Advanced Therapies & Rare Diseases

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Regal Intel specializes in guiding companies through the unique regulatory pathways for advanced therapies, particularly cell and gene therapies, with a focus on rare diseases and expedited review programs like Priority Review and Breakthrough Designation. We understand the urgency of bringing these life-changing therapies to patients and offer comprehensive consulting services to streamline development and regulatory approval.

We excel in developing regulatory strategies tailored to the complexities of cell and gene therapies, with a focus on leveraging expedited programs for rare diseases. Our expertise includes:

• Breakthrough Therapy Designation: Assisting with applications to expedite development and review.
• Priority Review Designation: Guiding submissions for accelerated approval timelines.
• Orphan Drug Designation: Navigating the regulatory landscape for rare disease therapies.
• Regenerative Medicine Advanced Therapy (RMAT) Designation: Supporting applications for this FDA designation.
• FDA and EMA Interactions: Facilitating productive communication with regulatory agencies.
• Global Regulatory Strategies: Tailoring approaches for international markets.

We provide expert guidance at every stage of cell and gene therapy development, from preclinical to post-market, encompassing:

• Vector Design and Manufacturing: Ensuring compliance with stringent manufacturing standards.
• Cell Line Development and Characterization: Guiding the development of robust cell lines.
• Gene Editing and Delivery Systems: Assisting with regulatory aspects of innovative gene editing technologies.
• CMC Strategy and Development: Developing robust chemistry, manufacturing, and controls strategies.
• Preclinical and Clinical Trial Design: Tailoring trials to the unique aspects of cell and gene therapies.

We recognize the unique challenges of developing therapies for rare diseases and provide specialized support, including:

• Natural History Studies: Assisting with the design and execution of studies to understand disease progression.
• Patient Advocacy Engagement: Facilitating effective collaboration with patient advocacy groups.
• Accelerated Approval Pathways: Leveraging available pathways to expedite access for patients.
• Compassionate Use and Expanded Access Programs: Guiding the implementation of these programs.

Clinical trials for cell and gene therapies require specialized expertise in patient monitoring and long-term follow-up. Regal Intel assists with:

• Clinical Trial Design for Cell and Gene Therapies: Incorporating unique safety and efficacy endpoints.
• Long-Term Follow-Up Studies: Ensuring patient safety and efficacy monitoring.
• Adverse Event Reporting and Management: Addressing the unique safety profile of cell and gene therapies.
• Data Management and Statistical Analysis: Handling complex data from cell and gene therapy trials.

Preparing comprehensive regulatory submissions for cell and gene therapies requires meticulous attention to detail and specialized expertise. Regal Intel assists with:

• IND and BLA Submissions: Guiding manufacturers through the submission process.
• Integrated Regulatory Dossier Preparation: Ensuring all components are thoroughly documented.
• eCTD Submissions: Streamlining the submission process.
• Labeling and Packaging Compliance: Meeting specific regulatory guidelines for cell and gene therapies.

Ensuring the long-term safety and efficacy of cell and gene therapies is paramount. Regal Intel provides:

• Long-Term Follow-Up Plans: Developing and implementing comprehensive plans.
• Risk Management Plans: Developing and implementing strategies to mitigate risks.
• Pharmacovigilance System Development: Tailored to the unique needs of cell and gene therapies.