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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Gene Editing Molecule's Destructive Side Effect
- A molecule intended to boost CRISPR gene editing, AZD7648, caused significant unintended genomic damage.
- Researchers advise finding alternatives, as this discovery hinders gene therapy development.
Gene Therapy for Placental Growth Restriction
- Placental growth restriction affects many pregnancies, especially in developing countries.
- A new gene therapy aims to reverse this by increasing insulin-like growth factor 1 and vascularization.
In Vivo CAR-T Therapy for Multiple Myeloma
- ESO-T01, a new in vivo BCMA CAR-T therapy for multiple myeloma, has entered clinical trials in China.
- Preclinical studies showed promising anti-tumor activity and durable CAR T-cell generation.