AskBio Bioreactor (Bayer AG)
26 Oct. 2020. Global drug maker Bayer AG is acquiring the company Asklepios BioPharmaceutical, developer of gene therapies to treat inherited disorders. Under the agreement, Asklepios Bio, or AskBio, could receive as much as $4 billion if all terms of the deal are fulfilled.
AskBio, in Research Triangle Park, North Carolina, designs treatments for genetic diseases, as well as diseases where genetic factors interact with environmental factors that contribute to their severity or frequency. The company’s work is based on pioneering research on adeno-associated viruses by one of its founders Jude Samulski, beginning as a graduate student in the 1970s and 80s. The adeno-associated virus or AAV is a benign and naturally occurring microbe that infects cells, but does not integrate with the cell’s genome or cause disease, other than at most mild reactions. As a result, the virus has become a workhorse for delivering healthy genes to treat inherited diseases.
For gene therapies, AskBio creates engineered AAVs by inserting a healthy human gene into the virus’s genome for delivery to human cells or tissue, as well as removing problematic viral components. In some cases, AskBio’s AAVs can deliver two genes at once. The company further modifies AAVs by designing their capsids, the virus’s protein shell, to carry out tasks such as identifying targets, performing navigation, and controlling dosage for therapeutic delivery. The company says it uses bioinformatics, computer modeling, and crystal structure analytics to design these viral capsids.
AskBio says it maintains a library of engineered capsids that can be assembled to perform specified tasks as needed by therapy designers. The company also maintains a library of components it calls promoters, also designed with bioinformatics, that control gene expression, and provide better targeting of cells and tissue. AskBio’s pipeline today includes gene therapies for the rare inherited disorder Pompe disease, Parkinson’s disease, and congestive heart failure, all in early- or mid-stage clinical trials. Treatments for other neuromuscular, neurological, or metabolic disorders are in preclinical stages.
The acquisition gives Bayer, headquartered in Berlin, full rights to AskBio’s gene therapy platform, as well as contract management and manufacturing capabilities. In addition, Bayer gains AskBio’s full intellectual property portfolio that AskBio says exceeds 500 patents. AskBio is receiving $2 billion in an initial payment, and the company is eligible for another $2 billion on completion of unspecified milestones, with the vast majority of those achievements expected in the next five years.
Bayer says AskBio will continue to operate as an independent entity on an arm’s-length basis from Bayer’s corporate headquarters. Bayer expects AskBio’s gene therapies to complement stem cell therapies for regenerative medicine gained from its acquisition last year of BlueRock Therapeutics, initially formed as a joint venture of Bayer and Versant Ventures, as we reported in 2016.
“As part of our strategy, we are building new therapeutic platforms including cell and gene therapies,” says Stefan Oelrich, head of Bayer’s pharmaceuticals division, in a statement. “As an emerging leader in the rapidly advancing field of gene therapies, the expertise and portfolio of AskBio supports us in establishing highly innovative treatment options for patients and further strengthens our portfolio.”
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