Your CRISPR Guide

CRISPR Therapeutic Regulatory Guidance and Expertise

Navigate the Therapeutic Landscape with Synthego's Regulatory Experts

CRISPR cell and gene therapies hold immense promise but navigating their unique regulatory challenges can be complex. Synthego simplifies this process, offering expert guidance in every phase - from CMC Module 3 preparation to direct interactions with regulators like the FDA and EMA. Our team stays ahead of evolving CRISPR regulations, ensuring your GMP CRISPR molecules and regulatory submissions meet the highest standards. By partnering with Synthego, you can smoothly transition your therapy from the lab to the clinic with the support and expertise needed to advance groundbreaking science.

Synthego's Regulatory Guidance Offerings

Prepare your regulatory strategy for IND submission

Starting preclinical experiments for your CRISPR therapy? Synthego provides the insights, feedback, and products you need to build a strong preclinical strategy. With expert guidance and advanced solutions like INDe gRNAs and GMP-grade nucleases we’ll support you in meeting regulatory requirements and setting your IND submission up for success. Early engagement with regulatory agencies, like INTERACT and pre-IND meetings, is critical, and we’ll assist in preparing comprehensive meeting packages covering your drug’s preclinical data, clinical plans, and CMC details.

File your IND/IMPD application with confidence

Your IND/IMPD application is the key to advancing your CRISPR therapy into clinical trials, and Synthego ensures you’re ready. With our high-quality CRISPR solutions, including INDe and GMP-grade options, you can minimize variability, eliminating unnecessary delays. We have supported 12 successful IND approvals, by providing comprehensive and expert regulatory strategies to smooth the transition to clinical development. Importantly, our team provides submission-ready documentation, including gRNA CMC sections and Drug Master File references, where applicable.

Achieve your clinical milestones

After IND approval, navigating regulatory requirements becomes increasingly complex. Our cGMP CRISPR solutions, scalable manufacturing, and robust quality control strategies ensure consistency and compliance throughout each trial phase. Synthego’s regulatory experts provide continuous support, helping you interpret feedback and formulate strategies to meet evolving requirements. With flexible solutions and reliable expertise, we’ll help save you time and minimize your risks as you progress through the clinical phases.

Why Trust Synthego for Regulatory Guidance?

Expedite Approval with Synthego's DMF

Synthego supports your regulatory documentation with a Drug Master File (DMF) for GMP gRNAs, designed to simplify and strengthen your submission process. A DMF is a confidential document submitted to regulatory authorities, such as the FDA, containing detailed information about the manufacturing, testing, and quality control processes of a drug product. It serves as a critical resource, enabling regulatory reviewers to assess the quality and safety of the materials used in your clinical product without requiring you to disclose proprietary information to third parties.

For CRISPR-based therapies, having a comprehensive DMF is essential. It ensures that the GMP reagents supporting your therapy are manufactured to the highest standards of quality and compliance, a key requirement for advancing through clinical development. Synthego’s expertly prepared DMF for GMP gRNAs streamlines the preparation of your regulatory documentation by providing pre-reviewed, submission-ready data that integrates seamlessly into your filings. This not only enhances the overall completeness and reliability of your submission but also reduces the back-and-forth with regulatory agencies, minimizing delays and expediting the review process.

Leverage Proactive Regulatory Intelligence

Synthego gathers regulatory intelligence with you in mind, to make the complex CRISPR regulatory landscape easier to understand and navigate. By keeping a close eye on ongoing clinical trials, updates to FDA guidelines, Advisory Committee discussions, and feedback from regulators, we provide actionable insights that help you stay prepared and ahead of the curve.

We focus on the critical touchpoints that matter most to your progress, like early development meetings (INTERACT and pre-IND) where you can address regulator feedback early in the process. Our team also pulls in valuable knowledge from informal discussions, like Town Halls, and observes milestones from other developers to give you a complete view of the regulatory landscape. This means fewer surprises and more informed decisions as you move forward.

Our deep expertise and proactive intelligence directly translate to accelerated timelines and reduced uncertainty, giving you the confidence to make strategic decisions effectively. Synthego’s regulatory expertise not only enhances compliance readiness but also empowers teams to stay competitive, bringing transformative solutions to patients faster.

CRISPR in the Clinic: Synthego’s Regulatory Experts Discuss Development of Cell and Gene Therapies

The CRISPR gene therapy regulatory space is changing rapidly. In this blog, we’re shining a light on regulatory scientists, the unsung heroes of clinical development.

Explore the Foundations of CRISPR Regulations

What are the current FDA CRISPR regulations?

If you're developing CRISPR therapies for approval in the U.S., you’ll be working with the FDA’s Center for Biologics Evaluation and Research (CBER). Currently, there aren’t any guidelines specific for CRISPR-based therapies, so you’ll need to rely on the FDA’s 34 guidances for cell and gene therapies - which can feel pretty broad for CRISPR’s unique needs. This lack of clarity can be frustrating, but with Synthego’s guidance and strategies, you can confidently steer your therapy through the regulatory process. By focusing on phase-appropriate Good Manufacturing Practice (GMP) standards, you’ll build a strong regulatory foundation to help move your therapy forward.

Learn more about the current FDA guidelines for cell and gene therapies.

What are the Challenges Unique to CRISPR-Based Therapies?

CRISPR-based therapies bring incredible potential to cell and gene treatments but face unique regulatory challenges. Unlike traditional gene therapies, CRISPR’s ability to create double-stranded DNA breaks raises concerns about off-target effects, genotoxicity, and immunogenicity. Regulators expect developers to present robust evidence proving these risks are minimal and managed effectively.

Learn more about off-target editing requirements for CRISPR-based therapies.

For CRISPR-edited allogeneic cell therapies, risks like graft-versus-host disease (GvHD) and immune rejection add further complexity. Additionally, patients in clinical trials require thorough monitoring to ensure safety and demonstrate a clear benefit over existing treatments, especially for rare diseases with limited options.

What is in the Future for CRISPR Regulations?

FDA regulations are continually updated to keep up with developments in cell and gene therapies, and the Agency works collaboratively with industry stakeholders to develop the most appropriate guidelines for genomic medicines. As real-world data concerning CRISPR-based therapies becomes available, regulatory policies are expected to evolve to reflect these insights.

Check out the list of current CRISPR-based therapeutics in clinical trials.

We anticipate that the FDA will release guidances that are specific to CRISPR therapies, particularly since there has now been an FDA-approved CRISPR therapy. Synthego’s regulatory team conducts proactive surveillance to stay up-to-date with new CRISPR regulations and provide feedback to regulatory bodies during public forums concerning these guidances.

Multinational CRISPR regulations and studies

Regulations for CRISPR-related clinical studies vary widely across the globe, with each country enforcing its own standards and ethical considerations. Regulatory bodies such as the European Medicines Agency (EMA) emphasize patient safety but operate under different frameworks and procedures compared to the FDA. Meanwhile, many countries are just beginning to establish oversight for CRISPR therapies, creating unique challenges for harmonizing global trial protocols.

For organizations planning multinational clinical studies, navigating these regulatory disparities can be daunting. This is where Synthego can make a difference. Our expertise not only streamlines U.S. submissions but also provides tailored strategies for aligning CRISPR therapeutic development with the standards of regulatory bodies worldwide. By leveraging our knowledge, you can confidently manage complex international requirements, accelerating your path to approval while maintaining compliance across diverse markets.

What are the CTD and CMC?

The Common Technical Document (CTD) is your roadmap to efficient regulatory submissions for new therapies across global markets. It organizes all essential information into a standardized format, making it easier to meet the requirements of agencies like the FDA and EMA. For CRISPR developers, the electronic CTD (eCTD) simplifies and streamlines filing processes, whether for an Investigational New Drug (IND) application in the US or a Clinical Trial Application (CTA) in the EU.

Central to the eCTD is Module 3 (M3), which focuses on Chemistry, Manufacturing, and Control (CMC). This section demonstrates the consistency and quality of your CRISPR therapy’s production. From raw material controls and manufacturing processes to product specifications, safety standards, and storage conditions, the CMC M3 ensures regulators see a complete picture of how your therapy is produced and maintained. Our DMF for GMP gRNAs provides critical data on the manufacturing and quality of this key CRISPR component, significantly simplifying the preparation of your CMC M3. By integrating our DMF content, you’ll spend less time gathering and organizing information while ensuring that your documentation meets regulatory standards.

Download our flyer with more detailed information on the eCTD and CMC.

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