Daniel McCoy

• Preclinical lead overseeing gene therapy programs in the metabolic and neurological disease spaces. • Work closely with program SMEs, internal CMC, Regulatory, Clinical, and C-suite leadership to ensure safe and timely advancement of preclinical assets to IND. • Responsible for the design and execution of in vivo proof of concept and pivotal IND-enabling pharmacology/biodistribution/safety studies for AAV-based gene therapy products

• Preclinical program scientific lead tasked with supporting the development of gene therapy products from vector design through IND-enabling in vivo studies • AAV vector design/optimization, small scale viral production/process development, preclinical assay development, in vivo POC/ROA/DRF/Tox study design and biodistribution support • Primary point of contact responsible for early stage program vector design and optimization

• Lead the Platform Development team (3 direct report career scientists) • Responsible for the design and evolution of synthetic AAV capsids for various gene therapy indications (CNS, Liver, Eye – related) • Responsible for the development of novel molecular and cell-based assays and SOPs to meet corporate goals and milestones • One of the first two lab-based hires of the company, in charge of setting up lab R&D operations • Part of the management team and intimately involved in the hiring and growth of the company (from 2 – over 25 full time employees)

• Developed resident immune cell polarization assays in human ex vivo skin to model inflammatory skin disease states and support the development of relevant therapeutics • Developed a lipid gene based endpoint assay to evaluate candidate molecules targeting lipogenesis-related pathways • Contributed to both biomarker strategy, and transcriptomic disease signature study design/execution/characterization interfacing with external vendors and the GSK Computational Biology group • Contributed to the target engagement data package for the candidate selection (CS) milestone for a GSK asset • Designed proposals, wrote protocols, and managed external CRO contracts and academic collaborations

• Developed complex plate-based primary neuronal co-culture assays and assay endpoints for neurodegenerative disease research. In these cultures, primary cortical and striatal neurons are cultured together on a bed of primary glia. The individual isolation of the cell types prior to culturing allows for cell-type-specific responses to be measured simultaneously in a context that better reflects their interactions in vivo. • Screened novel therapeutics for toxicity and target engagement for pathways relevant to Huntington’s Disease

“The expected and unexpected roles of TRPM8: Cold Pain and Metabolism” Project: 1) TRPM8 pore dilation can deliver therapeutics to cold-pain sensing neurons 2) Enhanced insulin clearance in mice lacking functional TRPM8 channels • Utilized extensive molecular cloning, live cell imaging, and mouse in vivo techniques to independently develop 6 unique transgenic mouse models for studying the roles of Transient Receptor Potential (TRP) ion channels in pain • Characterized a channel pore dilation model in vitro demonstrating that cationic therapeutics can be delivered to cold-pain sensory neurons in a highly specific manner. Results particularly novel and impactful as it was previously thought to not be possible using this technique. • Discovered and published a novel insulin clearance phenotype for the thermosensory channel TRPM8 with therapeutic implications in regards to the treatment of diabetes and other metabolic disorders

• Worked in the Bio-therapeutics department supporting the development of ACAM529, a preclinical vaccine against HSV-2 (genital herpes) • Supported both formulation and process development studies carrying out viral plaque assays and data quantification • Developed a chemiluminescent Southern blot protocol for identifying modified viral HSV-2 DNA in complementing cell lines • Carried out optimization studies for in house viral plaque assay SOP and presented findings to the ACAM529 team