Next Generation Biotherapeutics

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 known as cell 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.


Cell 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 briefing packages, investigational, and marketing applications).

Key challenges encompass


Mainly based on starting materials of human origin and often produced with sophisticated manufacturing processes, these advanced therapy medicinal products involve complex procurement, testing, processing, preservation, storage, and distribution. They require sequential testing and release from raw and starting materials to the drug product, as well as adapted control strategy.

Cell and Gene Therapy CMC requirements

Gene therapy and cell therapy companies has been increasing over recent years and in the coming year gene therapy and cell therapy products will begin to have more of a presence within the healthcare market. These treatments are vastly different than traditional pharmaceuticals but also require CMC activities to ensure the manufacturing process and treatments meet the regulations for producing safe products. To appropriately manufacture a specific manufacturing processes, product characteristics, and product testing must be defined in order to ensure that the product is safe, effective and consistent between batches. These activities are known as CMC, chemistry, manufacturing and control.


Characterization of the Manufacturing Materials

The IND should include specifications and testing off all materials and components using in the manufacturing process of gene and cell therapies.

  • Vectors
  • Cells
  • Reagents
  • Excipients

Product Manufacturing Procedures

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

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

Product Testing

The gene or cell therapy product must be tested during production and in its final stage. These tests consist of microbiological testing, identity, purity, potency, viability, and cell numbers (dose).

  • 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 New Active Substance 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 within gene therapy and cell therapy companies. DSI can provide interpretation of the FDA document and will include three main areas that are to be included in the IND for gene therapy and cell therapy 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.