Editor’s note: Each week WRAL TechWire takes a deep dive inside emerging technologies, companies and research and development.
RESEARCH TRIANGLE PARK – Gene therapy company Asklepios BioPharmaceutical (AskBio) of Research Triangle Park has signed a multi-year research collaboration and option agreement with ReCode Therapeutics to develop gene-editing technology for driving new precision therapies.
“The collaboration pairs AskBio’s expertise in synthetic DNA and CRISPR applications with ReCode’s novel SORT LNP delivery technology to potentially discover new genetic medicines,” said Sheila Mikhail, AskBio’s chief executive officer and co-founder. “Through this collaboration, we have an opportunity to advance AskBio’s gene editing and non-viral delivery efforts, which support and align with our commitment to serve as an industry-leading engine of gene therapy innovation.”
Under terms of the agreement, AskBio will combine its synthetic DNA and gene editing nucleases with ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) technology. That marriage could potentially create an “all-in-one solution” that enables full gene insertion by delivering with precision both the gene editing tool and DNA as mixed cargo in a single LNP to desired targets, according to the companies’ announcement of the collaboration.
“AskBio’s pioneering gene editing technology is a powerful complement to our modular SORT LNP genetic medicines platform, which enables the precise delivery of a wide variety of genetic cargoes to specific organs and cells, including large, complex genetic cargoes and mixed payloads,” said Shehnaaz Suliman, chief executive officer of ReCode. “We are excited to combine these unique platforms to develop next-generation genetic therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases.”
The joint work could extend the reach of gene editing for liver and lung disease targets, the two companies noted.
As part of the deal, ReCode will receive an undisclosed up-front cash payment with potential for downstream milestone payments and royalties on novel therapeutics resulting from the collaboration.
‘Another step forward’ for AskBio’s technology
AskBio, founded in 2001, is a pioneer in the use of adeno-associated viruses (AAV) as a delivery system for inserting therapeutic genetic material into living tissue. The company holds more than 750 patents in the field, driven by the scientific leadership of co-founder Jude Samulski, Ph.D., the first scientist to clone AAV.
Early work by Samulski’s team at AskBio and related entities was supported by grants and loans from the North Carolina Biotechnology Center totaling about $1 million. The support started with a $250,000 grant that helped recruit Samulski to the University of North Carolina at Chapel Hill School of Medicine from the University of Pittsburgh in 1993.
AAV gene therapy has broad therapeutic implications for an array of central nervous system, neuromuscular, metabolic and cardiovascular disorders. AskBio’s clinical-stage pipeline includes therapeutics for Pompe disease, Parkinson’s disease and congestive heart failure.
The company’s work with ReCode “represents another step forward for AskBio in developing its gene editing and other non-viral delivery technologies and expertise,” according to the announcement.
AskBio is a wholly owned and independently operated subsidiary of Bayer AG, which acquired AskBio in 2020 as a cornerstone of its newly formed cell and gene therapy platform. It was a blockbuster deal for AskBio, which received $2 billion up front and up to $2 billion in potential milestone payments.
A shared connection with Bayer
ReCode, based in Menlo Park, Calif., is a genetic medicines company using next-generation mRNA and gene-correction therapeutics.
In June 2022, ReCode closed on a $200 million Series B extension financing round, co-led by Leaps by Bayer, the investment unit of Bayer AG, to further strengthen Leaps by Bayer’s innovation portfolio in gene therapies.
The funds raised are being used to advance the development of ReCode’s platform and pipeline to selectively deliver genetic medicines to target organ and cell types in a “predictable and programmable fashion,” according to the company.
(C) N.C. Biotech Center