Unlocking genetic medicines to repair rare together. Rare REPAIRx is a multi-stakeholder initiative designed to transform the speed, economics, and scalability of developing therapies for rare genetic diseases.
Modern genetic technologies make it scientifically possible to design targeted therapies for many single-gene disorders. The prevailing development model remains poorly suited to diseases affecting small numbers of patients.
Rare REPAIRx introduces a new model that combines venture-philanthropic capital, coordinated clinical networks, modular RNA manufacturing, and federated knowledge sharing to dramatically accelerate the creation and delivery of bespoke genetic medicines.
Rather than treating each therapy as an independent program, Rare REPAIRx integrates patient identification, therapeutic design, manufacturing, regulatory development, and treatment delivery into a coordinated system designed to scale across many diseases.
Each therapy contributes data, optimization, and precedent that accelerates every program that follows. The system becomes progressively faster, more efficient, and more scalable.
Partnerships with leading genomic diagnostics providers create a direct pipeline from genetic diagnosis to therapeutic candidacy, identifying patients who can benefit from the platform.
Diagnosis → TherapyAdvanced gene-editing approaches capable of delivering permanent genetic correction, tailored to each patient's specific mutation using the programmable mRNA-CRISPR architecture.
ProgrammableScalable, modular RNA manufacturing infrastructure designed to produce individualized therapies cost-efficiently — breaking the economics that have excluded rare disease patients.
Capital EfficientStructured to rapidly achieve two early clinical successes, establishing regulatory precedents, manufacturing infrastructure, clinical expertise, and reimbursement pathways for the growing portfolio.
Precedent SettingThe initiative is anchored by partnerships with leading genomic diagnostics providers, world-class academic medical centers, and patient-driven rare disease organizations.
World-class clinical and research institutions providing scientific expertise and clinical trial infrastructure.
Leading providers creating a direct pipeline from genetic diagnosis to therapeutic intervention.
Rare disease communities and advocacy groups driving patient identification and clinical engagement.
Modular manufacturing partners enabling scalable, cost-efficient production of individualized therapies.
The Foundation for mRNA Medicines brings together leaders in RNA science, rare disease clinical development, and health policy to drive the Rare REPAIRx initiative forward.
Co-founder of both the Alliance for mRNA Medicines and the Foundation for mRNA Medicines. Deborah leads the Foundation's strategic direction and serves as Board Chair, driving policy leadership and public advocacy for mRNA science across North America, Europe, and Asia.
Senior Advisor to the Alliance for mRNA Medicines and the Foundation for mRNA Medicines. Morrie brings extensive experience in life sciences strategy, stakeholder engagement, and organizational development to the Rare REPAIRx initiative.
Chief Strategy Officer at Arcturus Therapeutics and At-Large Board Executive of the Alliance for mRNA Medicines. A recognized leader in mRNA commercialization strategy with deep expertise in scaling RNA-based therapeutic platforms from development through global commercialization.
Bloomberg Distinguished Professor of RNA Biology and Therapeutics at Johns Hopkins University and Director of the RNA Innovation Center. A pioneering researcher whose work on mRNA stability and codon biology has opened new avenues for therapeutic development, and co-founder of Tevard Biosciences.
Professor of Neurology and Neuroscience and Chair of Neuroscience at Mayo Clinic Florida. A leading neurogeneticist and PI of multiple large NIH consortia, bringing Mayo Clinic's clinical and scientific expertise in genetic disease to the Rare REPAIRx initiative.
Your support funds the platform infrastructure, clinical networks, and manufacturing capabilities needed to bring bespoke genetic medicines to rare disease patients.