Rocket Pharmaceuticals acknowledged the death of a patient in a pivotal trial assessing its Danon disease gene therapy candidate RP-A501, a study that the FDA has placed on clinical hold.

Scientists have used personalised gene-editing therapy to successfully treat a baby with a rare genetic condition. Could this ...

A research team led by Associate Professor Makoto Ikeya in the Department of Clinical Application at Kyoto University has ...

Researchers have identified a promising strategy to improve memory and brain cell function in models of 22q11.2 Deletion ...

Princeton University awarded honorary degrees to Omar M. Yaghi, Daniel Chee Tsui, Nancy Weiss Malkiel, Joshua ...

Typically, the goal of ALS treatments is to slow the disease or halt progression. But new data from the antisense oligonucleotide (ASO) jacifusen for FUS-ALS show that two patients went beyond the ...

The next-gen immunotherapies market is evolving with biotech advances, rising cancer cases, new therapies, funding, and ...

Rui Tostoes, Chief Technology Officer at ImmuneBridge, shares how his team is redefining preclinical development and ...