Bayer is making a big bet on gene therapy with the acquisition of North Carolina-based Asklepios BioPharmaceutical (AskBio). Bayer is paying $2 billion upfront for AskBios AAV-based gene therapy pipeline of treatments for Pompe disease, among others, and could pay an additional $2 billion in potential milestones.

AskBios Pro10 AAV manufacturing process has become something of a standard across the industry. The platform is used by multiple companies, including Pfizer, Takeda and Viralgen Vector Core SA. The company holds over 500 patents in areas such as AAV production, chimeric vectors and self-complementary DNA. AskBios technology has already seen regulatory success. It initially developed the gene therapy for spinal muscular atrophy that Illinois-based AveXis, a subsidiary of Novartis, won approval for from the U.S. Food and Drug Administration in 2019. AskBios lead research programs, which are focused on Pompe disease, Parkinsons disease and congestive heart failure are currently in early phases of clinical development.

Under terms of the deal, Bayer will own full rights to AskBios pipeline of treatments for Pompe disease, Parkinsons disease, as well as therapies for neuromuscular, central nervous system, cardiovascular and metabolic diseases. AskBio will remain an autonomous company under the Bayer umbrella and will operate on an arms-length basis, Bayer said this morning. AskBio Chief Executive Officer Sheila Mikhail noted that her company will retain its independent structure, which she said will allow them to provide accelerated development of gene therapies to treat more patients who can benefit from them.

Our innovation in capsid re-engineering and promoter design, coupled with our scaled manufacturing processes, gives us the tools to provide gene therapy solutions to more people suffering from a wider spectrum of disease that is not being adequately treated today, added Richard Jude Samulski, AskBios chief scientific officer. Samulski was the first scientist to clone AAV.

The acquisition of AskBio will bolster Bayers cell and gene therapy business and will lay the foundation for future partnerships in the area of adeno-associated virus (AAV) therapies, Bayer said. Besides multiple clinical-stage assets for indications with highly unmet needs, the acquisition includes a state-of-the-art gene therapy technology platform as well as existing gene therapy manufacturing platform, the company added.

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The addition of AskBio will complement Bayers other cell and gene therapy company, BlueRock Therapeutics, which it acquired last year. BlueRock is developing induced pluripotent stem cells (iPSC), with its most advanced program aimed at Parkinsons disease.

Werner Baumann, chairman of the Board of Management at Bayer, said the acquisition of AskBio significantly advances the establishment of a cell and gene therapy platform that can be at the forefront of breakthrough science and contribute to the development of therapies that can prevent or cure diseases caused by genetic defects. Baumann said the goal is in line with the companys purpose of science for a better life.

As part of our strategy, we are building new therapeutic platforms including cell and gene therapies, Stefan Oelrich, president of the Bayers Pharmaceuticals Division said 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. We want to help patients whose medical needs are not yet met by todays treatment options and we are looking forward to work together with the team at AskBio.

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Bayer Acquires AskBio to Expand Gene Therapy Platform - PharmaLive