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Inherited genes could help make CAR T therapies safer and more effective Analysis of two pivotal Yescarta clinical trials revealed that patients' germline DNA influences both toxicity risk and CAR T expansion, opening new opportunities for smarter cell therapy design. Written byBree Foster, PhD

Inherited genes could help make CAR T therapies safer and more effective Analysis of two pivotal Yescarta clinical trials revealed that patients' germline DNA influences both toxicity risk and CAR T expansion, opening new opportunities for smarter cell therapy design. Written byBree Foster, PhD https://www.drugdiscoverynews.com/inherited-genes-could-help-make-car-t-therapies-safer-and-more-effective-17405?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz--OP0iwRY17IhELkfD401G13cRlpE8GxfwbTQ8wqGXvvgUo5oiJu7-Z3VXYca-mtRDx2tjtgr9LiPQMG2YAJFVBBE0nlg&_hsmi=444660716&utm_content=444660716&utm_source=hs_email Since the first CAR T therapy won approval in 2017, these engineered immune cells have transformed treatment for several blood cancers. However, despite promising responses in many patients, CAR T remains unpredictable. Some patients experience severe cytokine release syndrome (CRS) or neurotoxicity, while others tolerate treatment well. Likewise, the extent to which CAR T cells expand after infusion, a key driver of therapeutic success, varies widely between individuals. In a study published in Science Immunology, investigators analyzed whole-genome sequencing data from patients enrolled in Kite Pharma's pivotal ZUMA-1 and ZUMA-7 clinical trials of axicabtagene ciloleucel (axi-cel), marketed as Yescarta. Their findings suggest that inherited genetic variants can shape both the safety and performance of CAR T therapy, offering new ways to predict toxicity and engineer improved cell therapies.

A new blueprint for the rational design of siRNA drugs For the first time, researchers imaged a human protein essential to the RNA interference process and found ways to design better siRNA drugs. Written byAllison Whitten, PhD

A new blueprint for the rational design of siRNA drugs For the first time, researchers imaged a human protein essential to the RNA interference process and found ways to design better siRNA drugs. Written byAllison Whitten, PhD https://www.drugdiscoverynews.com/a-new-blueprint-for-the-rational-design-of-sirna-drugs-17296?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-_8FqU5l4UXH7xA7y7xND1dOWXaaGbCkGgQWZPBEmH3TqxmpWDa8zLKC7houKjjgDRqf159BsDlRsXA-8lqFVlXC7Xq6A&_hsmi=444660716&utm_content=444660716&utm_source=hs_email Small interfering RNA (siRNA) drugs take advantage of RNA interference (RNAi) to “silence” genes with major advantages: They can target any gene without changing its underlying DNA. But designing these drugs has been difficult because so many different sequences of siRNA must be tested to find out which one works best to silence the target. So far, drug developers have been in the dark about why certain siRNA sequences work so much better than others, or how to predict which ones will be successful.

FDA announces new initiative for rare diseases Positive Alzheimer's trial data, workforce reductions, mRNA cancer therapy updates, and more led the news this week. Brought to you byDDN editorial team

FDA announces new initiative for rare diseases Positive Alzheimer's trial data, workforce reductions, mRNA cancer therapy updates, and more led the news this week. Brought to you byDDN editorial team https://www.drugdiscoverynews.com/fda-announces-new-initiative-for-rare-diseases-17591?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-_0WsEXv4_HPLhEO2oRpsUuhBRW69o_BGqi7g3XEGAZaBNoMYKGclt4cnQWrnVNKcn0thGX7v7yN2hY2yoXu7HDEbodVw&_hsmi=444660716&utm_content=444660716&utm_source=hs_email The FDA has unveiled a new Rare Disease Framework Initiative designed to “reimagine the development of drugs and biologics for rare diseases,” according to the press release. Recognizing that only five percent of rare diseases having an FDA-approved treatment, the announcement describes how the new initiative seeks to include patients’ voices, build shared resources on rare disease drug development and review, and champion the use of cutting-edge science, among other commitments. As outlined in the new white paper accompanying the framework, “The goal is a regulatory framework where fit-for-purpose evidentiary expectations are the norm for all rare disease applications; the FDA will work closely with sponsors and patients to support innovative approaches to generating evidence that will meet the FDA’s rigorous statutory standard of safety and effectiveness.” – Allison Whitten