Regulatory Affairs
Gene therapy Products Regulatory Services
We provide comprehensive regulatory support for gene therapy products, guiding sponsors through every stage of development — from preclinical studies to marketing authorization and post-approval compliance.
Overview
Gene therapies represent a rapidly evolving area of healthcare with unique scientific, technical, and regulatory complexities. Successful development and approval of these advanced therapies require specialized expertise to navigate stringent global regulatory frameworks.
At Techsol, we provide comprehensive regulatory support for gene therapy products, guiding sponsors through every stage of development — from preclinical studies to marketing authorization and post-approval compliance.
Our Expertise
Key Services:
Early Regulatory Intelligence
- Evaluation of current FDA and EMA guidelines for gene therapy (e.g., CBER Guidance, EMA ATMP regulations).
- Gap analysis for preclinical and clinical development readiness.
Product Classification & Regulatory Pathway Assessment
- US: IND → BLA (CBER/OTAT, now Office of Therapeutic Products – OTP).
- EU: ATMP classification, CAT (Committee for Advanced Therapies) evaluation, MAA.
Regulatory Roadmap Development
- Defining timelines, submission strategy, and interaction plan with FDA & EMA.
- Orphan designation, RMAT (US) / PRIME (EU) pathway evaluation.
US FDA:
- Pre-IND meetings with CBER/OTP.
- RMAT designation request and guidance alignment.
EU:
- ATMP classification request to EMA.
- Scientific Advice / PRIME eligibility request.
- Early dialogue with CAT & CHMP for development alignment.
Techsol Support Includes:
- Meeting request preparation, briefing package authoring, and Q&A strategy.
- Representation during meetings and managing regulatory follow-ups.
Activities:
Study Planning & Report Authoring:
- GLP-compliant safety, biodistribution, shedding, and tumorigenicity studies.
- Immunogenicity and germline transmission studies for viral vector-based therapies.
Document Preparation:
- US: Nonclinical sections of IND & BLA Module 4 (CTD).
- EU: Nonclinical Summaries, Overviews, and Environmental Risk Assessments for MAA.
Techsol Expertise:
- Template-based writing, QC, SME review, and alignment with latest FDA/EMA expectations.
Key Deliverables
- US FDA: IND preparation, amendments, and annual reports.
- EU: CTA submission, protocol assistance, IMPD preparation, and DSURs.
Ongoing Compliance
- Safety reporting (SAE/SUSAR), patient follow-up requirements, and long-term follow-up plans for gene therapy trials.
Techsol Approach
- Submission-ready CTD authoring, regulatory QC, and RIM (Regulatory Information Management) support.
Gene Therapy-Specific CMC Support:
- Vector characterization, potency assays, and comparability studies.
- Process validation and viral clearance studies.
Submission Documentation:
- IND/BLA Module 3 (CMC) preparation for US.
- IMPD / MAA Quality Module for EU.
Regulatory Interactions:
- FDA Type C meetings or EMA Quality & ATMP CMC consultation for complex issues.
US:
BLA Submission & Review Management
- eCTD compilation, RTF readiness, and FDA query management.
- Labeling negotiations with CBER.
Post-Approval Compliance:
- Annual reports, post-marketing commitments (PMCs/PMRs), long-term follow-up (LTR), REMS (if applicable).
EU:
MAA Submission & CHMP/CAT Interactions
- Dossier preparation, eCTD submission, RFI/LoQ management.
Post-Approval
- PSUR/PBRER, risk management plan (RMP) updates, post-authorization safety studies (PASS), variations & renewals.
- Orphan Drug Designation and Maintenance.
- RMAT / Breakthrough / PRIME expedited pathways.
- Environmental Risk Assessment (ERA) for EU.
- Long-term follow-up (LTR) study planning for integrating safety over 15 years.
- Advisory on new guidance updates (e.g., FDA Human Gene Therapy CMC, Clinical & Long-term Follow-up Guidance).
Capabilities & Value Proposition
- Dedicated Gene Therapy Experts with in-depth knowledge of ATMP guidelines
- Global Regulatory Experience across US, EU, Japan, and emerging markets.
- Proven Track Record in handling complex CMC and clinical challenges.
- Cross Functional Collaboration with nonclinical, clinical, and manufacturing teams.
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