Cell & Gene Therapy

After decades of research, cell and gene therapies offer new hope to patients with diseases and conditions previously considered untreatable. While there have been tremendous advances, companies must navigate a rapidly evolving regulatory environment. Greenleaf assists sponsors by optimizing FDA interactions, submissions, manufacturing technology, and quality systems to support regulatory compliance and review.

Our Cell & Gene Therapy Services

Product Development & Review

Greenleaf’s Drug and Biological Products Team helps sponsors understand and respond to the FDA requirements applicable to cell and gene therapy products and optimize FDA interactions and submissions to support development and regulatory review.

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

As development of cell and gene therapies often involves consideration of aspects of medical device and combination product regulation — another area of deep FDA experience and expertise at Greenleaf — experts from the Medical Device and Combination Products Team and the Drug and Biological Products Team collaborate regularly on such matters.

Learn more about the capabilities and services offered by Greenleaf’s Drug & Biological Products Team

Learn more about the capabilities and services offered by Greenleaf’s Medical Device & Combination Products Team

Quality, Manufacturing & Compliance

For companies striving to introduce cell and gene therapy products to the market, establishing manufacturing and quality controls is one of the primary challenges. Manufacturers of cell and gene therapies are responsible for complying with multiple current GXP regulations, including: good manufacturing practice regulations (GMPs) and/or quality system regulations (QSRs); good laboratory practice regulations (GLPs); good clinical practice regulations (GCPs); and good tissue practice regulations (GTPs).

Greenleaf’s Product Quality, Manufacturing, and Compliance Team, along with its network of independent compliance experts, provide clients with the strategic and technical support needed to meet FDA requirements, including pre- and postapproval inspection readiness and compliance assessments. The team offers credible, informed guidance on FDA communications and meeting preparation, provides regulatory benchmarking services, and assists clients with evaluating and responding to FDA Form 483s, warning letters, untitled letters, and other FDA regulatory correspondence.

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

Additionally, Greenleaf works with the FDA to support client development and implementation of cell and gene therapy manufacturing technology. Greenleaf has effectively engaged with the FDA on behalf of clients to discuss, identify, and resolve potential technical and regulatory challenges related to emerging manufacturing technologies prior to regulatory submissions.

Learn more about the capabilities and services offered by Greenleaf’s Product Quality, Manufacturing, and Compliance Team

Regulatory Landscape Analysis & Diligence

Greenleaf monitors and analyzes the regulatory environment for emerging trends in cell and gene therapy regulation that could potentially impact current development programs ⎯ including orphan drug designation and exclusivity, long-term follow-up requirements, companion diagnostics, and the development and use of real-world evidence ⎯ as well as changes to the competitive landscape for cell and gene therapies.

Our Advisory Services Team also offers focused due diligence and regulatory risk assessment in this space.

Learn more about the capabilities and services offered by Greenleaf’s Advisory Services Team

Success Stories

  • Evaluation & Development of a Cell and Gene Therapy Quality Management System

    A biopharmaceutical company well-versed in small molecule manufacturing processes requested Greenleaf’s support in building out its cell and gene therapy (CGT) manufacturing facility processes. Specifically, Greenleaf was asked to assist in developing the company’s Quality Management System (QMS) for new modalities in order to understand its positioning for sustainability and expansion of operations.  Greenleaf experts …

Cell & Gene Therapy Resources

View or download the PDFs below to learn more about Greenleaf and our cell and gene therapy capabilities.

Connect With an Expert

Greenleaf’s Cell & Gene Therapy Team is ready to help you achieve your regulatory goals. Please provide the information below and a team member will be in contact.

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