RESEARCH TRIANGLE PARK – Emboldened by a recent cash infusion of $225 million from investors, AskBio, a clinical stage gene therapy company in Research Triangle Park, has acquired a Scottish company to help it develop therapeutics for a wider array of genetic disorders than the rare diseases it has targeted to date.
AskBio
The acquisition of Synpromics of Edinburgh will give AskBio access to Synpromics’ gene-control technology and expertise in bioinformatics and data-driven design. The new tools will allow more precise cell targeting and gene expression, AskBio said in a news release announcing the deal.
The added capacity will strengthen AskBio’s recombinant Adeno-Associated Vector (AAV) gene therapy platform, which uses engineered virus nanoparticles as vectors to deliver corrective DNA to targeted cells in diseased patients.
“With the Synpromics acquisition, we have enhanced our collective ability to develop highly targeted and maximally expressed gene therapies,” said Sheila Mikhail, chief executive officer and co-founder of AskBio. “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.”
Terms of the acquisition were not disclosed, but Synpromics will operate as a wholly owned subsidiary and remain headquartered in Edinburgh. Both companies will continue to operate as separate entities but will share intellectual property and integrate their technologies immediately.
AskBio said it now has a comprehensive end-to-end AAV gene therapy platform consisting of the three essential components for advancing AAV gene therapy: production, capsids and promoters.
AskBio’s current pipeline. (AskBio image)
“Today AskBio has added the last critical component to our tool chest,” said Jude Samulski, Ph.D., co-founder of AskBio and a pioneer in AAV gene therapy. “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.”
Prolific scientist, entrepreneur
Samulski was recruited to the University of North Carolina School of Medicine from the University of Pittsburgh in 1993 with the help of about $250,000 in grant funding from the North Carolina Biotechnology Center. Several grants and loans from the Biotech Center have supported the development of Samulski’s academic research and commercial technologies respectively since his recruitment to UNC, where he led the School of Medicine’s Gene Therapy Center for several years.
Samulski was the first scientist to clone Adeno-Associated Virus, and his fellow AskBio co-founder, Xiao Xiao, Ph.D., was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne Muscular Dystrophy (DMD).
Samulski, Xiao and Mikhail established Asklepios BioPharmaceutical, now better known as AskBio, in 2001 to develop curative gene therapies for rare and generally untreatable genetic diseases. The company’s extensive intellectual property and manufacturing systems also help other companies develop gene therapies, such as Pfizer’s potential treatment for DMD and AveXis’ therapy for spinal muscular atrophy (SMA). (AveXis is investing $55 million in a new Durham manufacturing facility for gene therapy treatments that will create 200 jobs.)
AskBio has also spun out numerous gene therapy companies, including NanoCor Therapeutics, Chatham Therapeutics, Bamboo Therapeutics and Actus Therapeutics.
AskBio’s gene therapy platform includes a proprietary cell line manufacturing process known as Pro10 and an extensive library of AAV capsids, the protein shells of viruses. The company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing.
The company holds more than 500 patents in areas such as AAV production, chimeric vectors and self-complementary DNA.
AskBio has a portfolio of clinical programs across a range of genetic diseases including Pompe, limb girdle muscular dystrophy, cystic fibrosis, myotonic muscular dystrophy, hemophilia and DMD. Earlier this year, privately held AskBio raised $225 million in venture capital and another $10 million from founders and board members. The funding was the largest single round of venture capital financing ever for a North Carolina bioscience company.
Synpromics: focused on gene regulation
Synpromics, founded in 2010, designs synthetic promoters to better regulate gene activity and precisely control protein production in specific cells. This drives gene expression at “an uncompromised level of selectivity in any cell type, tissue, environmental or biological condition, enabling more effective current and future-generation cell and gene therapies,” according to the company. Synpromics’ technology has 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.
AskBio spins out new gene therapy company, Actus Therapeutics
“By combining AskBio’s AAV delivery platform with Synpromics’ gene regulation platform, we have created an unrivaled offering in gene medicine,” said Michael Roberts, Ph.D., founder and chief scientific officer of Synpromics. “In particular, integrating our inducible promoter systems into the AskBio platform will enable precision control in the next generation of therapies. This will also improve the AAV manufacturing process, further enabling the generation of stable producer cell lines.”
David Venables, chief executive officer of Synpromics, said, “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.”
(C) N.C. Biotech Center
