NEW HAVEN, Conn., July 28, 2022 — Modifi Biosciences today announced that the journal Science published a critical study validating the company’s novel oncology platform based on new classes of molecules that exploit tumor-associated DNA repair defects by directly modifying the DNA of cancer cells. The company has closed a $6.4 million funding round, which will support studies enabling IND and the expansion of the platform to target cancers with other DNA repair defects. Leading investors in this round include HighCape Capital; Connecticut Innovations; Capital of Ironwood; the Brain Tumor Investment Fund, an affiliate of the National Brain Tumor Society; and Yale Ventures.
“This approach redefines the rules for how to selectively kill cancer cells via direct DNA modification and positions our company as a leader in this emerging field of oncology drug development,” said co-lead author Ranjit Bindra. of the study and co-founder of Modifi Bio. MD, PhD. Bindra is the Harvey and Kate Cushing Professor of Therapeutic Radiology at Yale School of Medicine and Scientific Director of the Yale Brain Tumor Center at Smilow Cancer Hospital. “As a brain tumor physician treating patients for over a decade, my colleagues and I have seen gliomas claim the lives of too many patients. Our discovery represents a major step forward in shifting the treatment paradigm for this devastating disease, as well as many other cancers with intrinsic DNA repair defects.
The technology bypasses conventional approaches that indirectly target proteins in cancer cells and demonstrates robust anti-tumor activity in glioma, one of the deadliest forms of brain cancer, while sparing normal tissue. In the Science publication, the group’s new class of molecules have been shown to be extremely active and selective against cancer cells lacking the expression of a key DNA repair protein called MGMT (O6-methylguanine methyl transferase).
About half of all glioblastomas and up to 80% of gliomas lack MGMT. Emerging research indicates that MGMT deficiency is seen in many other tumor types, suggesting broad applicability of this strategy in cancer treatment.
Modifi Bio creates a new class of molecules, based on research conducted at Yale, that fragment within cells and induce irreparable DNA changes in cancer cells with DNA repair defects. Modifi Bio’s compounds are designed to be orally bioavailable and possess favorable drug-like properties, which will allow them to quickly file an Investigational New Drug Application with the United States Food and Drug Administration in anticipation phase I clinical trials in 2024.
“These molecules hold particular promise as therapeutics due to their ability to directly modify the DNA of cancer cells, which we believe will not only be effective in fighting cancer, but will also allow us to overcome the mechanisms resistance,” said Seth Herzon, PhD, study co-lead author, Modifi Bio co-founder and Milton Harris ’29 PhD Professor of Chemistry at Yale Faculty of Arts and Science.
“New treatments for these types of brain cancer are urgently needed. I am thrilled that this groundbreaking discovery could lead to the first clinically significant pharmacological advances in the treatment of glioma in more than two decades,” said Roger Stupp, MD, Modifi Bio Scientific Advisory Board Member, Paul C. Bucy Professor of Surgery neurologist and chief of neuro-oncology at Northwestern University. Stupp first published the pivotal studies more than 20 years ago, which define the current standard of care for glioma.
The research was conducted at Yale University and the co-first authors of the Science study are Kingson Lin and Susan Gueble. Co-authors are Ranjini Sundaram and Eric Huseman. The technology highlighted in the research has been exclusively licensed to Modifi Bio with support from Yale Ventures. Modifi Bio co-founders include Herzon, Bindra, Lin and Kevin Rakin.
Funding for the research came, in part, from the National Institutes of Health and the Yale Cancer Center. Additionally, Bindra received the CureSearch for Children’s Cancer’s Catapult Award. CureSearch’s mission is to end childhood cancer by conducting targeted, innovative research with measurable results in an accelerated time frame.
About Modifi Bio
Based in New Haven, CT, Modifi Bio is a leader in direct DNA cancer modification, developing novel therapies by uniquely targeting tumors with associated DNA repair defects. Aimed at improving the quality of cancer treatment and patient care, the Modifi Bio team includes world-renowned biotechnology scientists, clinicians and entrepreneurs with deep expertise in synthetic chemistry, DNA repair and translational research in oncology. The company has offices and labs at the Elm City Bioscience Center, a newly renovated 110,000 square foot facility within walking distance of Yale that supports emerging biotech companies in New Haven.
Human tissue samples
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