NysnoBio Awarded Michael J. Fox Foundation Grant to Advance Parkin Gene Therapy

  • The funding will support NB001 Parkin gene replacement therapy IND-enabling studies
  • Initial indication in a substantial, genetically defined Parkinson’s population: Parkin-PD
  • Parkin-PD mutations are the most penetrant, common cause of early-onset PD

SAN FRANCISCO, July 17, 2023 (GLOBE NEWSWIRE) -- NysnoBio, a biopharmaceutical company pioneering the development of Parkin gene replacement therapy for patients with severe diseases, today announced it has received a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to assist the advancement of the company’s lead Parkin gene replacement therapy candidate, NB001. The funding will support product manufacturing to enable IND safety studies in preparation for human clinical trials evaluating NB001 in Parkinson’s Disease (PD) patients with bi-allelic loss-of-function mutations in the Parkin gene, or Parkin-PD.

Parkinson’s Disease is characterized by the loss of dopamine neurons between the substantia nigra and striatum. The Parkin gene is a key regulator of dopamine neuronal health through its established role to orchestrate the removal of damaged mitochondria. Parkin-PD patients are unable to clear damaged mitochondria, resulting in PD at an age of onset younger than 35 years old, deeply affecting their personal and professional lives. NB001 Parkin gene replacement therapy aims to restore dopaminergic function and give these patients the prime of their lives back. Demonstration of therapeutic benefit in this focused patient population will allow expansion into other defined PD patient populations in need of motor improvement.

NysnoBio and its team of world-renowned Parkin experts have utilized an efficient and cost-effective strategy to drive NB001 towards the clinic. NB001 is based on a clinically validated AAV construct with a well-established safety profile and is delivered via a common procedure that directly targets multiple cell types. In addition, NysnoBio is developing innovative clinical biomarkers to optimize clinical development. The new funding by the MJFF will propel manufacturing and IND-enabling studies to validate biomarkers of efficacy, demonstrate safety and establish dosing parameters. The Company expects to file an IND within 18 months.

“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,” said Jennifer A. Johnston, PhD., Founder and CEO of NysnoBio. “We are thrilled with MJFF’s continued funding 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.”

Shalini Padmanabhan, PhD., MJFF Vice President, Discovery & Translational Research, remarked, “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.”

About NysnoBio
NysnoBio is a biopharmaceutical company developing a portfolio of gene therapy product candidates designed to address critical unmet medical needs in neurodegenerative diseases. NysnoBio pursues highly validated therapeutic targets based on human genetics, pathology, and patient phenotype. In parallel, NysnoBio’s innovative clinical biomarker programs provide robust markers of efficacy in the clinic. For additional information, please visit: www.nysnobio.com.

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