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

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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 Acquires Synpromics Ltd.
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AskBio’s AAV technology, capsid library, proprietary manufacturing systems and multidimensional
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.”