NeuroQure, a pioneering biotech startup specializing in gene therapy for neurocognitive disorders, has reached a pivotal milestone in the development of its first commercial product. The company has acquired the exclusive rights to license a patent portfolio from the UC Irvine Center for Autism Research and Translation (CART). This intellectual property, developed over 7 years with an investment of over $14 million, forms the cornerstone for the creation of precise diagnostic tools for autism spectrum disorder (ASD) and related intellectual disabilities.
The patented portfolio was developed by Dr. John Jay Gargus, MD. Ph.D. and his team during his tenure as Founding Director of CART. Dr. Gargus, a highly respected and world-renowned geneticist who has dedicated his career to advancing scientific research on neurological disorders, currently serves as NeuroQure’s Founding Scientist.
“The exclusive license of patents from UC Irvine has the potential for NeuroQure to bring medical diagnostic tools validated with top-tier publications and issued patents as a functional biomarker diagnostic for the broad ASD spectrum from the lab to market, enabling early intervention and improving children’s lives,” said David Justus, NeuroQure’s Founder and CEO.
Despite the prevalence of ASD affecting 1 in every 36 US children, a scientifically validated method for early diagnosis has been notably absent.
“The sooner your child receives a diagnosis, the sooner your child can start benefitting from essential early intervention and medical attention,” said Lisa Ackerman, founder of The Autism Community in Action (TACA). “Autism is treatable and the first step in addressing it begins with an accurate diagnosis.”
The Autism Community in Action (TACA) is a national nonprofit organization with programs and volunteers spanning over 41 states and is growing to support an estimated 1,500 families monthly.
Research consistently shows the earlier treatment such as behavioral therapy begins, the better the outcome for the child. Current methods either deliver results too late or prove ineffective, creating a pressing need in the community for a diagnostic tool capable of identifying ASD at the earliest age possible.
Committed to proactively addressing the critical gap in existing diagnostic technologies, NeuroQure’s mission responds to the urgency for a timely and accurate diagnosis.
NeuroQure’s acquisition positions the company to offer help to families within a few weeks of birth, dramatically shortening what is typically a long and painful, 5 plus year, diagnostic odyssey to a diagnosis of ASD. This is particularly acute in families with a prior affected child, where the risk is twenty times greater than that of the rest of the population.
Advancing the Science
“As part of the Autism Sequencing Consortium, CART contributed to the identification of more than 185 genes strongly implicated in Autism, but the heterogeneity of ASD and the complexity of that genetic signal still renders a polygenic risk score based on DNA sequencing alone of little practical utility, despite the fact that a number of labs are beginning to offer variations of such sequencing diagnostics,” said Dr. Gargus, NeuroQure’s Founding Scientist.
As detailed in the NIH’s National Library of Medicine article linked here PubMed, the patented calcium signaling technique enables early detection of ASD in newborns enabling early intervention with behavioral therapy and other medically proven therapies. This technique, as detailed in the several published papers, demonstrates that it is possible to use a skin biopsy, or discarded foreskin, and a functional biophysical assay to determine a newborn’s high probability of ASD.
Revolutionizing the Diagnostic Landscape
“An accurate diagnosis is the first step in treating those affected by Autism. We are honored to bring the groundbreaking discoveries made by Dr. Gargus and his team, from the lab to families in need, potentially revolutionizing the diagnostic landscape for ASD. The strategic acquisition of UCI’s CART patent portfolio places us at the forefront of innovation, offering parents and physicians a timely and accurate diagnosis that allows for early intervention, and improved outcomes to potentially altering the trajectory of a child’s life,” said Justus.
Positioned for Innovation
In securing the exclusive license of UC Irvine’s CART patents, NeuroQure is poised to revolutionize the landscape of Autism diagnostics. The company’s dedication to addressing the gap in current technologies underscores its commitment to transform the lives of children and families affected by ASD.
