RNA therapeutics are making a tremendous impact on medicine, recently exemplified by the rapid development and deployment of mRNA vaccines to combat the COVID-19 pandemic. In addition to vaccines, RNA-targeting drugs are being developed for diseases with significant unmet medical needs.

Recent advances in genome sequencing have revealed the genetic causes of many human diseases. This information is ushering in the modern era of DNA and RNA therapeutics. While DNA targeting can result in durable and potentially permanent cures, RNA therapeutics enable tunability and reversibility without permanent off-target changes.

Several challenges must be overcome in order to realize the therapeutic potential of RNA editing to patients. Beyond exquisite guide RNA engineering that enables highly efficient and specific RNA editing, we need to address issues of delivery and manufacturing that broadly impact the field of gene therapy. In addition, we need non-clinical and clinical assays to quantify editing efficiency and transcriptome integrity to establish safety metrics to support clinical development.

In collaboration with Prashant Mali’s lab at UCSD, we published an article in Molecular Therapy to review the progress in the field of therapeutic RNA editing, highlight recent research advances, and discuss the key challenges on the path of clinical development.   

Booth BJ, Nourreddine S, Katrekar D, Savva Y, Bose D, Long TJ, Huss DJ, Mali P. RNA editing: Expanding the potential of RNA therapeutics. Mol Ther. 2023 Jan 7:S1525-0016(23)00005-9. doi: 10.1016/j.ymthe.2023.01.005. Epub ahead of print. PMID: 36620962; PMCID: PMC9824937.

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23 Top Biotech Startups According to VCs (Business Insider, July 28, 2022)

Shape Therapeutics named one of Fierce Biotech’s “Fierce 15” Companies of 2022

Fierce Biotech’s 2022 Fierce 15 (Fierce Biotech, September 12, 2022)

Pushing Beyond Gene Editing Limits with Programmable RNA Medicines (Biospace, June 13, 2022)

Shape Therapeutics to Present Data Highlighting Advances in Its AI-Driven RNA Technology Platforms at the 2022 ASGCT Annual Meeting

Base Editing Marches on the Clinic (Nature Biotechnology, May 9, 2022)

Shape Therapeutics Welcomes Google and Stripe Veterans Gayathri Rajan, Kelly Sims to its Board of Directors

Two small biotechs hope that gene therapy will be the future of Alzheimer’s disease treatment. Here’s what we know so far. (Business Insider, January 25, 2022)

Shape Therapeutics to Present at the 40th Annual J.P. Morgan Healthcare Conference

RNA Editing Seeks Its Place in the Gene Editing World (Timmerman Report, January 5, 2022)

In editing RNA, researchers see endless possibilities (The Scientist, December 1, 2021)

Shape Therapeutics Appoints David J. Huss, Ph. D., as Chief Scientific Officer and Lisa Taylor Ash, J. D., as General Counsel

RNA-editing race intensifies as big pharma buys in (Chemical & Engineering News, October 23, 2021)

tRNA therapies could help restore proteins lost in translation (Chemical & Engineering News, September 19, 2021)

The Point of Base Editors: Correcting Point Mutations (Genetic Engineering & Biotechnology News (GEN), September 1, 2021)

Shape Therapeutics enters into a strategic research collaboration with Roche to advance breakthrough AAV-based RNA editing technology for neuroscience and rare disease indications

Shape Therapeutics raises $112M in bid to make RNA editing – and a whole lot else – a reality (Endpoints, July 15, 2021)

Shape Therapeutics Secures $112 Million in Series B Financing to Advance Breakthrough RNA Technology Platforms to Democratize Gene Therapy

Shape Therapeutics to Present the Discovery of Muscle-Specific AAVs at the Jefferies 2021 Virtual Healthcare Conference

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Shape Therapeutics Announces Appointment of Matt Valentino as Chief People Officer

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ShapeTX Expands Strategic Collaboration with Roche with Addition of a New Target

Spotlight Video: Dr. Adrian Briggs’ Next Scientific Puzzle

Spotlight Video: Where Science and Art Collide

Otsuka collaborates with ShapeTX to develop novel AAV gene therapies for ocular diseases

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Spotlight Video: Solving puzzles as a team

ShapeTX Announces the Appointment of Adrian W. Briggs, Ph.D. As Chief Technology Officer

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ShapeTX Announces Seven Presentations at ASGCT 2023 Showcasing the Potential of Generative AI and RNA in CNS Disorders and Beyond

ShapeTX at ASGCT 2023: Programmable Medicine Takes Center Stage

Spotlight Video: Charting New Courses

Dispatches from The Atlantic Progress Summit: How mRNA Technology Could Save the World

RNA editing: Expanding the potential of RNA therapeutics

ShapeTX to Showcase Groundbreaking Advances in CNS in vivo RNA Editing, CNS-Targeting AAVs, and TruStable™ AAV Manufacturing at the J.P. Morgan 41st Annual Healthcare Conference

Spotlight Video: Inspired by Nature

Spotlight Video: The Art of RNA

Building an RNA Toolbox

Repairing the genetic causes of disease

Big strides, new website, same core mission

23 Top Biotech Startups According to VCs (Business Insider, July 28, 2022)

Shape Therapeutics named one of Fierce Biotech’s “Fierce 15” Companies of 2022

Fierce Biotech’s 2022 Fierce 15 (Fierce Biotech, September 12, 2022)

Pushing Beyond Gene Editing Limits with Programmable RNA Medicines (Biospace, June 13, 2022)

Shape Therapeutics to Present Data Highlighting Advances in Its AI-Driven RNA Technology Platforms at the 2022 ASGCT Annual Meeting

Base Editing Marches on the Clinic (Nature Biotechnology, May 9, 2022)

Shape Therapeutics Welcomes Google and Stripe Veterans Gayathri Rajan, Kelly Sims to its Board of Directors

Two small biotechs hope that gene therapy will be the future of Alzheimer’s disease treatment. Here’s what we know so far. (Business Insider, January 25, 2022)

Shape Therapeutics to Present at the 40th Annual J.P. Morgan Healthcare Conference

RNA Editing Seeks Its Place in the Gene Editing World (Timmerman Report, January 5, 2022)

In editing RNA, researchers see endless possibilities (The Scientist, December 1, 2021)

Shape Therapeutics Appoints David J. Huss, Ph. D., as Chief Scientific Officer and Lisa Taylor Ash, J. D., as General Counsel

RNA-editing race intensifies as big pharma buys in (Chemical & Engineering News, October 23, 2021)

tRNA therapies could help restore proteins lost in translation (Chemical & Engineering News, September 19, 2021)

The Point of Base Editors: Correcting Point Mutations (Genetic Engineering & Biotechnology News (GEN), September 1, 2021)

Shape Therapeutics enters into a strategic research collaboration with Roche to advance breakthrough AAV-based RNA editing technology for neuroscience and rare disease indications

Shape Therapeutics raises $112M in bid to make RNA editing – and a whole lot else – a reality (Endpoints, July 15, 2021)

Shape Therapeutics Secures $112 Million in Series B Financing to Advance Breakthrough RNA Technology Platforms to Democratize Gene Therapy

Shape Therapeutics to Present the Discovery of Muscle-Specific AAVs at the Jefferies 2021 Virtual Healthcare Conference

Shape Therapeutics RNAfix® Editing Platform Achieves >90% Editing in Mouse and Non-Human Primate Brain

Shape Therapeutics Announces Appointment of Matt Valentino as Chief People Officer

Shape Therapeutics to Showcase RNA Editing in the Brain and Validation of their Stable AAV Producer Cell Line at ASGCT 2024

ShapeTX Expands Strategic Collaboration with Roche with Addition of a New Target

Spotlight Video: Dr. Adrian Briggs’ Next Scientific Puzzle

Spotlight Video: Where Science and Art Collide

Otsuka collaborates with ShapeTX to develop novel AAV gene therapies for ocular diseases

How AI will revolutionize drug discovery: ShapeTX CEO Francois Vigneault on Tech Nation

Spotlight Video: Solving puzzles as a team

ShapeTX Announces the Appointment of Adrian W. Briggs, Ph.D. As Chief Technology Officer

Generative AI is creating the next generation of therapies: programmable medicine

ShapeTX Announces Seven Presentations at ASGCT 2023 Showcasing the Potential of Generative AI and RNA in CNS Disorders and Beyond

ShapeTX at ASGCT 2023: Programmable Medicine Takes Center Stage

Spotlight Video: Charting New Courses

Dispatches from The Atlantic Progress Summit: How mRNA Technology Could Save the World

RNA editing: Expanding the potential of RNA therapeutics

ShapeTX to Showcase Groundbreaking Advances in CNS in vivo RNA Editing, CNS-Targeting AAVs, and TruStable™ AAV Manufacturing at the J.P. Morgan 41st Annual Healthcare Conference

Spotlight Video: Inspired by Nature

Spotlight Video: The Art of RNA

Building an RNA Toolbox

Repairing the genetic causes of disease

Big strides, new website, same core mission

23 Top Biotech Startups According to VCs (Business Insider, July 28, 2022)

Shape Therapeutics named one of Fierce Biotech’s “Fierce 15” Companies of 2022

Fierce Biotech’s 2022 Fierce 15 (Fierce Biotech, September 12, 2022)

Pushing Beyond Gene Editing Limits with Programmable RNA Medicines (Biospace, June 13, 2022)

Shape Therapeutics to Present Data Highlighting Advances in Its AI-Driven RNA Technology Platforms at the 2022 ASGCT Annual Meeting

Base Editing Marches on the Clinic (Nature Biotechnology, May 9, 2022)

Shape Therapeutics Welcomes Google and Stripe Veterans Gayathri Rajan, Kelly Sims to its Board of Directors

Two small biotechs hope that gene therapy will be the future of Alzheimer’s disease treatment. Here’s what we know so far. (Business Insider, January 25, 2022)

Shape Therapeutics to Present at the 40th Annual J.P. Morgan Healthcare Conference

RNA Editing Seeks Its Place in the Gene Editing World (Timmerman Report, January 5, 2022)

In editing RNA, researchers see endless possibilities (The Scientist, December 1, 2021)

Shape Therapeutics Appoints David J. Huss, Ph. D., as Chief Scientific Officer and Lisa Taylor Ash, J. D., as General Counsel

RNA-editing race intensifies as big pharma buys in (Chemical & Engineering News, October 23, 2021)

tRNA therapies could help restore proteins lost in translation (Chemical & Engineering News, September 19, 2021)

The Point of Base Editors: Correcting Point Mutations (Genetic Engineering & Biotechnology News (GEN), September 1, 2021)

Shape Therapeutics enters into a strategic research collaboration with Roche to advance breakthrough AAV-based RNA editing technology for neuroscience and rare disease indications

Shape Therapeutics raises $112M in bid to make RNA editing – and a whole lot else – a reality (Endpoints, July 15, 2021)

Shape Therapeutics Secures $112 Million in Series B Financing to Advance Breakthrough RNA Technology Platforms to Democratize Gene Therapy

Shape Therapeutics to Present the Discovery of Muscle-Specific AAVs at the Jefferies 2021 Virtual Healthcare Conference