Shape Therapeutics Unveils AAVid™ Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery Challenge in Gene Therapy

Key Messages:

• AAVid^TM platform screens massive diversity libraries of more than 1 billion unique AAV capsid variants in a single campaign for optimal tropism and manufacturability.
• ShapeTX confirms the identification of novel liver-tropic AAV5 variants.
• Data presented at the 2^nd RNA Editing Summit, Dec. 1-3, 2020, by David J. Huss, Ph.D., Vice President and Head of Research at ShapeTX.

(BUSINESS WIRE)-- Shape Therapeutics, Inc. (ShapeTX), a next-generation gene therapy company with an industry-leading RNA targeting technology platform, announces today the unveiling of the AAVid^TM capsid discovery platform and results from its first AAV5 variant library in a non-human primate selection campaign.

The AAVid^TM capsid discovery platform uses non-random mutational fitness to create massive capsid libraries of billions of unique AAV variants for direct-to-NHP in vivo biological selection. By combining cutting-edge DNA synthesis, advanced synthetic biology, next-generation sequence barcoding and machine learning algorithms, ShapeTX generates industry-leading library size and diversity to enable the development of best-in-class human therapeutics.

“Wildtype first-generation AAVs are enabling the recent advances in gene therapy, but they have been plagued by toxicities in the clinic due in part to a lack of tissue specificity, resulting in the need for high doses. Our AAVid^TM platform solves the issue by creating novel capsid variants with specific tissue-tropism,” said Francois Vigneault Ph.D., President and CEO at ShapeTX. “We’ve stayed quiet for the past three years while developing a superior AAV platform technology and are excited to announce that we have best-in-class AAV variants in hand. Today, we are announcing our novel liver-tropic AAV5 variants – stay tuned for more to come.”

David J. Huss, Ph.D., Vice President and Head of Research added, “The vast structural space for exploration at the AAV capsid/target cell interface necessitates enormous library size and diversity, which until now, has only been probed with capsid library sizes in the tens of thousands to millions. At ShapeTX, we set out to create a superior AAV capsid discovery platform with library sizes in the billions of unique variants, thereby maximizing the opportunity for novel virus/target cell interactions.” Dr. Huss presented the details of the platform at the 2^nd RNA Editing Summit on Dec. 2, 2020.

About Shape Therapeutics, Inc

Shape Therapeutics is a biotechnology company developing next-generation RNA-targeted therapies to treat the world’s most challenging diseases. The ShapeTX technology platform includes RNAskip™, a proprietary suppressor tRNA technology that enables premature stop codon readthrough; RNAfix^TM, a precision RNA editing technology using endogenous Adenosine Deaminase Acting on RNA (ADAR); and AAVid^TM, a next-generation engineered adeno-associated virus (AAV) platform producing highly specific, tissue-tropic AAVs. The power of the ShapeTX platforms resides in redirecting the cellular machinery already present in our cells, thereby bypassing the risks of immunogenicity and DNA damage seen with other contemporary editing technologies. ShapeTX is committed to data-driven scientific advancement, passionate people and a mission of providing life-long cures to patients. Shape Life!

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