We sat down with Dr. Lilia Bi, RareMoon's newest CMC consultant for cell and gene therapies, to discuss her extensive regulatory experience and insights for gene therapy developers navigating the complex regulatory landscape.
About Our Expert
Lilia Bi, MD, PhD brings over 20 years of specialized experience in gene therapy product review and regulatory policy. She previously served as a master CMC reviewer at FDA/CBER, leading reviews of gene therapy submissions. She holds a PhD from Johns Hopkins University and an MD from Peking Union Medical College.
Q: What are you most excited about in your new role at RareMoon?
A: "I feel incredibly honored and rewarded with my current role as a CMC consultant for cell and gene therapies at RareMoon. What excites me most is the opportunity to guide innovative therapeutic development from the ground up."
She highlights the importance of considering factors like product design, manufacturing processes, and regulatory compliance throughout different stages of product development. "Every stage of development presents unique challenges and opportunities, and having the right regulatory strategy can make all the difference in bringing life-changing therapies to patients."
Q: How could gene therapy manufacturers and sponsors best utilize your regulatory and scientific expertise?
A: Lilia brings her 20 years of experience in evaluating and reviewing CMC submissions to help sponsors navigate the complex regulatory landscape. Her expertise spans multiple critical areas:
- Advising on regulatory meetings and strategy development
- IND and BLA submission preparation and review
- Having participated in AAV BLA reviews at the FDA
- CMC development planning and risk assessment
"My experience on the reviewer side gives me unique insights into what regulators are looking for and how to present data in the most compelling and compliant way possible."
Q: What advice would you give to a sponsor manufacturing a gene therapy at an early stage (Pre-IND)?
A: "My strongest recommendation is to contact the FDA early via pre-IND meetings to obtain critical comments and guidance. This early engagement can save months or even years of development time."
She advises sponsors to approach these meetings strategically: "Ask clear, focused questions about CMC aspects and prioritize the most critical development questions. The FDA is generally very responsive to well-structured inquiries that demonstrate a sponsor's commitment to quality and safety."
💡 Expert Insights
Planning Ahead in CMC Development: Early strategic planning in CMC development can prevent costly delays and regulatory setbacks later in the development process.
Critical Development Stages: Each stage of gene therapy development presents unique regulatory challenges that require specialized expertise and forward-thinking strategies.
Regulatory Engagement: Proactive engagement with regulatory authorities through pre-IND meetings and scientific advice can significantly improve development outcomes.
The Importance of Early CMC Strategy
Dr. Bi emphasizes that successful gene therapy development begins with comprehensive CMC planning. "Many sponsors underestimate the complexity of CMC requirements for gene therapies," she explains. "These products have unique manufacturing challenges, quality control requirements, and regulatory expectations that differ significantly from traditional pharmaceuticals."
The key areas where early CMC strategy can make a significant impact include:
- Manufacturing Process Development: Establishing scalable, reproducible manufacturing processes early in development
- Quality Control Strategy: Implementing robust analytical methods and quality standards
- Regulatory Pathway Planning: Understanding and preparing for regulatory requirements across different jurisdictions
- Risk Assessment: Identifying and mitigating potential CMC-related risks before they impact development timelines
Leveraging FDA Experience for Client Success
Having served as a master CMC reviewer at FDA/CBER, Dr. Bi brings an invaluable perspective to RareMoon's clients. "Understanding the reviewer's mindset and priorities helps me guide sponsors in preparing submissions that address key regulatory concerns proactively."
This experience translates into practical benefits for clients, including:
- More efficient regulatory meeting preparation
- Strategic submission planning that anticipates reviewer questions
- Risk mitigation strategies based on regulatory precedent
- Clear communication of complex CMC concepts to regulatory authorities
"Working as a clinical consultant at RMC places me at the forefront of new medical therapies, giving me the opportunity to see innovative treatments and drugs come to life. There's nothing more rewarding than knowing that the regulatory guidance we provide today could help bring life-changing therapies to patients tomorrow."
Looking Forward: The Future of Gene Therapy Regulation
As gene therapy continues to evolve, Dr. Bi sees several trends that will shape the regulatory landscape:
- Increased Regulatory Flexibility: Agencies are becoming more adaptive to innovative manufacturing and analytical approaches
- Enhanced Guidance Documents: More detailed guidance on CMC requirements for different gene therapy modalities
- International Harmonization: Greater alignment between regulatory agencies on gene therapy standards
- Real-World Evidence Integration: Growing acceptance of post-market data to support regulatory decisions
"The regulatory environment for gene therapies is more supportive than ever," Dr. Bi concludes. "But success still requires careful planning, strategic thinking, and expert guidance to navigate the complexities effectively."