This Week’s CGT News: Base Editing Takes Aim at Cholesterol
Objective:
To report on recent advancements in genetic therapies targeting cholesterol and related conditions, including specific therapies and their mechanisms.
Key Findings:
- VERVE-102 showed dose-dependent reductions in PCSK9 and LDL-C with durable effects.
- SynCav1 demonstrated neuroprotective effects in mouse models, preserving learning and memory.
- Lilly's acquisition of Engage Bio aims to enhance non-viral DNA delivery systems.
- Korecyte Bio's CAR-T platform targets solid tumors with reduced off-target toxicity.
- United Immunity's approach aims to develop in vivo CAR-M therapies without ex vivo manufacturing.
- Cartesian's Descartes-08 is an outpatient CAR-T therapy with data expected in 2027.
- Scribe's STX-1150 aims for long-lasting LDL-C lowering through CRISPR technology.
Interpretation:
Limitations:
Conclusion:
