Led discovery programs for delivery of potential therapeutic payloads. Spearheaded cell-type specific, targeted delivery programs. Conceived, planned and executed proof of concept experiments.• Generated decision-making dataset for using ARMMs to deliver protein degraders for therapeutic intervention (p-tau, KEAP1-Nrf2)• Generated decision-making dataset for using ARMMs to deliver tumor suppressor protein p53• Generated decision-making dataset for using ARMMs to deliver zing-finger nucleases (ZFN) or meganucleases• Established ARMMs as efficient vehicles for delivery of Cas9/gRNA and ABE8/gRNA genome editors• Achieved proof of principle for multiple gene editing therapeutics discovery programs with accelerated timelines: Charcot-Marie-Tooth disease type 1A/PMP22, Friedreich’s ataxia/FXN, Usher Syndrome/USH2A, Hemophilia/TFPI• Secured funding from Friedreich's Ataxia Research Alliance through grant writing and delivering milestone data; advanced the program to in vivo efficacy studies• Advanced hemophilia program to in vivo efficacy studies as a lead pipeline program• Advanced ocular Usher Syndrome program to test in human retina explants via CRO• Achieved targeted delivery to CD8-positive T cells ex vivo and in vivo by engineering ARMMs with CD8 engagers• Established an engineered in vitro testing platform for targeted delivery to proprioceptive neurons (Friedreich’s ataxia)• Led and coordinated manuscript preparation (https://www.biorxiv.org/content/10.1101/2024.09.24.614725v2)• Contributed to multiple patent applications• Delivered 4 first-author poster presentations at ASGCT2023, ASGCT2024• Selected for podium presentation that was scheduled at ICAR conference in November, 2024Publication:Wang Q*, Liu SL*, Zhao WN*, …., Nabhan JF. Engineering Human ARMMs as Therapeutic Non-Viral Vehicles for in vivo Delivery of Genome Editing Payloads. Submitted, 2024 (*equal contribution)

• Part of the management team of high-content imaging facility at the CDoT• Contributed to small molecule therapeutics development for a rare kidney disease, MUC1 Kidney Disease, from early discovery to in vivo efficacy studies • Contributed to CRISPR-Cas9 knockout genetic screen for misfolded pathogenic protein MUC1-fs degradation

Head, Therapeutic Screening Core, 2014-Group Leader, 2016-Associate Researcher, Stanley Center for Psychiatric Research, Broad Institute, 2010-1) Led drug discovery programs to develop novel therapeutics for CNS disorders via iPSC modeling and using iPSC-derived neural progenitors or neurons•Established histone acetylation assays that were integrated into the pipeline for novel HDAC inhibitor discovery program at the Stanley Center for Psychiatric Research•Completed large-scale high-throughput screening for Wnt signaling modulators (NIMH MLPCN & Broad Institute screening platform); hit and lead compound selection and validation; target identification via L1000 gene expression profiling•Developed and performed a high-content imaging assay and screen for lithium mimetics; in vivo efficacy studies via collaboration•Conducted mechanistic study of a pathogenic mutation in Disheveled protein in Schizophrenia (Stanley Center for Psychiatric Research)•Completed a R01 grant funded study to investigate the neuroprotective potential of fish oil component DHA•Led iPSC modeling of a NDD patient with CRISPR genome editing. Developed a gene expression ddPCR assay and initiated stop codon read-through therapeutics screening2) Led operation of Therapeutic Screening Core. Manage high-throughput screening facility. Manage small molecule compound libraries. Coordinate collaborations involving high-throughput screening and assay development. 3) Oversaw lab operation from 2013 to 20184) Supervised and mentored next-generation scientists including research assistants, visiting graduate students, Harvard undergraduate students, and summer internsHighlights: The John Dowling Prize for the best undergraduate senior thesis in Neuroscience at Harvard, 2018High Magna Cum Laude designation on senior thesis in Neuroscience at Harvard, 20205) Produced 6 first-author publications out of total 31 publications with Dr. Haggarty6) Delivered 3 podium presentations at conferences

• Identified potential phosphorylation sites modulating the disease-relevant SMN protein degradation for novel therapeutics development for the Spinal Muscular Atrophy (SMA)• Involved in using stem cell derived motoneurons for high-throughput screening of therapeutic compounds that enhanced survival of motor neurons in both SMA and ALS models• Characterized 15 human ES cell lines for their motor neuron differentiation potentials • Firsthand experience with confocal automated microscope OPERA and assisted image analysisPUBLICATION:Makhortova, N.R., Hayhurst, M., Cergueira, A., Sinor-Anderson, A.D., Zhao, W.-N., Heiser, P.W., Arvanites, A.C., Davidow, L.S., Waldon, Z.O., Stern, J.A., Lam, K., Ngo, H.D. and Rubin, L.L. A Screen for Regulators of Survival of Motor Neuron Protein Levels Nature Chemical Biology 7(8):544-552 (2011)