Genetics company ASKBio RTP to pursue more gene therapies in new partnership

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search triangle garden – Gene therapy company Asklepios BioPharmaceutical (AskBio) of the Research Triangle Park has signed a multi-year research collaboration and option agreement with ReCode Therapeutics to develop gene-editing technology to drive new precision therapies.

“The collaboration pairs AskBio’s expertise in synthetic DNA and CRISPR applications with ReCode’s new SORT LNP delivery technology for the potential to discover new genetic medicines,” said Sheila Michael, CEO and co-founder of AskBio. “Through this collaboration, we have an opportunity to advance AskBio’s efforts in gene editing and non-viral delivery, which support and align with our commitment to serving as the industry’s leading driver of gene therapy innovation.”

Under the terms of the agreement, AskBio will combine synthetic DNA and gene-editing endonucleases with ReCode Selective Organ Targeting Technology (SORT) of lipid nanoparticles (LNP). This marriage potentially creates an “all-in-one solution” that enables complete gene insertion by precisely delivering both a gene-editing tool and DNA as mixed goods in a single LNP to the desired targets, according to the companies’ announcement of the collaboration.

AskBio CEO Sheila Michael is fighting a two-front battle against diseases — their genetic threats and others

“AskBio’s pioneering gene-editing technology is a powerful complement to the modular SORT LNP genetic drug platform, which enables precise delivery of a variety of genetic cargoes to specific organs and cells, including large and complex genetic cargoes and mixed payloads,” said Shahnaz, CEO of ReCode. . “We are excited to combine these unique platforms to develop next-generation gene therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases.”

The two companies note that the joint work could expand gene editing for liver and lung disease targets.

As part of the deal, ReCode will receive an undisclosed cash advance with the ability to make principal payments and royalties on new treatments resulting from the collaboration.

Another “step forward” for AskBio technology

AskBio, founded in 2001, is a pioneer in the use of adeno-associated viruses (AAV) as a delivery system for the introduction of therapeutic genetic material into living tissue. The company holds more than 750 patents in this area, driven by the scientific leadership of co-founder Judd Samulsky, Ph. D., the world’s foremost AAV cloning scientist.

Early work by Samulski’s team at AskBio and related entities was supported by grants and loans from the North Carolina Biotechnology Center totaling nearly $1 million. Support began 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 a range of diseases of the central nervous system, neuromuscular, metabolic, and cardiovascular disorders. AskBio’s clinical-stage pipeline includes treatments for Pompe disease, Parkinson’s disease, and congestive heart failure.

According to the announcement, the company’s work with ReCode “marks another step forward for AskBio in advancing gene-editing and other non-viral delivery technologies and expertise.”

AskBio is a wholly owned and independently operated subsidiary of Bayer AG, which acquired AskBio in 2020 as the cornerstone of its newly formed cell and gene therapy platform. It was a huge deal for AskBio, which received $2 billion in advance and up to $2 billion in potential milestone payments.

Joint connection with Bayer

ReCode, headquartered in Menlo Park, California, is a genetic pharmaceutical company that uses next generation mRNA and gene correction therapies.

In June 2022, ReCode closed a $200 million Series B extension funding round, co-led by Leaps by Bayer, the investment unit of Bayer AG, to strengthen the Leaps by Bayer innovation portfolio in gene therapies.

The funds raised are used to advance the development of the ReCode platform and pipeline to selectively deliver gene drugs to target types of organs and cells in a “predictable and programmable manner,” according to the company.

(c) NC Biotech Centre

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