Dimension Therapeutics will partner the Perelman School of Medicine at the University of Pennsylvania (Penn Med) to advance gene therapies for inherited metabolic diseases, under a research collaboration whose value was not disclosed.
Dimension’s collaboration with Penn Med will focus on research conducted with the laboratory of James Wilson, M.D., Ph.D., professor of medicine and pediatrics and director of the Orphan Disease Center at Penn Med.
Penn Med and Dr. Wilson are both equity holders of Dimension, while Dr. Wilson holds shares of REGENXBIO, a gene therapy developer that went public in September, raising $139 million in its IPO. REGENXBIO’s proprietary NAV® Technology Platform has been licensed worldwide exclusively in each disease area by Dimension.
Dimension aims to advance therapies based on a next-generation adeno-associated virus (AAV) platform that has shown early validation and preclinical in vivo proof of concept across multiple disease states in academic research centers. Dimension’s current efforts focus on AAV8 and AAVrh10, two forms of AAV vectors that selectively target liver cells and have been optimized to deliver missing intact genes in diseases associated with the liver.
According to Dimension, AAV gene therapy in patients living with hemophilia B has yielded initial indications of positive net benefit, and robust gene expression has been shown in multiple preclinical in vivo models and commercially viable novel Chemistry, Manufacturing, and Controls (CMC) approaches.
The collaboration will include exclusive programs designed to treat citrullinemia type 1, phenylketonuria (PKU), and Wilson disease, a rare genetic disease associated with the liver that causes severe copper poisoning of the liver, brain, and other vital organs.
Penn Med has agreed to oversee all preclinical translational research to support candidate selection and IND filings in each disease area. Dimension will be responsible for the manufacture of the vectors for IND-enabling studies and subsequent advancement into clinical testing.
Dimension also agreed to oversee funding for all research activities, with Penn Med eligible to receive research based milestones and royalties on future product sales.
“We are pleased to continue our productive partnership with Dimension, which combines the unique translational capabilities of my lab with Dimension’s drug development expertise, enabling the advancement of new AAV-based therapies for liver-associated diseases,” Dr. Wilson said in a statement. “We believe these therapies, based on extensive, peer-reviewed preclinical validation and testing, have the potential to transform patient care and give clinicians new options to treat previously intractable diseases.”
Dr. Wilson is clinical development editor of Human Gene Therapy, a journal published by GEN publisher Mary Ann Liebert Inc., and was editor-in-chief of the journal from 2003 until last year.