The Drug Discovery World Podcast
DDW Highlights: 16 December 2024
Dec 16, 2024
This week highlights cutting-edge advancements in cell and gene therapies. There's exciting news about the first in vivo BCMA CAR-T candidate reaching clinical trials. Researchers are making strides against placental growth restriction, aiming to enhance maternal and child health. The podcast dives into groundbreaking cancer treatments, including promising trials for multiple myeloma. New research centers are emerging, focusing on gene therapies for heart disease and rare disorders, paving the way for future medical breakthroughs.
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Quick takeaways
- The recent discovery of AZD7648 reveals risks associated with CRISPR-Cas9 gene editing, highlighting the need for safer alternative methods.
- A promising gene therapy from the University of Florida aims to improve placental function, potentially reducing stillbirths and premature deliveries.
Deep dives
CRISPR-Cas9 Gene Editing Challenges
Recent research unveiled that a molecule named AZD7648, designed to enhance CRISPR-Cas9 gene editing efficiency, poses significant risks by unintentionally damaging other parts of the genome. While it was anticipated to streamline gene therapies, studies demonstrate that it can lead to extensive genetic changes and the deletion of thousands of DNA bases. The research team led by Jacob Korn highlighted that although localized edits appear correct initially, broader genomic analysis reveals considerable alterations. This revelation has prompted calls for alternative methods to mitigate these risks in the development of gene therapies.
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