10-year CRISPR anniversary: How gene editing revolutionized medicine, and what lies ahead – Genetic Literacy Project

Posted: October 8, 2022 at 4:03 pm

Ten years ago, a little-known Science paper authoredby Jennifer Doudna, Ph.D., and Emmanuelle Charpentier, Ph.D., proposedusing CRISPR/Cas9 for gene editing. As the first wave of gene-editing-based therapies post clinical data and head to the FDA, biopharma executives at the forefront of the burgeoning field highlighted the innovations and challenges before gene editing is ready for prime time.

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Now, some of the early efforts of turningCRISPRgene editingtechnologyinto viable therapies are coming to fruition. Vertex and partner CRISPR Therapeutics justannouncedtheir plan to file their CRISPR/Cas9-edited cell therapy exagamglogene autotemcel (exa-cel) for a rolling review at the FDA in sickle cell disease and beta thalassemia starting in November. If approved, exa-cel could be the first CRISPR-based therapy available.

Meanwhile, Intellia Therapeutics, after being thefirst to showthat systemic infusion of CRISPR inside the human body could treat disease, recentlyreportedmore positive early data for its in vivo gene editing candidates for transthyretin amyloidosis (ATTR) and hereditary angioedema.

For now, most gene editing projects are focused on rare diseases and some blood or cancer indications with well-established genetic drivers. Those diseases have clear clinical endpoints and risk-benefit understanding to allow for a quick drug development path.

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10-year CRISPR anniversary: How gene editing revolutionized medicine, and what lies ahead - Genetic Literacy Project

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