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.
AERS Mine, A novel approach to clinical trial design, toxicology prediction, identifying polypharmacy confounders, and drug repurposing by datamining and synthesizing information from extensive drug, disease, and adverse effect databases
Therapy for neuronopathic Gaucher Disease using gene therapy and a specialized neuronal lineage iPS cell that facilitates entry to and engraftment in the brain.
Cloud-based, HIPAA-compliant clinical decision-support platform that brings pharmacometrics and model-informed precision dosing directly to the bedside.
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.
Quantitatively identifying diffuse white matter anomalies using MRI as a systematic approach provides early identification of the need for developmental deficit interventions in preterm infants.
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.
Children with medical complexity account for a significant portion of healthcare spending and most care occurs at home, with limitations in current digital tools.
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.
VR3S provides an interactive environment for surgeons to visualize, manipulate and plan complex repairs using patient-specific 3D models. Planning is enabled on-screen or in virtual reality, along with training and remote case consultation.