Location: London, UK

Reporting to: Director, Global Technical Development

Job Summary

Orchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on transforming the life of patients with rare disorders through innovative gene therapies based on gene-modified hematopoietic stem cell (HSC). Orchard has its global headquarters in London and U.S. headquarters in Boston.

Technical Development sits within Technical Operations at Orchard and is responsible for the development and characterization of Lentiviral Vectors (LVVs) and Drug Substance/Drug Product production processes, which meet patient demand for therapeutic products of the best quality.

Reporting to the Director, Technical Development, the Principal Scientist will be primarily responsible to drive and lead project activities for development of ex-vivo gene therapy manufacturing processes along the development pathway to the point of process qualification. The creation of high-quality source documents and expert review of regulatory documents to support IND/CTA and BLA/MAA filings is a key component of this role. They would be a key part of the CMC team representing the cell process development function and working collaboratively with other functions to drive the key project goals.

Key Elements and Responsibilities

• Manage the transfer of cell processes, analytics and controls of research projects into CMC development.
• Design and implement improvements in processes and characterize gene modified HSC-based processes and products to deliver robust platform processes for early and late-stage clinical and commercial development.
• Provide expert compilation, review, and approval of documents including batch records, standard operating procedures, investigations, risk assessments, development protocols and reports.
• Provide strategic input on regulatory plans and preparation and review of sections for OTL CMC regulatory submissions (e.g. INDs, BLAs, Scientific advice) and source documents.
• Provide strategic input into development plans, program CMC strategies and departmental/functional activities, and deliver on agreed actions.
• Presentation of development strategy plans and results to the wider department and functional teams, and discuss/suggest conclusions, impact and next steps.
• Provide guidance and direction on experiments, data analysis and reports suitable for regulatory submissions.
• Co-ordinate development activities with the research, pre-clinical, QA and other departments to ensure the achievement of company timelines and milestones.
• Lead oversight of development (and manufacturing, as required) at company designated Contract Development and Manufacturing Organizations (CDMOs).
• Travel as required to provide on-site support oversight during key DS/DP manufacturing activities & provide support to CDMOs.
• Lead & participate in multi-disciplinary teams to perform risk-based analysis of processes where appropriate and providing impact assessments as required.
• Identify and implement validation requirements pre PPQ, in accordance with current industry standards, CMC regulatory standards and best practices in the evolving field of cell and gene therapy.
• Other activities as may be assigned.

Required Knowledge & Experience

As a proven technical scientific expert in the development, manufacture and delivery of cell-based therapy products with significant applied experience in the biotechnology industry, this candidate will possess:

• Substantial experience in the biotechnology or pharma industry e.g. PhD+ >7 years’ experience (depending on scope and relevance), with a primary focus on the development, manufacture, distribution and delivery of cell and gene therapy products
• In-depth technical experience with a proven track record of developing, integrating and implementing equipment and processes for clinical and/or commercial application.
• Understanding of Tech transfer and GMP manufacture, process development
• In-depth knowledge of process characterisation and comparability strategies
• Experience with risk-based approaches, such as Failure Modes and Effects Analysis
• Experience and understanding of Process Validation, ideally in cell and gene therapy
• Experience with and authoring regulatory filings and associated reports/sections writing.
• DOE and QBD experience a plus
• Experience of external CDMO management and building strong external relationships

Skills and Abilities

• Excellent interpersonal skills and demonstrated ability to work in a matrix environment
• Excellent written and oral communication skills, including presenting to senior management/executives
• Outstanding organizational skills
• Creative problem solver
• Ability to operate in a fast-paced, multi-disciplinary industrial environment
• Collaborative Communication – works effectively across diverse teams and external partners; communicates clearly and persuasively.
• Adaptability – thrives in dynamic, fast-paced environments with shifting priorities.
• Critical Judgment – makes sound decisions based on data, risk, and strategic goals.

Education

• Advanced Degree preferably a PhD or equivalent, in bioprocessing, biological sciences, gene therapy or related subject