Gene and Cell Therapy – News and Features

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.

UCSF's clinical trial uses CRISPR-Cas9 gene editing to directly correct the genetic mutation causing sickle cell disease. By modifying patients' own blood stem cells without viral vectors, the therapy offers a safer alternative to donor transplants.

Researchers developed a gene therapy to enhance placental function by delivering IGF-1 DNA using nanoparticles. Tested in rhesus macaques, the therapy improved placental health without adverse effects.

Using adeno-associated viruses (AAVs), scientists mapped neural circuits in frogs, tracing changes during metamorphosis. This method reveals how amphibian nervous systems adapt as locomotion transitions from swimming to walking.

New research has illustrated how hepatitis A virus uses cellular proteins, like PDAP1, to infect liver cells, replicate and spread.

RNA therapeutics promise rapid, personalized and flexible treatments against multiple diseases. In this article, we will explore whether RNA therapeutics can realize their full potential.

A new gene editing therapy for a rare heart condition has shown promise in an early trial.

A novel CAR-T therapy targeting p95HER2 shows complete and durable tumor responses in HER2+ breast cancer preclinical models. Combining CAR-T cells with a bispecific antibody, this approach enhances immune activation while avoiding toxicities.

Researchers developed a dual-AAV gene therapy for Usher syndrome type 1F, restoring hearing and balance in mice. The approach effectively delivered the large PCDH15 gene and showed promise in retinal organoids and nonhuman primates.