AskBio Acquires Synpromics Ltd. and Expands its Gene Therapy Technology Portfolio

RESEARCH TRIANGLE PARK, N.C., Aug. 13, 2019 /PRNewswire/ -- AskBio, the world's foremost clinical stage and gene therapy platform company, has acquired Synpromics, the leader in gene control synthetic promoter technology, bioinformatics and intelligent data-driven design that enables more precise cell targeting and gene expression. Synpromics will operate as a wholly owned subsidiary and remain headquartered in Edinburgh, United Kingdom.

"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."

AskBio was founded by Jude Samulski, Ph.D., the first scientist to clone Adeno-Associated Virus (AAV), along with Xiao Xiao, Ph.D., who was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne Muscular Dystrophy, and Ms. Mikhail, an accomplished life sciences executive. Focused on the development and delivery of curative gene therapies, AskBio's dynamic AAV technology platform and therapeutics pipeline serve patient populations with rare and generally untreatable genetic diseases. The company's extensive IP portfolio and manufacturing systems also enable other gene therapy companies such as Pfizer to advance AAV gene therapy for DMD and Avexis for SMA.

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, Synpromics' synthetic promoters are designed to better regulate gene activity and precisely control protein production. This drives gene expression at an uncompromised level of selectivity in any cell type, tissue, environmental or biological condition. In addition to enabling more effective current and future-generation cell and gene therapies, the technology also has advanced bioprocessing applications.

"The significant possibilities that our two companies represent for the advancement of gene therapy cannot be overstated," said David Venables, CEO of Synpromics. "Aligning the scientific expertise of our company with the unparalleled vision of Dr. Samulski and AskBio's AAV platform technology can transform the quality, efficacy and safety of gene therapy vectors, ultimately allowing for a wider scope of diseases treatable by AAV therapeutics."

AskBio's AAV technology, capsid library, proprietary manufacturing systems and multi-dimensional gene therapy platform combined with Synpromics' promoter and bioinformatics technology 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 but 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.

"For some time, the field has understood that the three essential components for advancing successful AAV gene therapy has centered around production, capsids and promoters. Today AskBio has added the last critical component to our tool chest," added Dr. Jude Samulski. "There is no doubt in my mind that the shared value of AskBio's and Synpromics' technologies marks a major step forward in the evolution of AAV gene therapy."

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. 

"By combining AskBio's AAV delivery platform with Synpromics' gene regulation platform, we have created an unrivalled offering in gene medicine. In particular, integrating our inducible promoter systems into the AskBio platform will enable precision control in the next generation of therapies," added Dr. Michael Roberts, the founder and Chief Scientific Officer of Synpromics. "This will also improve the AAV manufacturing process, further enabling the generation of stable producer cell lines. Our shared vision is that the synergies of our two platforms will evolve hand-in-hand to create solutions that can address more complex pathway diseases in the years to come."

About AskBio
Founded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy platform company dedicated to improving the lives of children and adults with rare genetic disorders. AskBio's gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10^TM and an extensive AAV capsid library. Based in Research Triangle Park, N.C., the company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors, and self-complementary DNA. AskBio maintains a portfolio of clinical programs across a range of indications including Pompe, Limb Girdle Muscular Dystrophy, Cystic Fibrosis, Myotonic Muscular Dystrophy, Hemophilia (Chatham Therapeutic/Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics/Pfizer). For more information, visit www.askbio.com.

About Synpromics
Synpromics is the leader in gene control, improving human health by enabling safer, more effective cell and gene medicines through proprietary genomics, bioinformatics and intelligent data-driven design. The company has developed PromPT™, its multi-dimensional bioinformatics database, that enables product-specific promoter design and selection empowering the next generation of cell and gene-based medicines and bioprocessing applications. The company operates in a diverse range of fields, including broad applications in cell- and gene-based medicine, biologics manufacturing and viral vector bioprocessing. Current partners include Audentes, BioMarin, uniQure, AGTC, Takeda, Solid Biosciences, Lonza, Oxford Biomedica and Regeneron, as well as numerous undisclosed partners in the gene therapy and bioprocessing sectors. For more information, visit www.synpromics.com.

SOURCE AskBio

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