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First Rare is a biotechnology company focused on developing transformative therapies for rare genetic diseases with high unmet medical needs. The company's lead program is centered on treating cystinosis, a lysosomal storage disorder, by targeting the underlying transport mechanisms of cystine. By leveraging deep expertise in rare disease biology and medicinal chemistry, First Rare aims to improve the standard of care and quality of life for patients suffering from debilitating orphan conditions.
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