·Lead the development of Clinical Development Plan (CDP) for assigned projects

·Develop clinical study protocols and implementation plan, monitor study execution and respond to the medical questions

·Be a key member to drive the IND/NDA submission and approval. Working closely with Regulatory Affairs to support regulatory communications with the agency

·Liaison with KOLs, clinical investigators, clinicians, scientists, and experts in clinical development activities, including but not be limited to, strategy, trial design, result interpretation and communication

·Oversee and manage external service providers

·Support clinical data review and analyses

·Support preparation of study related documents

·Closely work with internal groups like Process Development, Research, Business Development, Legal, etc. to ensure the alignment and progress of the projects

·Participates in CRO and other vendor selection, negotiation and management, including monitoring of associated budgets and contracts

·Execute and deliver assigned studies according to business needs (timeline and cost) and quality requirements as defined in the study protocol

·Ensure the quality of the data

·Perform co-monitoring visits, if appropriate

·To be responsible for working to coordinate for audits and take responsibility for the responses and corrective actions to audit finding

·Provides training and coaching to CRAs regarding all aspects of the clinical trial process

·Proactively identifies and resolves issues and participates in process improvement initiatives as required

·Performs other duties as assigned

·Evaluate potential sites and provide PM with recommendations for selection of qualified sites

·Conduct site initiation, monitoring and site close-out visits in compliance with monitoring plan, ICH-GCP, SOPs, local regulations and other applicable project-specific plans

·Prepare and manage the clinical trial agreements with investigators/sites

·Monitor and report the project status at the sites in a timely manner

·Maintain essential documentation in compliance with ICH-GCP, local regulations both in the office and at site

·Manage study supplies (ISF, CRF, etc.), drug supplies and drug accountability at study sites

·Assist the investigator for auditing/inspection of project

·Ensure completeness and quality of data in the investigational sites

·Proactively identify study-related issues and escalates as appropriate

·Performs other duties as assigned

·Execute in vivo studies using different animal models following protocols and study designs

·Perform animal necropsies including perfusion, tissues and blood collections, knowledge of tissues processing for IHC and in situ hybridization will be a plus

·Analyze and interpret results with high quality and reproducibility in a timely manner

·Participate in discussions in cross-disciplinary team and program meetings

·Contribute to writing study reports, study protocols, SOPs, and regulatory filings

·Finish the other tasks assigned by the supervisor

·Design, construct, and optimize AAV transgenes for various pipeline programs

·Characterize the expression and activity AAV transgene in cells and animal tissues, optimize the transgene based on program need

·Develop and validate assays for characterizing the products encoded by AAV transgenes

·Plan and write protocols for in vitro characterization of AAV vectors

·Carry out small scale production of AAV vectors using triple transfection for both in vitro and in vivo studies

·Perform AAV vector purification, evaluate AAV vector quality by qPCR, silver staining, denaturing gel, and other assays

·Write, execute, and troubleshoot protocols, adapt new protocols into practice

·Analyze data from studies, summarize them and write study reports, and present results in various meetings

·Finish the other tasks assigned by the supervisor

·Execute in vivo studies using different animal models following protocols and study designs

·Perform animal necropsies including perfusion, tissues and blood collections, knowledge of tissues processing for IHC and in situ hybridization will be a plus

·Analyze and interpret results with high quality and reproducibility in a timely manner

·Participate in discussions in cross-disciplinary team and program meetings

·Contribute to writing study reports, study protocols, SOPs, and regulatory filings

·Finish the other tasks assigned by the supervisor

·Execute in vivo studies using different animal models following protocols and study designs

·Perform animal necropsies including perfusion, tissues and blood collections, knowledge of tissues processing for IHC and in situ hybridization will be a plus

·Analyze and interpret results with high quality and reproducibility in a timely manner

·Participate in discussions in cross-disciplinary team and program meetings

·Contribute to writing study reports, study protocols, SOPs, and regulatory filings

·Finish the other tasks assigned by the supervisor

·Support method development, qualification, and transfer of biochemical, immunological, and molecular methods relevant for gene therapy products

·Design and execute experiments to support development of qualitative and quantitative assays for gene therapy process development as well as product characterization

·Conduct experiments to support vector characterization, in-process material testing, and stability studies in a timely manner

·Lead the development of robust assays in support of drug substance and drug product manufacturing and release testing

·Support analytical method investigations and troubleshooting

·Provide regular updates to internal team members and write SOPs, development reports, and characterization protocols, qualification protocols and reports as needed

·Maintain laboratory activities according to GMP compliance as needed

·Train colleagues and associates when needed

·Execute in vivo studies using different animal models following protocols and study designs

·Perform animal necropsies including perfusion, tissues and blood collections, knowledge of tissues processing for IHC and in situ hybridization will be a plus

·Analyze and interpret results with high quality and reproducibility in a timely manner

·Participate in discussions in cross-disciplinary team and program meetings

·Contribute to writing study reports, study protocols, SOPs, and regulatory filings

·Finish the other tasks assigned by the supervisor

·Perform routine tests in a GMP QC lab, such as ELISA, RT-PCR, ddPCR, HPLC-based assays, viral infectivity, routine maintenance of adherent and suspension cell cultures and various cell-based assays as required

·Generate internal and external documents such as analysis report, COAs, summary of reports, and SOPs

·Document writing (laboratory investigations, out of specifications, deviations, CAPAs), data interpretation/presentation, statistical analysis, and trending analysis

·Work closely with Manufacturing and Quality Assurance staff to resolve quality issues, including experiment troubleshooting, and data interpretation

·Work on project teams developing and improving assays

·Training other QC scientists through established on-the-job training processes

·Additional duties as assigned; work under general supervision to meet project goals

·Execute in vivo studies using different animal models following protocols and study designs

·Perform animal necropsies including perfusion, tissues and blood collections, knowledge of tissues processing for IHC and in situ hybridization will be a plus

·Analyze and interpret results with high quality and reproducibility in a timely manner

·Participate in discussions in cross-disciplinary team and program meetings

·Contribute to writing study reports, study protocols, SOPs, and regulatory filings

·Finish the other tasks assigned by the supervisor