Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.
An Ultragenyx gene therapy has scored a win at the midway point of its phase 3 trial, successfully reducing ammonia levels in patients with a rare urea cycle disorder.
Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...
A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...
Proof-of-concept trial in a single patient shows that cells can survive transplantation without immunosuppression ...
Since scientists first discovered that human immune cells could be modified to become cancer-fighting agents, they've been trying to engineer a cell that's effective against solid tumors, which ...
Fractyl Health, Inc. announced promising preclinical results for its gene therapy RJVA-001 at the 2025 ASGCT Annual Meeting, showing that a single dose leads to substantial metabolic improvements ...
Retiree Chip Hailey, who has a severe form of hemophilia B, participated in a groundbreaking gene therapy trial. Hailey experienced a life-threatening delay in treatment after a car accident, ...
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
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