Accelerating the Path to Commercialization for Advanced Therapies
As the biotechnology landscape evolves, cell and gene therapies (CGTs) are revolutionizing patient care, particularly for rare and orphan diseases. However, these cutting-edge therapies present unique regulatory, CMC, and quality challenges compared to traditional biologics. At DSI, we provide comprehensive regulatory and development support to help clients navigate the complexities of gene editing, cell-based therapies, and regenerative medicines.
We partner with biotechnology companies, clinical laboratories, and investors to accelerate the development, approval, and commercialization of cell and gene therapy products. Our expert-driven solutions ensure compliance with FDA, EMA, and other global regulatory authorities, streamlining the IND, BLA, and post-market approval processes.
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.
Comprehensive Cell and Gene Therapy Regulatory Support
Strategic Regulatory & CMC Guidance
- Regulatory Pathway Assessment: Identifying the optimal regulatory classification and development strategy for cell and gene therapies, including differences in FDA and EMA frameworks.
- Pre-IND & Pre-BLA Meeting Support: Preparing meeting briefing packages, strategy documents, and regulatory interactions to streamline the approval pathway.
- CMC Strategy & IND Application Preparation: Ensuring comprehensive CMC data inclusion for FDA and EMA submissions.
- Regulatory Filing & Submission Support: Authoring and reviewing INDs, BLAs, CTAs, amendments, and post-approval variations.
- Fast Track, RMAT & Breakthrough Therapy Designations: Assisting clients in obtaining accelerated pathways for regulatory approval.
CMC Requirements for Cell and Gene Therapy Development
Cell and gene therapies present unique manufacturing, quality control, and supply chain challenges. DSI provides expert CMC guidance to establish a robust development and production strategy.
Characterization of Manufacturing Components & Raw Materials
- Plasmid DNA, Viral Vectors & Host Cells: Establishing testing specifications and qualification criteria.
- Reagents & Excipients: Ensuring material compliance with ICH Q7 and GMP guidelines.
- Chain of Identity (CoI) & Chain of Custody (CoC) Requirements: Developing traceability systems for autologous and allogeneic therapies.
Process Development & Manufacturing Optimization
- Vector Production & Purification: Ensuring scalability and regulatory compliance for lentiviral, AAV, and other vector-based therapies.
- Gene-Modified Cell Processing: Defining critical process parameters (CPPs) for ex vivo and in vivo gene therapy applications.
- Process Timing & Storage Considerations: Addressing the challenges of fresh vs. cryopreserved cell therapies.
- Final Formulation Development: Ensuring long-term stability and viability of CGT drug products.
Quality Control & Product Testing
Comprehensive testing ensures safety, potency, and reproducibility across batches. DSI provides expertise in:
- Identity, Purity & Potency Testing
- Microbial Contamination & Sterility Assessment
- Viability & Dose Determination
- Analytical Development & Validation for CGT Assays
- Stability Testing & Expiry Determination
Key Regulatory & CMC Challenges in Cell and Gene Therapy Development
- Regulatory Classification & Strategy: Navigating differences between U.S. and EU regulatory expectations.
- Complex Product Characterization: Defining standards for identity, purity, potency, and heterogeneity.
- Potency Assay Development: Establishing meaningful potency assays aligned with FDA & EMA guidance.
- Sterility Testing Limitations: Addressing real-time release testing for autologous therapies.
- Manufacturing Scale-Up & Comparability Studies: Ensuring reproducibility from clinical to commercial manufacturing.
- Short Shelf-Life & Supply Chain Logistics: Managing distribution strategies for personalized therapies with narrow administration windows.
GMP Compliance & Quality System Development
At DSI, we help cell and gene therapy developers implement GMP-compliant quality systems, ensuring adherence to global regulatory expectations.
GMP & Quality Assurance (QA) Support
- Quality System Implementation & Regulatory Compliance (FDA, EMA, PMDA).
- Deviation & CAPA Management for CGT Manufacturing.
- Supplier & Vendor Qualification for GMP Materials.
- Facility & Equipment Compliance Assessments.
- Traceability & Serialization Solutions for CGT Products.
Project Management & Commercialization Support
Efficient project execution is critical for successful CGT product launches. DSI provides strategic project management from preclinical through commercialization.
Comprehensive Project Management Services
- Regulatory Roadmap Development & Execution.
- CMC Milestone Tracking & Risk Management.
- Supply Chain Planning & Vendor Oversight.
- Technology Transfer & Manufacturing Scale-Up.
Why Choose DSI for Cell & Gene Therapy Consulting?
Industry-Leading Expertise & Hands-On Support
- Trusted Regulatory & CMC Experts: Our team of 60+ SMEs has successfully guided multiple cell and gene therapy INDs and BLAs through global regulatory agencies.
- End-to-End Solutions: From early-stage development to post-market compliance, we offer a full suite of regulatory, quality, and CMC services.
- Seamless Execution & Strategic Guidance: We provide both high-level strategic insights and hands-on execution to help clients accelerate approval timelines.
- Global Regulatory Experience: Expertise in FDA, EMA, MHRA, PMDA, Health Canada, and ROW regulatory frameworks.
- Commitment to Quality & Compliance: Ensuring regulatory readiness, manufacturing excellence, and quality assurance for advanced therapies.
Partner with DSI Today
Advance your cell and gene therapy program with DSI’s expert regulatory, CMC, and manufacturing consulting services. Contact us today to discuss how we can support your development and commercialization efforts.
