A novel non‑viral, non‑lipid delivery system enables safe transfer of large genetic payloads, avoiding key limits of current gene therapies and improving uptake using targeted siRNA or drug-based enhancers.
Therapy for neuronopathic Gaucher Disease using gene therapy and a specialized neuronal lineage iPS cell that facilitates entry to and engraftment in the brain.
Next-generation orally bioavailable degrader programs targeting DUSP1 and NFKB1 suppress JAK-STAT and NFκB signaling to restore treatment sensitivity in relapsed and refractory leukemia and potentially other cancers.
Engineered lentiviral platforms leveraging Myomaker/Myomerger-mediated muscle targeting overcome key limitations, enabling delivery of large genetic payloads with the potential for reduced immunogenicity and repeat dosing in muscular dystrophies.
Pharmacologic and genetic inhibition of UBE2N suppresses the function and viability of MDS/AML cells lines and patient samples. A commercially available and a novel compound has been identified.
A newly established link between the mechanistic role of thrombin and fibrinogen and their contribution to AD provides a novel mechanism for oral treatment.