Shape Therapeutics Unveils AAVid ™ Capsid Discovery Platform to Discover New Tissue-Specific AAV Variants, Solving Fundamental Delivery Problems in Gene Therapy

7 December 2020

ShapeTX, a next-generation gene therapy technology company with the industry's leading RNA targeting technology platform, today launches the AAVid ™ capsid discovery platform And announced the results in the first AAV5 variant library in a selection campaign for non-human primates.

The AAVid ™ capsid discovery platform uses non-random mutant fitness to create a large library of billions of unique AAV variants, biology directly in vivo in non-human primates (NHPs). Realize the choice. ShapeTX is one of the largest libraries in the industry to combine state-of-the-art DNA synthesis, advanced synthetic biology, next-generation sequence bar coding, and machine learning algorithms to develop best-in-class human therapeutics. And realize diversity.

Dr. Francois Vigneault, President and CEO of ShapeTX, said: "Wild-type first-generation AAV has enabled recent advances in gene therapy, but toxicity is a source of concern in the clinic, partly due to lack of tissue specificity. Dosage is required. Our AAVid ™ platform solves this problem by creating new capsid variants with specific tissue orientation. We have been refraining from publishing for the past three years. However, in the meantime, we are thrilled to announce that we have developed a superior AAV platform technology and obtained the best-in-class AAV variant. We today announced our new hepatotrophic AAV5 variant. Please pay attention to future announcements. "

Dr. David J. Huss, Vice President and Research Head of ShapeTX, said, "The vast structural space for exploring AAV capsid / target cell interfaces requires the size and diversity of large libraries. However, it has only been explored in tens of thousands to millions of capsid libraries. ShapeTX's plan is to create an excellent AAV capsid discovery platform with a library scale of billions of unique variants. , Maximize new interactions between virus / target cells. " Dr. Huss presented the details of the platform at the 2nd RNA Editing Summit on December 2, 2020.

About Shape Therapeutics

Shape Therapeutics is a biotechnology company that develops next-generation RNA-targeted therapies to treat the world's most incurable diseases. ShapeTX's technology platform includes RNAskip ™, a proprietary suppressor tRNA technology that enables skipping of immature stop codons, and RNAfix, a precise RNA editing technology that uses endogenous ADAR (adeno-associated aminase that acts on RNA). ™ includes AAVid ™, a next-generation artificial adeno-associated virus (AAV) platform that produces highly specific and tissue-tropic AAV. The reason each ShapeTX platform is so powerful is that it redirects the cellular mechanisms that already exist within our cells, avoiding the risks of immunogenicity and DNA damage found in other editing techniques today. There is. ShapeTX is committed to providing patients with data-driven scientific advances, passionate talent, and lifelong healing. We build the lives and lives of our patients (Shape Life!).

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Contacts
Shape Therapeutics, Inc.
Investors & Media
John Suliman
CFO & Head of Business Strategy
John@shapetx.com