Gene and Cell Therapy – News and Features

This article explores some of the challenges in developing cell lines for therapeutics, such as cell line stability, and how they are being addressed.

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model.

New potential therapeutic targets have been identified for diabetic kidney disease, the leading cause of kidney failure worldwide.

Researchers used glioblastoma-derived organoids to predict patient responses to CAR T cell therapy with remarkable accuracy. These organoids mirrored the tumor’s reaction to treatment in real time, offering insights into efficacy and neurotoxicity.

UCSF researchers have engineered T cells to temper overactive immune responses by targeting inflammation and soothing immune activity. The technology successfully protected transplanted pancreatic islet cells in a type 1 diabetes model.

Researchers have uncovered surprising ways transcription factors – the genetic switches for genes – regulate plant development, revealing how subtle changes in a lipid-binding region can dramatically affect growth.

Researchers have built a synthetic receptor with broad potential to program cell activity, including immune response and neurological signaling.

The therapy molecule AZD7648 can promote precise DNA repair during CRISPR gene editing, but creates massive genetic changes elsewhere.

A novel CAR T-cell therapy has benefitted patients with relapsed acute lymphoblastic leukemia, showing high response rates in a Phase Ib/II trial.

Dr. Eric Kelsic, CEO and co-founder of Dyno Therapeutics, explains how AI could reduce the cost of gene therapy delivery to $0.