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CDX-6114 is an orally administered, gastrointestinal-stable, modified phenylalanine ammonia lyase (PAL) enzyme developed for the treatment of phenylketonuria (PKU). It acts by converting dietary phenylalanine (Phe) in the gastrointestinal tract into cinnamic acid and ammonia before systemic absorption, thereby reducing blood Phe levels. This mechanism compensates for the missing or deficient natural enzyme in PKU patients. The drug is designed to be stable in the GI tract and offers a potential oral alternative to restrictive diets currently used for PKU management. Clinical trials have shown that single doses are safe and well tolerated[1][6][5].
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