Advancing a Phase 3 Gene Therapy Program to BLA Readiness

A gene therapy company had generated strong Phase 3 clinical data and received FDA alignment to proceed with a Biologics License Application. The company needed to translate a successful clinical program into a complete, inspection-ready CMC package capable of supporting BLA filing and review.

Critical late-stage activities remained, including process performance qualification, control strategy development, analytical method validation, BLA gap closure, and generation of Module 3.

Syner-G served as an embedded extension of the company, integrating process, analytical, quality, manufacturing, and Regulatory CMC expertise to strengthen overall BLA preparedness and support generation of a cohesive Module 3 package.

PPQ PROTOCOL DESIGN & IMPLEMENTATION

• Helped design the process performance qualification protocol and align the approach with the intended commercial manufacturing process.
• Supported protocol implementation, execution planning, acceptance criteria, and cross-functional coordination.
• Connected PPQ activities with validation, manufacturing, quality, and BLA documentation needs.

CONTROL STRATEGY DEVELOPMENT

• Developed an integrated control strategy linking process parameters, material controls, in-process controls, release testing, and product quality attributes.
• Assessed late-stage control strategy gaps and clarified the supporting evidence required for the BLA.
• Aligned control strategy content across validation, analytical, manufacturing, and regulatory documentation.

ANALYTICAL METHOD VALIDATION

• Guided analytical method validation planning for assays supporting release, stability, characterization, and process validation.
• Reviewed validation readiness, protocols, acceptance criteria, data packages, and documentation needed for BLA inclusion.

BLA READINESS & GAP ASSESSMENT

• Conducted a comprehensive BLA CMC gap assessment across process, analytical, validation, manufacturing, quality, and regulatory documentation.
• Prioritized gaps, established owners and closure plans, and integrated remaining activities into the BLA readiness plan.
• Assessed consistency and traceability across source records, validation deliverables, and the regulatory narrative.

MODULE 3 GENERATION

• Supported generation and integration of BLA Module 3 content across process, analytical, manufacturing, control strategy, validation, and quality sections.
• Established a structured review framework to drive completeness, consistency, traceability, and submission readiness.

Syner-G provided the integrated late-stage CMC and Regulatory CMC support needed to convert positive Phase 3 results and FDA filing alignment into an actionable, coordinated BLA readiness program.

• 01 – PPQ protocol design and implementation supported
• 02 – Commercial control strategy developed
• 03 – Advanced analytical method validation
• 04 – Priority BLA gaps identified and addressed
• 05 – Supported integrated Module 3 generation