CMC Leader, AAV Gene Therapy

Job Title: CMC Lead, AAV Gene Therapy

Location: UK or US East Coast, hybrid or remote, with regular travel to manufacturing and partner sites in Europe and the US

Job Type: Full-time, permanent

Reports to: Development leadership

Cpl Life Sciences is partnering with an innovative stealth-mode biotech to appoint a CMC Lead. The company is developing an AAV gene therapy for a neurological indication, now in development. The business uses an established, experienced manufacturing partner for process execution, analytical testing and release. It also applies AI and data science methods to manufacturing and analytical data, with the aim of building a stronger, better-evidenced control strategy and regulatory narrative.

The role

You will take sole sponsor-side responsibility for chemistry, manufacturing and controls on the programme. You own the CMC strategy, the control strategy and the regulatory CMC narrative, and you provide sponsor oversight of manufacturing quality, timeline and cost. Because the company does not manufacture or test product itself, you will review, approve and oversee all partner work on its behalf. You will also contribute manufacturing and CMC assessment to due diligence on prospective partnerships, licences and acquisitions.

Responsibilities

CMC strategy and governance

• Set and maintain the CMC development plan through process validation, PPQ and marketing application, aligned to the clinical and regulatory timeline
• Maintain the CMC risk register and readiness assessments, and report status, risk and mitigation to leadership and the development board
• Bring open CMC decisions to a documented conclusion, including batch strategy, comparability approach, specification setting and scale decisions
• Maintain the CMC budget, forecast and purchase order position with finance, and reconcile partner invoicing to the development plan

Manufacturing partner oversight

• Track partner deliverables under the agreements and development plan, and raise timeline or scope risks early through joint governance forums
• Provide sponsor review and approval of process, analytical and stability protocols and reports, batch records, deviations and investigations
• Manage clinical supply planning, batch scheduling, release timing and shelf-life position to protect the trial schedule
• Oversee process characterisation, process validation and PPQ planning and execution, and pre-approval inspection readiness

Analytical and quality

• Maintain the sponsor position on specifications, critical quality attributes, potency, empty-to-full capsid ratio, aggregation and stability
• Direct comparability strategy and assessment across process and scale changes, and defend the approach with regulators
• Work with quality colleagues on the quality agreement, person responsible for release, audit findings and change control affecting the product

Regulatory

• Author and review CMC sections (Module 3) of regulatory submissions, briefing documents and responses to agency questions across FDA, EMA and MHRA
• Represent the sponsor on CMC matters in agency meetings and inspections, with regulatory and quality colleagues

Partnership due diligence

• Lead the manufacturing and CMC workstream of due diligence on prospective partnerships, in-licensing and acquisition opportunities, covering process and analytical maturity, manufacturing route, capacity, supply chain, cost of goods, comparability history and regulatory CMC status
• Deliver clear written findings, risk ratings and cost and timeline implications to the deal team and leadership, and support negotiation of supply, technology transfer and quality terms

Data and AI

• Work with data science colleagues applying AI methods to process and analytical data, and translate that work into a defensible control strategy and regulatory narrative

Essential skills and experience

• Command of AAV or viral vector drug substance and drug product CMC, including upstream and downstream process, formulation and fill, and the analytical package for potency, capsid content, aggregation, comparability and stability
• At least one AAV or viral vector programme taken toward or through Phase 3, with working familiarity with process validation, PPQ and inspection readiness
• Track record of managing manufacturing through a CDMO or partner organisation, including deliverable tracking, technical review and joint governance
• Experience authoring Module 3 content and responding to CMC questions from FDA, EMA or MHRA on advanced therapy products
• Experience building and maintaining CMC budgets and cost-of-goods estimates, and reviewing partner pricing and invoices
• Experience delivering CMC in a small sponsor organisation with broad scope and lean support functions
• Ability to hold sole sponsor-side accountability for CMC delivered entirely by third parties, and to make and defend decisions on incomplete information
• Ability to present CMC risk, cost and timeline clearly to leadership, boards and investors
• Degree in the life sciences, chemistry, engineering or a related field, or equivalent demonstrated experience

Desirable

• Experience with neurological or intraparenchymal delivery programmes
• Experience with drug-device combination products and the CMC interface with device development
• Participation in a BLA or MAA submission or pre-approval inspection for an advanced therapy product
• Experience assessing manufacturing and CMC in due diligence for partnerships, licensing or M&A, including CDMO or site evaluation
• Exposure to statistical, modelling or AI methods applied to process development, control strategy or analytical data

This is an amazing opportunity for a CMC expert to join one of the most innovative biotech companies. Join a team of like-minded experts at the heart of AI and Science innovation.

For more information, please reach out to me at lucy.kirkaldy@cpl.com