Gene editing for rare diseases using Crispr-Cas9 and AAV

GCT CMC executive

Head of Quality, in charge of CDMO organization’s QA/QC/Validation for Gene and Cell Therapy Products

• Participate in regular technical development team meetings and provide clear and timely feedback to the teams.• Communicate timelines and issues to all relevant Quality departments in a timely manner.• Work with groups within and outside Quality as well as external partners to ensure production, testing and release, and regulatory submission timelines are achieved without compromising product quality.• Communicate effectively and efficiently at different levels of the organization and at meetings.• Influence and facilitate groups with diverse perspectives. Be proactive in raising quality issues to the technical development teams and/or management for discussion.• Work with analytical and quality control group on specification establishment based on scientific data and rational, participate specification review committee meeting, conduct quality and TDT assessment for the drug substance and drug product specifications.• Work with analytical and quality control group on drug substance and drug product expiry memos approval and update based on available stability data to support clinical studies.• Work with formulation group on drug product formulation record generation and approval. Generate and update the formulation information in Roche’s database Roformis for small molecule projects assigned.• Work with appropriate SMEs in CMC functional groups and present product investigations to quality review board.

PAI readiness program:- Review ANDA submissions up to 15 projects;- Review related ANDA amendments and supplements;- Review related API specifications;- Review related excipients specifications;- Review related finished product specifications;- Review related master batch records;- Review related product development reports- Review stability chamber qualification and PM/Cal program;- Review manufacturing equipment qualification and PM/Cal program:- PAI readiness, gap analysis, and remediation based on documents and programs reviewed.

Led and managed group of scientists and associates that developed, qualified, and validated methods for characterization of pharmaceuticals, including drug substance and drug product, in development organization. Created methods, including API, formulation, and aerosol characterization methods, as well as characterization data used to support formulation development, process development, and manufacturing efforts as well as for release of GMP or GLP samples. Directed internal analytical development team in supporting routine testing during early and late stage of development. Served as the SME to support late-stage product of company.Provided analytical expertise to support management of five CMOs/CROs, including analytical method development, method validation, testing data review, material qualification, process validation, and preparation for launching an MDI commercial product. Served as the SME of bioanalytical data, including method development and validation data, and bioanalytical study protocols and reports. Created source technical documents and assisted in preparation of reviews.

Heading Micropoint Bioscience's global quality and Regulatory functions.

Heading Alexza's Quality Control department for 7 years. Responsible for Product and componenets testing, Drug Product Release testing, R and D development supportsd, Stability Function, and Environmental Monitoring

Leading Analytical Group to support method development, validation and transfer activities.

Maneging AD and QC group to support product development and commercial product release activities

Biosensor development