The largest study to date to analyse millions of both genetic and patient records on the long-term health conditions of later life has identified opportunities for new ways to prevent and treat ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

The second person ever has received a transplant with a genetically modified pig kidney.

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying ...

UMD researchers have discovered key mechanisms in gene regulation that could improve the design of RNA-based medicines.

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for ...

Why are CRISPR companies not OK? Will Robert F. Kennedy Jr. keep his promises if he's confirmed to lead the Department of ...

Gene editing promised a revolution, but biotech layoffs, stock slumps, and industry struggles reveal a stark reality: CRISPR ...

The FDA has granted orphan drug designation (ODD) and RPDD to Arbor Biotechnologies' ABO-101, to treat primary hyperoxaluria ...

Treatment for sickle cell disease, the most common inherited blood disorder in Kenya, has been approved for use in England for older children and adults with a severe form of the disease.The treatment ...

The management of Leishmania donova ni (LD), responsible for fatal visceral leishmaniasis (VL), faces increasing challenges due to rising drug-unresponsiveness, leading to increasing treatment ...