Welcome to the future of gene therapy.
From bench to bedside faster and with greater global access
With international coalitions to support the rare disease community
GEMMABio is a new biotechnology company dedicated to developing transformative therapies for rare diseases.
We are committed to making these treatments accessible to patients around the world.
Rare Diseases: A Global Imperative
350 million individuals globally are affected by rare diseases
Around 70% of the patient population is children
More than 10,000 rare diseases have been identified
Only 5% of rare diseases have available treatments
GEMMABio is taking a novel, platform-based approach to developing gene therapies for rare diseases with innovative, next generation AAV technology.
Our portfolio encompasses several rare disease programs spanning lysosomal storage diseases, motor neuron disorders, and skeletal and cardiomyopathies.
GEMMABio was founded by gene therapy pioneer Dr. James M. Wilson in 2024.
Our scientists are leaders in the field of genetic medicines, translational research, and clinical development.
We are located in the Greater Philadelphia region.
Meet the Team.
Photo credit: Levi Gershkowitz
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