NYSNOBIO Awarded Prestigious ASAP Grant to Study Fundamental Causes of Parkinson’s Disease
San Francisco | June 1, 2021 - Together with Aligning Science Against Parkinson’s (ASAP), and the Michael J. Fox Foundation for Parkinson’s Research, NysnoBio and its founder Dr. Jennifer A. Johnston announce the launch of an innovative, multi-year study into the most common genetic causes of Parkinson’s Disease (PD).
Led by principal investigator Deniz Kirik, M.D., Ph.D., Lund University, Sweden, this $10 million global research effort will focus on patient cell lines associated with three validated genetic causes of PD: Parkin, α-synuclein, and Leucine Rich Repeat Kinase (PRKN, SNCA and LRRK2). This work will help unearth the role of these genes in PD pathobiology by developing innovative technologies to identify commonalities and differences in how each gene leads to PD. Among several novel techniques, this study will be the first to use diseased, patient-derived human pluripotent stem cells (iPSCs) transplanted into animals. This, combined with advanced data analytics and long-run observational data in real patients’ disease progression, will shed heretofore undiscoverable light on the underlying biology of this devastating disease.
Of hundreds of top applicant labs worldwide, Kirik, Johnston, and the rest of the team are proud to lead this study, and will provide new research models for the PD community by providing a new paradigm for in vivo PD research.
According to Jennifer A. Johnson, NysnoBio Founder and CEO:
“PRKN, SNCA and LRRK2 are established causes of early-onset PD, and our goal is to test and establish their relationships to sporadic PD. Just as the development of transplanted tumor cells transformed the field of oncology, our goal is to develop transplantation technology that will transform models of Parkinson’s Disease research. Building on our decades of work in genetic causes of PD, and leveraging our team’s cutting-edge capabilities in iPSC differentiation, xenografting, gene therapy and data analytics, we will chart a course through a new frontier for all Parkinson’s researchers.”
The four-year research plan will be led by the following team:
Dr. Deniz Kirik, Lead Investigator: Coordinate all research activities across all teams, and lead iPSCs generation and gene grafting.
Dr. Lachlan Thompson: Optimize cell transplantation activities and grafting procedures in order to ensure the surgical transplantation has predictable outcomes.
Dr. Clare Parish: Optimize pluripotent stem culturing for accurate in vitro differentiation into distinct cell types and in vivo verification and assessment.
Prof. Carolyn Sue: Harmonize iPSC lines and gene-edited clones. Also responsible for assessment of translational biomarkers, such as mitochondrial phenotyping, to ensure integrity of previous disease progression outcomes.
Prof. Glenda Halliday: Guide team effort in interpreting the various pathological hallmarks of human PD and how these hallmarks manifest in the iPSCs.
Dr. Jennifer A. Johnston: Contribute specific Parkin cell lines for the study. Along with nonprofit NysnoBioMetrix, will build and operate the data repository responsible for internal data analytics and ultimate sharing of this research platform with the global PD research community.
The study, In Vivo Approach to Elucidate the Pathobiology of Parkinson’s-associated Genes Using Human Diseased Neurons, commenced in January 2021 with the design and build-out of a secure, global data repository that combines artificial intelligence, data analytics, and privacy protections to fulfill ASAP’s mission of sharing its research findings in a responsible way with all PD researchers around the globe.
Contact NysnoBio to get involved or learn more:
info@nysnobio.com
