Parkin Gene Therapy: A Diamond in the Rough
The lack of tailored treatments for sufferers of Young Onset Parkinson’s Disease (YOPD) has destroyed countless lives. The good news: recent advances at NysnoBio may point toward a gene-based cure for YOPD, as well as late-Onset Parkinson’s and other challenging CNS disorders.
To guide this promising new research, NysnoBio has assembled an unmatched team of scientific advisors to advance the gene science, clarify viral vector deliveries, design safe and effective surgical techniques, and develop the testing and monitoring regimes needed to bring these new medicines to market.
Human beings who are missing both copies of the Parkin gene are destined to be diagnosed with Parkinson’s Disease between the ages of 25 and 40. These patients suffer from profound loss of dopamine neurons. NysnoBio’s gene therapy program is designed to deliver meaningful clinical validation within this focused patient population. Our ultimate goal is to translate this positive clinical outcome to all Parkinson’s Disease patients.
Parkin is a highly validated E3 ubiquitin ligase with demonstrated therapeutic potential in neurology, and we at NysnoBio have dedicated our careers to studying it. Neuroprotection using Parkin gene therapy is already validated through a legion of animal studies using established model systems. There is also a mountain of data validating the efficacy of the ability of the Parkin protein to protect against cellular stress. For these reasons we are able to quickly translate from preclinical validation to our current status of IND-enabling studies.
Our founder was the first to publish a high-resolution crystal structure showing critical features of how the Parkin enzyme works.
NysnoBio has unparalleled depth of expertise to deliver on the promise of the therapeutic potential of the Parkin protein. Our team members:
were the first to identify the Parkin gene linked to Parkinsons’s Disease,
were first to demonstrate Parkin is an E3 ubiquitin ligase,
were first to publish a crystal structure demonstrating key aspects of enzymology,
have greater than 20 years expertise in gene therapy using AAV,
and have clinical neuroimaging expertise, ensuring definitive results in the clinic.
We made the discovery. Our goal is to make the cure. ™