Cell & Gene Therapy

Members of Greenleaf’s Drug and Biological Products Team specialize in strategic communications with the FDA at every stage of product development ⎯ early INTERACT meetings, pre-IND and other formal meetings throughout development, correspondence, clinical trial design, submissions, FDA review communications and labeling discussions, and postmarket requirements.

Greenleaf also assists clients in interpreting FDA guidance and properly applying it to ensure effective, efficient product development.

When issues arise related to comparability of early-phase and later-phase data, our experts help sponsors address the FDA’s questions and make the strongest arguments in support of their submission.

For sponsors developing cell and gene therapies to treat rare and ultra-rare diseases, Greenleaf offers guidance on maximizing trial design using appropriate clinical endpoints and natural history study data in support of efficient product development. Our experts also assist sponsors preparing requests for orphan drug and rare pediatric disease designations.

Greenleaf advises sponsors on the FDA’s expedited programs for development and review, including:

• Regenerative medicine advanced therapy (RMAT) designation
• Breakthrough therapy designation
• Fast track
• Priority review
• Accelerated approval and confirmatory studies

Greenleaf and our network of experts conduct compliance assessments and gap analyses at both the facility and corporate levels for manufacturing and quality practices and systems, including GMP, GCP, GTP, and QSR assessment, as well as:

• Identification of critical quality attributes (CQA)
• Chemistry manufacturing and controls (CMC) readiness
• Manufacturing platform (e.g., manufacturing steps, implementing new technologies, and automated systems)
• Quality- and compliance-related processes and procedures
• Corporate training programs and corporate-wide quality systems
• Audit programs
• Risk management
• Quality/compliance/clinical organizations

In these same areas, Greenleaf evaluates compliance assessments and delivers strategic and technical guidance and recommendations that will help the client implement system-wide quality enhancements.

Greenleaf and our consultant network offer the following mock FDA inspection services to help clients prepare for routine GXP inspections and to align the client’s organization with regulators’ expectations:

• Preparing and conducting mock inspections
• Conducting gap analyses
• Facilitating communications with CBER’s Advanced Technologies Team
• Identifying and prioritizing deficiencies
• Providing corrective action support
• Providing guidance during inspections
• Assisting with revision and preparation of all relevant documents
• Training and preparation in advance of an FDA inspection, covering such topics as: FDA inspection authority; inspection expectations; inspection terminology and guidelines; and inspection process, roles, and responsibilities

Greenleaf experts help clients prepare for and respond to identified GXP compliance problems with knowledgeable, objective analysis and evaluation of:

• FDA Form 483s and company responses
• Warning letters and company responses
• Untitled letters and company responses
• Internal and external audit reports
• Establishment inspection reports
• FDA regulatory correspondence and company responses
• FDA communications relating to inspectional observations
• Investigation and root cause analysis of deficiencies
• Corrective and preventive action plans
• Preparation for FDA meetings
• Comprehensive corporate-wide and facility implementation plans