Ron (Guanrong) Huang

•Design and implement assay development strategies to support exploratory research activities and mid-stage preclinical developments. Serve as a team lead and key contributor for project transitions. •Advise on immunogenicity, biomarker, AAV analytical assay development efforts.

•Led in vitro potency release assay development effort. Generated key in vitro/in vivo expression data to triage novel vector/transgenes. •Developed a screening assay for a DMD target. •Managed in vivo study at CROs, achieved key PoC. •Provided due diligence for BD activities.

•Led proof-of-concept and efficacy animal studies in cross-functional teams, for the development of multiple pre-clinical candidates, early development compounds, and novel drug targets in MS and ALS. One compound reached phase II clinical trials, one compound reached phase I clinical trials, two reached no-go decisions post lead optimization, and multiples are in exploratory or Hit-ID stages. •Led the screening effort for remyelination compounds using high content imaging (IncuCyte, ArrayScan) and ELISA/MSD-based assays (Oligodendrocyte differentiation). Identified multiple hits and advanced into animal studies. •Championed a new target for MS with reducing oxidative damage as mechanism of action. •Managed animal studies with CROs. •Provided due diligence for BD opportunities

•Led animal efficacy studies to test remyelination compounds for ALS treatment. Developed multiple efficacy readouts for ALS mouse model SOD1-G93A, such as electrophysiology (CMAP), immunohistochemistry (neuromuscular junction), and fluid biomarker (serum/CSF neurofilament). •Led animal efficacy studies to validate/screen novel targets/compounds for CNS/peripheral nerve regeneration using optic and sciatic nerve crush models. Developed histology efficacy readouts such as nerve axon labeling and skin IENF. One target reached lead ID stages. •Developed cell-based assays to study MOA/SAR, screen IHC antibody. •Managed human tissue procurement for the lab, and evaluate target expression in diseased human and animal tissues by immunohistochemistry.

• Developed a FACS based biomarker assay (immune cells activation) for inflammatory disease program. • Established a biomarker assay (apoptosis) to define the biologically active dose range for internal compounds. • Set up a MSD based biomarker assay (cytokine profile) for pain targeted therapeutics.

• Investigated the mechanism by which cargo proteins are targeted to glucose storage vesicles. Demonstrated that insulin-regulated aminopeptidase (IRAP, a major GSV cargo protein) enters GSVs by its luminal domain interaction with Glut4. • Demonstrated that sortilin (another major cargo protein of GSVs) drives the formation of GSVs, sortilin might also confer insulin responsiveness to GSVs. Identified sortilin binding protein, long-chain acyl-CoA synthetase (ACSL), showed that ACSL is also important for GSV formation. • Assisted in screening proteins that promote Glut4 translocation to the cell surface.

• Conducted market research on technologies arising from Boston University faculty research, assessed the readiness of technologies, potential for market acceptance, market size, relevant companies, and potential licensees. • Conducted prior art searches on related patents. • Interviewed industry experts and inventors.