MJFF grant to NysnoBio will advance gene therapy for Parkin-PD

Treatment aims to help Parkinson's patients with PRKN gene mutation

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

Share this article:

Share article via email
A hand holds a coin alongside dollar signs and stacks of paper money.

A new grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) will help NysnoBio to advance the development of its experimental gene therapy for Parkinson’s patients with loss-of-function mutations in the Parkin gene — officially the PRKN gene — commonly known as Parkin-PD.

The award will fund preclinical studies of the gene therapy candidate NB001, which are expected to support an investigational new drug application, or IND. That formal request to U.S. regulatory authorities seeks permission to begin clinical testing in people.

NysnoBio expects to submit an IND within the next 18 months, according to a company press release.

“Advancing novel therapies is core to the mission of MJFF. … We are thrilled to continue funding NysnoBio to support their pioneering efforts in delivering this important new approach for PD [Parkinson’s disease],” said Shalini Padmanabhan, PhD, vice president of discovery and translational research at the MJFF.

Jennifer Johnston, PhD, NysnoBio’s founder and CEO, said the company also is “thrilled” with the award, and hopes to use the “funding to move closer to changing the course of disease for … young genetic patients, and ultimately for many [Parkinson’s] patients.”

Recommended Reading
An illustration highlights the words

Dosing Begins in Phase 1 Clinical Trial of AAV-GAD Gene Therapy

MJFF grant will fund preclinical studies of NB001

The PRKN gene provides instructions for making a protein called parkin, which is known to play a critical role in maintaining the health of dopaminergic neurons — the nerve cells that make the chemical messenger dopamine. All forms of Parkinson’s are characterized by the death and dysfunction of these neurons.

Mutations in the PRKN gene, which interfere with this protein’s function, are the most common cause of early-onset Parkinson’s. Patients with mutations in this gene often develop the disease before age 35.

“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,” Padmanabhan said.

NysnoBio’s NB001 is a gene therapy that basically aims to deliver a healthy copy of the PRKN gene to the body’s cells. By doing so, it would allowing these cells to produce a functional version of parkin protein.

The therapy works using an engineered virus called adeno-associated virus or AAV, which has been commonly used in gene therapies. It is easy to engineer in a lab and does not cause illness in people.

NB001 is initially being developed to treat patients with bi-allelic loss-of-function mutations in the PRKN gene — in other words, individuals whose specific mutations prevent any parkin protein from being produced at all.

The preclinical studies that will be funded by the MJFF grant “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” Parkinson’s, Johnston said.

The studies funded here by MJFF will advance the first Parkin-based therapeutic through the required studies needed in preparation for human clinical trials.

Preclinical work also will be undertaken to identify biomarkers that can be used in early clinical testing to check whether the therapy appears to be working as intended.

“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,” Johnston said.

“The studies funded here by MJFF will advance the first Parkin-based therapeutic through the required studies needed in preparation for human clinical trials,” Johnston added.

The amount of the MJFF grant was not disclosed.