One of the assets shelved by Eli Lilly is a gene therapy for dementia, which it obtained in its $1.04-billion acquisition of ...

Administration in the mid-to-high dose cohorts of the gene therapy trial will pause pending further evaluation.

The first clinical attempt to make human beings biologically younger is no longer a thought experiment. A small gene therapy trial, cleared quietly by regulators in the United States, is now moving ...

The development of a brain tumor in a study participant led regulators to suspend a Hurler syndrome therapy in early testing ...

The FDA has placed clinical holds on a pair of investigational gene therapies developed to address two rare neurodevelopmental disorders in children, according to the manufacturer. In a press release, ...

The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial.

Intellia Therapeutics is pioneering in vivo CRISPR gene editing, aiming for groundbreaking therapies, with NTLA-2001 showing promising results in ATTR amyloidosis, potentially achieving blockbuster ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort. Delandistrogene ...

Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...

Precision DNA editing gene therapy achieves deep remissions in aggressive leukemia previously considered incurable.

Case Western Reserve University chemist Divita Mathur was awarded a National Science Foundation (NSF) Faculty Early Career Development Program (CAREER) grant for her research in synthetic DNA ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London ...