WVE-004

MeiraGTx presented four posters at ASGCT 2025, showcasing advancements in gene therapies for severe pediatric obesity, ALS, and frontotemporal dementia, highlighting their innovative approach to addressing significant unmet medical needs.

NKGen Biotech's innovative NK cell therapy troculeucel has received FDA Fast Track Designation for treating moderate Alzheimer's disease, potentially accelerating its development pathway.

Wave Life Sciences' WVE-006 demonstrates successful RNA editing in early trial for alpha-1 antitrypsin deficiency (AATD).

Passage Bio has received positive feedback from the FDA to expand its phase 1/2 upliFT-D clinical trial to include patients with frontotemporal dementia (FTD) due to C9orf72 mutations, following encouraging data from the trial's first cohort. The gene therapy PBFT02 aims to increase progranulin levels in the central nervous system, showing promising results in initial clinical data.

Passage Bio reports promising progress in its phase 1/2 clinical trial for a genetic form of FTD, with updated interim data showing elevated progranulin levels in patients. The company plans to dose a second cohort in 2024 and expects further data by 2025.