About UsWe are an early stage biopharmaceutical company focused on developing novel protein-based therapies to treat inherited diseases for which there are few alternative therapies. We engineer these proteins so that they are able to travel from the bloodstream across the cell membrane into the cell, where they correct metabolic defects. Our first goal is to develop an enzyme replacement therapy for Barth Syndrome, an inherited disorder characterized by mutations in the Tafazzin gene.
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Our Vision |
Many inherited disorders result from defective or absent proteins critical for normal cellular function. Since these diseases are often rare, progressive and lethal, few treatments exist and patients are often left in despair. By replacing deficient proteins, we will provide patients who suffer from these diseases with restorative therapy that will improve both quality and quantity of life and provide hope for the future.
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Our Values |
We aim to develop new treatment options for those patients that most need attention today- patients with severe and intractable diseases. We will develop tomorrow’s therapies today.
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