Bayer is the latest pharmaceutical company to join the gene therapy club, with plans to acquire Asklepios BioPharmaceutical, a private gene therapy firm based in Research Triangle Park, North Carolina, for $2 billion. Asklepios, often called AskBio, could earn another $2 billion in milestone payments.
AskBio was cofounded in 2001 by gene therapy scientist Jude Samulski and life-science attorney Sheila Mikhail, chief scientific officer and CEO, respectively. The company was based on Samulski’s pioneering work 40 years ago when he engineered adeno-associated viruses (AAVs) as vessels for delivering DNA into cells.
Those so-called AAV vectors are now the workhorses of the gene therapy boom. The first two commercial gene therapies in the US, Luxturna and Zolgensma, are made with AAV vectors. Novartis acquired the company behind Zolgensma for $8.7 billion in 2018, and Roche acquired the company behind Luxturna for $4.8 billion last year. Astellas Pharma and Biogen, acquired clinical-stage gene therapy firms in the past two years. Bayer wanted in on the action, too, and saw an opportunity in a company that has yet to see one of its own gene therapies approved.
“We decided that we want to be a leading player in the cell and gene therapy field,” says Marianne De Backer, who joined Bayer as head of global business development and licensing for pharmaceuticals last year. The acquisition will build on Bayer’s recent stem cell–therapy investments, she adds, including its acquisition of BlueRock Therapeutics and a $215 million investment in the start-up Century Therapeutics.
AskBio previously spun off its gene therapy programs for hemophilia and Duchenne muscular dystrophy into separate companies, which are now owned by Pfizer and Takeda Pharmaceuticals, respectively. AskBio now has several early-stage programs for cardiovascular, metabolic, neurological, and neuromuscular diseases.
Mikhail says Bayer’s experience in cardiovascular drug development will be a boon to one of AskBio’s most unique programs: a gene therapy for congestive heart failure. Most gene therapies in development are for rare diseases with small numbers of patients. Developing gene therapies for more common conditions, such as heart failure, will require larger, more expensive trials.
Bayer’s small-molecule drug expertise could benefit AskBio as well. Last year, AskBio acquired the British firm Synpromics, which has a technology for turning a gene on or off with synthetic promoters controlled by small molecules. Many people require chronic injections of antibody therapies. AskBio is developing an AAV vector that contains a gene for a therapeutic antibody. The vector could turn the liver into a personal bioreactor that churns out the antibody, but only when activated by a small molecule.
“We are hopeful that we can work with the Bayer medicinal chemistry team and their small-molecule library to find more molecules” that work with the Synpromics gene-controlling technology, Mikhail says.
AskBio will remain autonomous after the acquisition, De Backer says. The group will continue to develop improved AAV vectors and other gene therapy technologies while advancing its existing programs. “We have the full intention to leave AskBio as independent as humanly possible, so Sheila and Jude can focus on the science,” she says.