We match the urgency of our mission with

the diligence of our work ethic.

Our work proceeds along four pathways.

Drug Discovery
& Therapeutic Development

1

Last summer, we launched our flagship therapeutic development project, Pathways to Hope (P2H) for MEF2C Haploinsufficiency, in partnership with our colleagues at the MEF2C Foundation. Under the leadership of leading MEF2C expert Dr. Christopher Cowan at the Medical University of South Carolina, P2H has shown immense promise in developing therapeutic candidates and testing their efficacy in mouse models.

Our MEF2C Brain Trust is a key vehicle for brainstorming promising pathways for therapeutic discovery and moving ideas from concept to practice. Rare Bird seeks to support these projects and, in doing so, cultivate a cohort of next-generation MEF2C experts.

We welcome scientists who are interested in carrying out MEF2C research or joining our Brain Trust to reach out to our foundation (please email rarebirdorg@gmail.com with a bio, timeline, and short description of your proposed project). Please note Rare Bird does not reimburse for indirect costs.

We work with experts in the field to make sure we are satisfying relevant tolerability and toxicology requirements. The safety of the therapeutics we seek to develop is paramount to our mission.

Tolerability & Toxicology Studies

2

Patient Pre-Clinical Readiness/
Natural History Study

3

With our global partners, we are preparing our MCHS community for clinical trial, including by collecting high-quality natural history information from our worldwide population. Our approach to clinical readiness is rigorous and calibrated to the requirements set by the FDA and the EMA. While we are anchored in the United States, our readiness work involves multiple international sites with the goal of ensuring access, involvement, and regulatory compliance.

Regulatory Affairs
(FDA and EMA)

4

We are regularly engaged with regulatory entities and expert consultants to ensure that we are equipped to satisfy regulatory requirements and maximize the seamlessness of our regulatory approval processes. Our approach to regulatory approval—like everything we do—is grounded in the United States but globally inspired to maximize the reach and access of our clinical trial process and—eventually—our therapeutic itself.

Patient Listening Session
On Thursday, September 19, 2024, the U.S. Food and Drug Administration (FDA) Patient Affairs office hosted a Patient Listening Session (PLS) with representatives of the MEF2C Haploinsufficiency Syndrome (MCHS) community. Seven parents of children and adults with MCHS shared their first-hand experiences with this disease and its impact on everyday life. Over 20 FDA offices attended to learn about MCHS and ask questions.