NysnoBio Awarded Michael J. Fox Foundation Grant
San Francisco | May 1, 2021 – Dr. Deniz Kirik (Lund University), Dr. Matt Goldberg (University of Alabama at Birmingham), and Dr. Jennifer Johnston (NsynoBio and NysnoBioMetriX) proudly announce continued funding from The Michael J. Fox Foundation (MJFF) for work to benefit the broader Parkinson’s community. This new study, launched March 2021, will work to identify the unexplored similarities between hereditary and idiopathic Parkinson’s Disease (PD) and will test the ability of the Parkin gene to provide therapeutic benefits in idiopathic PD.
The study, Efficacy of PRKN Gene Delivery toward Mitigation of Alpha-synuclein-induced Degeneration, will explore how the delivery of functional Parkin genes can prevent the buildup of the alpha-synuclein protein that is the hallmark of both hereditary and non-hereditary forms of PD.
This new research will take place across two phases in 2021 and 2022:
Phase 1 will establish robust assays to measure the ability of our gene therapy approach to deliver the Parkin genes to target neurological tissues in live animals.
Phase 2 will deploy these Phase 1 assays to analyze the ability of the Parkin gene to produce functional Parkin protein in rats with genetic forms of PD. We will then be able to measure the impact of Parkin gene delivery as a path to neuronal recovery.
The combined outcome from Phase 1 and Phase 2 is critical not just for our study, but for the broader community of PD researchers. Our findings will also determine the extent to which Parkin protein is an effective tool to fight a broad range of PD pathologies and degeneration of central nervous system tissues.
Potential Impacts for PD Diagnosis and Treatment
Biomarker endpoints for Parkin activity that provide definitive results in animals lead to novel clinical translational biomarkers which allow the assessment of therapies in human clinical trials. If successful, the biomarker assays developed in Phase 1 of this grant will transform the field of Parkinson’s Disease from clinical trials to diagnosis to treatment. This transformation may create new pathways to a cure for PD. One thing is certain: the physiological readouts developed in P1 will have broad applicability across a large range of neurological disorders including Huntington’s, ALS, Alzheimer’s, and other devastating conditions.
Next Steps for the NysnoBio Team
The project began in 2021 and will share key data with the global PD research community, in keeping with the MJFF's commitment to open data access.
Says NysnoBio Founder and CEO Jennifer A. Johnston:
“These studies will help fill critical gaps in understanding why and how Parkin gene mutations cause Young Onset Parkinson’s Disease, and the extent to which Parkin Gene replacement can affect the course of the disease.
Over the last 20 years, the NysnoBio team has pioneered the basic biochemistry and potential for gene therapy using the Parkin gene. We already know that Parkin is effective for neuroprotection and brain health. But the studies funded here by The Michael J. Fox Foundation will address the role of Parkin therapeutic benefits as related to two other validated pathways to Parkinson’s: alpha-synuclein and PINK1.
As we build our understanding of the broader applications of Parkin gene therapy, and work together to deliver it to affected patients, our goal is to eliminate the helpless, inexorable decline causing human suffering.”
Says Michael J. Fox Foundation CEO Dr. Todd Sherer:
“People with Parkinson’s urgently need better options to slow or stop disease progression. The Parkin pathway is a validated contributor to PD, and this project is a promising step in pursuit of therapies against that dysfunction. The potential for impact against the hallmark pathology of Parkinson’s—alpha-synuclein aggregation, and thereby this therapy’s possible widespread applicability—is encouraging. We are proud to support this work and, with the patient community, look forward to reviewing its outcomes.”
We are looking forward to sharing results of Phase 1 in March 2022, and to using the findings across the entire study to deepen our collaboration with the entire PD and neurological and scientific communities.
Contact NysnoBio to get involved or learn more:
info@nysnobio.com
