Northeastern Center for Research Innovation (CRI) recently executed a licensing agreement with pacDNA, a Northeastern faculty-founded spinout company. This agreement highlights CRI’s role in fostering innovation and protecting and licensing technology for university researchers.
As part of the university’s offerings, the CRI offers express licenses to support university IP-based startups. Throughout this process, the team worked closely with pacDNA to understand their needs and secure a license that supports their technology development and commercialization goals.
“We hope our support for the startup will aid the translation of this IP into therapeutics for patients,” says Vaibhav Saini, CRI Commercialization Manager for pacDNA.
Oligonucleotide Delivery Technology
pacDNA is a therapeutics company that works with a class of drug molecules called oligonucleotides, which are short strands of DNA or RNA. These molecules have the potential to address challenging conditions, including rare genetic disorders and cancer. Delivering these molecules to the correct tissues and cells has been challenging, but pacDNA’s Brushield™ technology addresses this problem.
The Brushield™ platform is a proprietary oligonucleotide enhancer technology. The 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. This shows remarkable progress over previous treatments.
Oligonucleotides for DM1 Treatment
pacDNA is initially targeting Myotonic dystrophy type 1 (MD1), a condition that causes progressive muscle dysfunction. MD1 is classified as a rare disease and is estimated to affect anywhere from 1 in 8000 to 1 in 2000 individuals, depending on the report.
This condition unleashes a cascade of debilitating symptoms, rendering muscles unable to relax once contracted. As the disease progresses, patients experience progressive symptoms, including loss of mobility, speech, and even the ability to breathe. Life expectancy with MD1 is relatively short.
Professor Ke Zhang and his team aim to develop a safe and efficient oligonucleotide delivery technology to treat MD1. They aim to change the status quo by developing a gene regulation technology that treats the disease at the genetic source, addresses non-liver organs, reduces cost, and minimizes off-target effects.
Through pacDNA Inc., founded in 2023, they are working to bring revolutionary treatments for muscular diseases to market. The team plans to dose the first human in three years and have at least one drug available within ten years.
“Our goal is to get our technology into human clinical trials to assess the safety and efficacy of our drug candidates, with the hope of getting a drug approved to treat several challenging conditions,” says Carl LeBel, CEO of pacDNA.
“Even if one patient benefits from pacDNA’s medicine, I feel that my academic career is worth the while,” says Dr. Zhang.
Commercialization with the CRI: Launching from Lab to Industry
The research behind pacDNA’s technology began in Dr. Zhang’s research group in 2015, and the team has been enhancing various aspects of it ever since.
“To this day, we are still gaining a deeper understanding of how it works and expanding its applications,” says Dr. Zhang.
The CRI supports commercially viable projects, even during ongoing research, by helping found spinout companies, protecting valuable IP, and forming strategic industry partnerships.
The CRI has been working with Dr. Zhang and pursuing broad Intellectual Property coverage.
“In many scenarios, there are commercially viable projects that can be pursued while the research is ongoing,” says Saini. “That’s why the CRI is passionate about helping get important projects out of the lab, founding spinout companies, and finding strategic industry partnerships, even when the research is still in progress.”
With no DM1 drugs currently available on the market, pacDNA 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 CRI provides valuable services for startups focused on commercialization, as we help make processes like licensing proceed with speed and ease,” says Saini. “We value professional interactions and support university IP-based startup teams that demonstrate a willingness to learn the commercial mindset and engage with CRI.”
With the help of Northeastern resources like the CRI, Dr. Zhang’s team is deftly navigating business development, licensing agreements, and industry connections, garnering significant interest from pharmaceutical companies and propelling their vision closer to a reality for patients.
“Getting pacDNA off the ground means I had to talk to many people,” says Dr. Zhang, “and these conversations showed me different perspectives. The mindset of industry can be quite different from academia.”
“A biotech startup faces immense challenges and a path filled with surprises,” says Dr. LeBel. “My approach is to generally expect the unexpected and develop a series of options to address the challenges when they arise.”
Funding for pacDNA
pacDNA has raised about $3M in non-dilutive funding through government grants like SBIR and STTR, corporate partnerships, and various pitch competitions. The team is also pursuing dilutive funding from VCs and plans to open a seed funding round in the near future.
“The license through CRI is an important step forward into drug development,” says Dr. LeBel. “The next step will require us to raise a seed round of financing to achieve our goal of advancing into human trials.”
Northeastern CRI’s Spark Fund Award partly funds this technology, as the team was selected as one of the Fall 2023 Spark Fund awardees. The team was also a recipient of The Spark Award in December 2022.
Written by Elizabeth Creason