CELL AND GENE THERAPY

The Key to Commercializing Revolutionary Gene Therapies

In today’s evolving, value-based environment for specialty drugs, high-touch services that enhance patient outcomes are playing an increasingly important role in market access and patient care delivery. Nowhere is this more apparent than in the world of orphan drugs and, in , the emerging technology in cellular and gene therapy. Overcome complicated and unique challenges in the development and commercialization of genetic therapy. We provide regulatory pathway assistance to biotechnology companies, clinical laboratories, and biotech investors.


Cellular and Gene Therapy Regulatory Consulting

Consultation for the development of regulatory strategies to facilitate rapid development of cell, tissue and gene-based therapies. Authoring of regulatory submissions (pre-IND and pre-BLA meeting briefing packages, investigational, and marketing applications).

Key challenges encompass


Cell and Gene Therapies present unique manufacturing and quality control challenges compared with traditional biologics such as monoclonal antibodies. Autologous therapies have the additional challenge of maintaining a Chain-of-Custody (CoC) and Chain-of-Identity as material is transported from the patient to the manufacturing site and then back to the patient. These advanced therapy medicinal products also require complex procurement, testing, processing, preservation, storage, and distribution networks.

Cellular and Gene Therapy CMC requirements

Commercialized Cell and Gene therapy products have been increasing over the last decade and will continue to have more of a presence within the healthcare market. The scale and equipment can be significantly different than that used for traditional pharmaceuticals and provide additional challenges for producing a safe and efficacious product. Product characteristics and product testing must be defined in order to ensure that the product is safe, effective and consistent between batches.


Characterization of the Manufacturing Materials

Testing requirements and specifications need to be established for the unique raw materials used in Cell and Gene therapy manufacturing processes. .

  • Plasmids
  • Viral Vectors
  • Host Cells
  • Reagents
  • Excipients

Product Manufacturing Procedures

The IND should include all procedures used during the collection, production and purification of cell and gene therapy products.

  • Vector Production/Purification
  • Preparation of Ex Vivo Gene-Modified Cells
  • Processing Timing and Intermediate Storage
  • Final Formulation

Product Testing

In-process and final testing needs to be identified and specifications established.

  • Microbial Testing
  • Identity
  • Purity
  • Potency
  • Viability
  • Cell number/Dose

CMC regulatory requirements for IND Applications

With enthusiasm continuing to grow around these novel therapies, it is imperative to understand and assess the Chemistry, Manufacturing and Controls (CMC) factors required for manufacturing and development of commercially viable products. In 2018, the FDA published a guidance for FDA reviewers and sponsors titled, “Content and Review of Chemistry, Manufacturing and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)” which lays out FDA’s expectations for CMC operations for cell and gene therapy companies. DSI can provide interpretation of the FDA document and the three main areas that need to be included in the IND for these products: Characterization of the manufacturing components and materials, product manufacturing procedures, and product testing.


Key CMC Regulatory challenges

  • Regulatory classification and strategy, differences in EU and US regulatory classifications
  • IND Applications, Amendments
  • Product characterization (identity, viability, purity, potency, viral safety)
  • Release testing (due to short shelf life and limited sample availability)
  • Sterility assessment (with results often not available prior to infusion to patient)
  • Complex mode of actions and challenges to develop relevant potency assays
  • Definition of product heterogeneity versus impurity
  • Process and analytical development / validation, comparability and process reproducibility, cGMP alignment

GMP Regulatory Assessment, QA System Design

  • QA Management
  • Facilities
  • Organization and Personnel
  • QC Testing
  • Packaging and Labeling
  • Identification and Traceability
  • Deviation Management
  • FDA Registration and Reporting

Project Management

  • Define scope and implement project plans in accordance with the client's needs.
  • Provide hands-on project management and actively manage plan and deliverables.
  • Prepare communication plan and related project status reports to key stakeholders.
  • Define and manage overall change control processes and quality assurance aspects of the project.