Gene & Cell Therapy
Transforming Lives Through Gene Therapy
We develop next-generation gene and cell therapies for patients with rare and serious diseases — combining rigorous science with a deeply human mission.
Our Mission
"Science is our tool. Changing lives is our purpose."
At Transforma Bio, we believe that the most complex science should serve the most human of goals. We are building a platform to deliver transformative therapies to patients who have waited long enough — and to the families who stand beside them.
1 in 5,000
Rare diseases affect roughly 1 in 5,000 people. We are building therapies for those who have been left behind.
300M+
People worldwide are living with a rare disease, most with no approved treatment.
95%
Of rare diseases currently have no approved therapy. We are working to change that.
Our Platform
A Platform Built for Precision
Gene Delivery
Our proprietary delivery vectors are engineered for precision — targeting the right cells, in the right tissue, with minimal off-target effects.
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Cell Engineering
We engineer patient-derived cells to carry corrective genetic payloads, creating living therapies that can persist and function long-term.
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Translational Science
From bench to bedside, our translational team bridges discovery and clinical development — accelerating the path to patients.
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Programs in Development
TRB-001
Rare Metabolic Disorder
TRB-002
Inherited Retinal Disease
Investors
Partner With Us
We are seeking strategic partners and investors who share our conviction that gene therapy can rewrite the future for patients with rare diseases. If you are building the future of medicine, we want to build it together.