Aug 22, 2019   SOURCE: PR NewsWire

AskBio Applauds Pfizer's Continuing Investments in Gene Therapy

RESEARCH TRIANGLE PARK, N.C., Aug. 22, 2019 /PRNewswire/ -- In 1978, when a doctoral student named Jude Samulski started a journey of discovery for how the adeno-associated virus (AAV) could be used to safely deliver corrected genes to cells with genetic defects, the idea that one of the world's largest pharmaceutical companies would build its gene therapy research program on his work never crossed his mind.

And that's exactly what happened.

On August 21, 2019, Pfizer announced a $500 million investment in a state-of-the-art gene therapy manufacturing facility that is based on Dr. Samulski's and AskBio's AAV technology discoveries. The facility, not even an hour's drive from Dr. Samulski and AskBio, the company he co-founded, further validates AskBio's AAV technology leadership as well as the Research Triangle region's status as a world-leading center for gene therapy research and development.

We at AskBio send heartiest congratulations to our colleagues at Pfizer on this enormous step forward. Our hope has always been that our technology would further not only AskBio's success, but also the potential for success throughout the gene therapy industry.

Pfizer's AAV therapeutic platform was made possible when they purchased Bamboo Therapeutics from AskBio in 2016, that led to promising phase 1/phase 2 results for a potential Duchenne Muscular Dystrophy cure. As the company moved that research forward, they recognized the need to expand manufacturing capabilities for late-stage clinical and commercial supply.

Through AskBio's industry leading Pro10(TM) cell line licensed to Pfizer, AskBio is proud to play a role in its manufacturing initiatives. By relying on our AAV and Pro10 technology, we believe this announcement serves as another validation of AskBio's unrivaled gene therapy platform that includes an extensive capsid library, advanced manufacturing capability and synthetic promoter technology, the three components critical to safe, accurate and efficient delivery of gene therapies.

We celebrate Pfizer's foresight and commitment. Investments such as this bring us all closer to bringing cures to the millions of patients in need around the world, and we invite other scientists and research companies to join us in this mission.

To that end, we believe that it is important to recognize the truly innovative AAV discoveries made by Dr. Samulski, Dr. Xiao and others over the past three decades that have made potential treatments and other AAV therapies possible.

    --  Dr. Samulski's discovery of how the AAV could be safely used to deliver
        corrected genes to cells with genetic defects propelled one of the most
        exciting and inspiring fields in medical research today, now the
        foundation for an entire industry.
    --  Dr. Xiao Xiao was the first to create a mini-dystrophin gene that opened
        the door for the development of potential DMD therapies.
    --  Dr. Josh Grieger, AskBio's Chief Technology Officer, helped to pioneer
        the Pro10 producer cell line that is now paving the way for increasing
        the efficiency of developing and producing potentially curative
        therapies to reach all patients.

AskBio is on the forefront of the gene therapy industry not only with an unrivaled gene therapy platform, but also a robust pipeline of clinical stage therapeutics. The company's commitment to bring effective therapies to market is only rivaled by its commitment to reduce the cost of production and increase access with new innovative technology for advancing gene therapy development.

About AskBio

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), an extensive AAV capsid library and synthetic promoter technology. The company has generated hundreds of proprietary third generation gene vectors, several of which have entered clinical testing, and maintains a portfolio of clinical programs across a range of indications including Pompe, Limb Girdle Muscular Dystrophy, Cystic Fibrosis, Myotonic Muscular Dystrophy, Huntington's, Hemophilia (Chatham Therapeutics/Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics/Pfizer). For more information, visit www.askbio.com.

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SOURCE AskBio