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KK

Katalin Kauser, PhD, MD, Sc.D

SVP, Translational Biology

Metagenomi

Metagenomi Pipeline

DrugIndicationPhase
MGX-001Severe Hypertriglyceridemia (via APOC3 knockout)Preclinical
Type V-K CAST SystemSite-specific integration of therapeutic transgenes (e.g., Factor VIII for Hemophilia)Preclinical
Ultra-Compact Type V NucleaseIn vivo genome editing (all-in-one AAV delivery)Research
Compact Type II CRISPR SystemCNS gene knockdown via AAV deliveryResearch
Metagenomics-derived Base EditorHuman therapeutic applicationsResearch
Engineered Adenine Base EditorsNot specifiedResearch