NysnoBio Awarded Michael J. Fox Foundation Grant for Advancement of Parkin Gene Therapy to IND
San Francisco | July 17, 2023 – NysnoBio is pleased to announce additional funding from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance their Parkin gene therapy development candidate NB001 towards IND status. This funding will support NB001 product manufacturing to enable the IND safety studies in preparation for human clinical trials.
This research will take place across 2023 and 2024:
Phase 1 will validate biomarkers of efficacy in the Parkin KO rodent models and generate NB001 for IND-enabling safety testing.
Phase 2 will establish dosing parameters and initiate the IND-enabling safety study required to achieve IND status for human clinical trials.
The combined outcome from Phases 1 and 2 is critical not just for this study, but for the broader community of PD researchers. Our studies will demonstrate the safety of Parkin gene therapy and open new avenues of therapeutic development to tackle this challenging disease.
Potential Impacts for Parkinson’s Disease (PD) Diagnosis and Treatment
Parkin-PD is the most potent known genetic form of PD, with almost 100% penetrance. In addition, the patients affected are typically younger than 35 years old, with significant disability in the prime years of their life. This therapy has the potential to restore dopaminergic function alleviating the movement disorder for these patients and restoring a high quality of life.
If successful, the ability to restore health to dopamine neurons can be applied to additional PD patient populations. All PD patients have dopamine neuron loss, regardless of genetic status, thus many could benefit from this therapy once success is demonstrated in this focused, genetic population.
Says NysnoBio Founder and CEO, Jennifer A. Johnston, PhD:
“These studies will help fill critical gaps in the PD therapeutic landscape with the advancement of the first therapeutic for the Parkin protein—one of the most highly validated genes related to PD. We are honored to be working with MJFF to move closer to changing the course of disease for these young genetic patients, and ultimately for many PD patients.”
Over the last 20 years, the NysnoBio team has pioneered the basic biochemistry and therapeutic potential for the Parkin gene. We already know that Parkin is effective for neuroprotection and brain health. The studies funded here by MJFF will advance the first Parkin-based therapeutic through the required studies needed in preparation for human clinical trials.
Says MJFF Vice President, Shalini Padmanabhan, PhD:
“Advancing novel therapies is core to the mission of MJFF. Parkin is a highly validated genetic target relevant to many in the PD community with younger disease onset, for whom new treatments could offer significant benefit. We are thrilled to continue funding NysnoBio to support their pioneering efforts in delivering this important new approach for PD.”
Read today’s press release here.
Contact NysnoBio to get involved or learn more:
info@nysnobio.com
