Asklepios BioPharmaceutical, a developer and manufacturer of Adeno-Associated Virus (AAV) gene therapy therapeutics for underserved patient populations with rare and generally untreatable genetic disorders, raised $235m in funding.
TPG Capital and Vida Ventures are investing $225m for a minority stake in the company. As part of the transaction, AskBio’s founders and board members are co-investing $10m.
The company intends to use the funds to advance and expand clinical trials, enhance its manufacturing capabilities and capacity, and drive long-term growth.
Founded in 2001 by Dr. Jude Samulski, Ph.D., the first scientist to clone AAV, along with Dr. Xiao Xiao, who was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne Muscular Dystrophy, and company CEO Sheila Mikhail, an accomplished life sciences executive, Asklepios BioPharmaceutical is a gene therapy platform company dedicated to improving the lives of children and adults with rare genetic disorders.
Its gene therapy platform includes a proprietary cell line manufacturing process known as Pro10 and an extensive AAV capsid library. The company has generated hundreds of proprietary third generation gene vectors that have entered clinical testing, and maintains a portfolio of clinical programs across a range of indications including Pompe,
AskBio has partnered with companies on two of its programs:
– Bamboo Therapeutics, a therapy targeting Duchenne Muscular Dystrophy and other rare diseases, and
– Chatham Therapeutics, which focuses on Hemophilia A and B.
The platforms were sold to Pfizer and Baxter (programs now owned by Takeda), respectively.
The company recently advanced a therapeutic for Pompe Disease into the clinic based on the work by Dwight Koeberl, M.D., Ph.D., and Priya Kishnani, M.D., M.B.B.S., at Duke University. The company is also developing therapeutics for heart failure, Limb Girdle 2i, Myotonic Dystrophy, and several other muscular and neurological disorders. Its AAV manufacturing process, Pro10, currently yields 1018 in 200-liter bioreactors and is recognized within the industry as the gold standard in scale manufacturing.
Pro10 is used by Pfizer, Takeda, and Viralgen Vector Core SA. Recently, AskBio formed a partnership with Touchlight Genetics Ltd. for the commercialization of a safer, efficient alternative to plasmid DNA.
The team also includes Dr. Jude Samulski, Ph.D., President and Chief Scientific Officer.