February 2024 EAB Meeting
Meeting Notes
Attendees: Kapil Bharti,Mary Haak Frendscho, Chad Jackson, David Schaffer, Kris Saha, David Gamm, Sarah
Gong, Ross Schwartz, Adam Silverman, Bikash Pattnaik, Bill Murphy, Michael Nork
[Absent: Jason Comander]
Background: The EABgathered virtually to chart the course for our gene therapy program, focusing
on a strategic pathway to IND filing by 2028. This initiative prioritizes
addressing genetic eye disorders, leveraging our platform technology for
broader mutation applicability.
Discussion Topics
1. Clinical Precision:
· Emphasis onrefining inclusion/exclusion criteria ensures patient selection aligns with our
therapy's unique capabilities.
· Thedevelopment plan underscores the necessity of mutation-specific approaches, a
critical factor for regulatory success and therapeutic efficacy.
2. Regulatory Pathway:
· Detailedstrategies for navigating the regulatory landscape, targeting statistical
significance in ultra-rare diseases.
· A definitivegoal for IND filing by 2028 sets a clear timeline, with preparatory steps
including critical FDA interactions and consortium-based data sharing outlined.
3. Innovative Partnerships:
· The NIH SCGE CommonFund project exemplifies our commitment to innovation, aiming to revolutionize
treatment for eye conditions.
· Strategicdiscussions underscored the potential of our platform technology, highlighting
the importance of data sharing and collaboration for regulatory and clinical
advancements.
4. Manufacturing Strategy:
· Aforward-looking approach to manufacturing, including carrier-free therapy
options and local GMP facility collaborations, signals our preparation for
scalable production.
· Earlyengagement with regulatory experts and manufacturing partners is crucial for
navigating the complex landscape of biologic vs. device classification.
5. Commercialization Pathways:
· Anticipationof future commercialization strategies, whether through licensing or new
venture creation, indicates proactive planning for market entry.
· The necessityof early dialogue with potential partners and investors was emphasized,
ensuring a robust foundation for the therapy's commercial success.
Immediate Focus Areas
· RefineClinical Criteria: Sharpeningpatient selection criteria to ensure clinical trials are as effective and
targeted as possible.
· AccelerateRegulatory Engagement:Preparing for IND filing, emphasizing the importance of strategic FDA
interactions and consortium collaboration for shared learning.
· AdvanceManufacturing and Technology Partnerships: Initiating dialogues with potential GMP and fill-finishpartners, focusing on innovative manufacturing solutions to meet therapy
delivery challenges.
· Explore Commercialization Strategies: Engaging potential partners early to navigate the commercializationlandscape effectively, ensuring our therapy reaches patients in need with a
viable economic model.
Conclusions: The meetingcrystallized our strategic directives: precision in clinical development,
clarity in regulatory navigation, innovation in manufacturing, and foresight in
commercialization. This comprehensive approach ensures our gene therapy program
is on track to deliver transformative treatments for genetic disorders, with an
initial focus on eye conditions. Leadership's commitment to these strategies
will propel the program towards its IND filing goal and beyond, setting a new
standard in patient care.
Key Questions Provided For Discussion
Overall Strategy
- If you had a do over the BRILLANCE (EDIT-101) trial today, would you design it differently? For example, would you structure the partnerships
and studies differently? - What additional activities or resources should we consider now, so that we hit our milestone of an INTERACT meeting this year for the Lead
Trailblazer Project on LCA? - What competitive programs in the commercial or academic sectors do you see?
- What parts of our development should be outside academia? at a CRO? Or at a CMO?
- How should we consider a freedom-to-operate analysis? When should the analysis be done? by whom?
- What external partners do you think we should engage and when?
CMC
- What are the general pitfalls in CMC? For example, what contaminants will the FDA focus on?
- What are the potential benefits and risks in locking in a partnership with a specific reagent or service provider (e.g., Synthego, Danaher)? Do
you lose nimbleness? What provisions would there be with alternative
suppliers?
Clinical
- What challenges do you foresee in patient recruitment? We have identified 22 patients identified so far with mutations in KCNJ13 and hundreds of patients with mutations in BEST1.