Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...

Medicaid is doing a novel payment system for the new, promising and expensive sickle cell treatment. It may become a model ...

The AI model "Eden" is intended to generate new gene therapies for cancer and rare diseases, as well as active ingredients against multidrug-resistant bacteria.

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

This article is part of Nature Outlook: Skin, an editorially independent supplement produced with financial support from LEO Pharma. About this content. KaDee Troop is the mother of seven adopted ...

Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...

Urnov is a professor of molecular therapeutics at the University of California, Berkeley, and a director at its Innovative Genomics Institute. In May, news broke of a biomedical first: the on-demand ...

Recent advances in gene editing technology, which allows for targeted repair of disease-causing mutations, can be applied to hematopoietic stem cells with the potential to cure a variety of hereditary ...