Our Mission

At TransformaTx Biotherapeutics LLC, our mission is to revolutionize the treatment of rare genetic disorders, starting with Angelman syndrome, through the development of innovative gene therapies. We are committed to bringing transformative solutions to patients who need them the most, using cutting-edge science to restore health and hope.

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At TransformaTx, we believe in the power of collaboration between patient advocacy groups, researchers, and world-class scientists. We are driven by the understanding that rare disease drug development requires not only scientific innovation but also a fierce commitment to patient safety and well-being. Our approach is rooted in science, collaboration, and the relentless pursuit of transformational treatments.