Asklepios Biopharmaceutical (AskBio), a US clinical-stage gene therapy platform company dedicated to rare genetic disorders, is currently expanding its technological capabilities. This AAV gene therapy company has now acquired Synpromics, a UK-based leader in gene control synthetic promoter technology, bioinformatics and intelligent data-driven design that enables more precise cell targeting and gene expression. “By combining Synpromics’s customized synthetic promoters with AskBio’s capsids and Pro10™ manufacturing cell line, we have achieved technical leadership in each of the critical components for successful development of rAAV-based therapeutics,” said Sheila Mikhail, CEO and co-founder of AskBio. With the Synpromics acquisition, we have enhanced our collective ability to develop highly targeted and maximally expressed gene therapies. Today AskBio is better positioned to tackle larger pathway diseases, as we continue our efforts to bring curative therapies for rare disease to patients in need.”
Improving efficacy of AAV-based vectors
Founded in 2010 by Michael L Roberts, Ph.D., Synpromics created PromPT™, a proprietary data-driven promoter design and bioinformatics platform. PromPT™ has helped yield the production of groundbreaking cell selective synthetic promoters and regulated and inducible gene expression solutions. While naturally occurring promoters have limitations when utilized for industrial or therapeutic applications, these synthetic promoters are designed to better regulate gene activity and precisely control protein production. Synpromics’ strategic portfolio of synthetic promoters for application in cell and gene therapy and human healthcare biomanufacturing significantly enhances the AskBio AAV gene therapy platform and therapeutic portfolio. Combination of Synpromics’ promoter and bioinformatics technology with AskBio’s AAV technology, capsid library, proprietary manufacturing systems and multi-dimensional gene therapy platform could create a powerful opportunity to more accurately target complex diseases and improve the efficacy of AAV gene therapy vectors. Both companies will continue to operate as separate entities. Synpromics will operate as a wholly owned subsidiary and remain headquartered in Edinburgh (UK). But AskBio and Synpromics will share intellectual property with immediate plans to integrate Synpromics’ technology with AskBio’s AAV platform ecosystem and current therapeutics portfolio under development. This places AskBio in a unique position to be one of the only companies in the industry with a comprehensive end-to-end AAV gene therapy platform.
Earlier in August, AskBio has already announced a significant, strategic partnership with Selecta Biosciences. This collaboration will leverage proprietary technology platforms of both companies with a human proof of concept trial to validate the potential for re-dosing in patients with genetic diseases. Currently the ability to re-administer systemic AAV gene therapy is limited by the development of neutralizing antibodies. The ability to safely re-dose AAV should help achieve therapeutic benefit in patients who are under-dosed; it should also help restore transgene expression in patients, particularly growing pediatric patients, who may lose expression over time. Selecta is the first company with preclinical evidence to support the potential for re-dosing patients receiving gene therapy. When used in combination with AAV gene therapy vectors, Selecta’s ImmTOR™ inhibits the immune response to the vector. Last October, Selecta’s and Genethon’s researchers have published results indicating that this co-administration of synthetic vaccine particles encapsulating rapamycin with adeno-associated virus gene therapy vectors induced safe and effective mitigation of immune responses against the capsid in an antigen-selective manner (Nature Communications)