of Wisconsin–Madison.
LEAPER (Leveraging endogenous ADAR for programmable editing of RNA) employs short engineered ADAR-recruiting RNAs (arRNAs) to recruit native ADAR enzymes to change specific adenosine to inosine. LEAPER is a robust, precise, and efficient RNA editing technology uniquely suited for in vivo therapies with broad therapeutic applicability. It is developed by Professor Wensheng Wei’s lab at Peking University. Professor Wensheng Wei is the Scientific Founder of EdiGene.
"Earlier this year, we announced the first patient enrolled in multicenter Phase I clinical study of our investigational ex vivo gene-editing hematopoietic stem cell therapy ET-01. This collaboration furthers our efforts in RNA editing and in vivo gene-editing therapies," said Dong Wei, Ph.D., CEO of EdiGene. "Along with the expansion of our U.S. R&D Center, such effort will help accelerate the translation of our proprietary gene-editing technology for patients in need worldwide."
About the David Gamm Laboratory
The Gamm laboratory was established at the University of Wisconsin-Madison in 2003 to advance the use of human pluripotent stem cells (hPSCs) in the study and treatment of retinal degenerative diseases. To facilitate these efforts, the Gamm lab developed the first hPSC-based 3D retinal organoid culture method, which has since yielded key insights into mechanisms of early human retinal development. Moreover, their studies have established the authenticity of hPSC-derived retinal progeny, including photoreceptor cells (rods and cones), retinal pigmented epithelium (RPE) cells, and neural retinal tissue. Lastly, they were pioneers in the use of patient-specific and gene-modified iPSCs to model retinal disorders and to test therapeutic strategies, and have advanced efforts to adapt this technology for human use.