Though labeled as a rare disease, myotonic dystrophy type 1 (MD1) affects a surprisingly significant portion of the population, with estimates ranging from 1 in 8000 to as many as 1 in 2000 individuals. This condition unleashes a cascade of debilitating symptoms, rendering muscles unable to relax once contracted.
The disease often strikes early in childhood development, with symptoms surfacing as young as two years old. As it progresses, patients face a heartbreaking reality: the loss of mobility, speech, and even the ability to breathe. Life expectancy with MD1 is quite short.
Professor Ke Zhang and his team imagine a world where effective treatments for muscular diseases like DM1 exist, and patients no longer die from the disease. They aim to change the status quo by developing a safe and efficient oligonucleotide delivery technology, a gene regulation technology that treats the disease at the genetic source and addresses non-liver organs, reduces cost, and minimizes off-target effects.
The team’s work on a novel oligonucleotide delivery technology for muscular dystrophy diseases such as MD1 has earned them selection as one of the Fall 2023 Spark Fund awardees.
Oligonucleotide Delivery Technology for DM1 Treatment
Through the Northeastern spin-out company pacDNA Inc., Zhang and the team have been at the forefront of creating revolutionary treatments for muscular disease. Prior to their work on DM1, the team has been working to improve drug delivery technology for Duchenne Muscular Dystrophy (DMD) using the proprietary oligonucleotide enhancer technology, The Brushield™ platform.
The Brushield™ platform can rapidly generate potent clinical leads with reduced side effects and enhanced delivery to non-liver sites. In some preclinical models, Brushield™ reduces the dosage requirement by two orders of magnitude while suppressing side effects and immunogenicity, showing remarkable progress over previous treatments.
With the Spark Award, Zhang’s team is now applying this technology to develop a novel therapy for MD1.
“It is a devastating disease,” says Zhang, “so ideally, we’d like to remedy this and create a world where people do not die from DM1.”
Commercialization with the CRI
With no DM1 drugs currently available on the market, Zhang’s team is forging strategic partnerships within the pharmaceutical industry to bring the groundbreaking therapy to patients. They expect clinical trials to be relatively short, enabling the drug to go to market quickly and making it an attractive prospect for partnerships and licensing.
“The short clinical trials are appealing to the companies developing therapeutics, and the drug has the potential to be best-in-class,” says Zhang. “We have a real shot at taking this all the way to the patients.”
Leveraging Northeastern’s robust support ecosystem, including the Center for Research Innovation (CRI), Zhang’s team is deftly navigating the intricacies of business development, forging licensing agreements, and fostering connections with industry giants. Their efforts have garnered significant interest from prominent pharmaceutical players, propelling their vision closer to a reality for patients.
The Spark Fund Award
Northeastern CRI’s Spark Fund Award partly funds this technology, as the team was selected as one of the Fall 2023 Spark Fund awardees. Recognizing the potential of his team’s technology, the Spark Fund has provided vital resources, propelling their journey from concept to commercialization.
Zhang attributes Northeastern’s strong support of entrepreneurship as a key to his team’s success, empowering them to bridge the gap between lab innovation and tangible patient impact.
“The entire process has been very exciting and beneficial to us,” says Zhang. “The judges personally helped us move our ideas forward.”
The team was also a recipient of The Spark Award in December 2022.
Learn More about the Spark Fund
Learn more about Zhang’s research and the other Fall Spark Award grantees here.
Written by Elizabeth Creason