Drug Profile

AP 003 - Alltrna

Alternative Names: AP-003 - Alltrna; Arg-TGA

Latest Information Update: 11 Jul 2025

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Preclinical Acidaemia; Inborn genetic disorders; Inborn urea cycle disorders; Phenylketonuria

11 Jul 2025 Preclinical trials in Phenylketonuria, Acidaemia and Inborn urea cycle disorder in USA (unspecified route) (Alltrna pipeline, July 2025)
09 Dec 2024 Pharmacodynamics data from preclinical trials in Inborn genetic disorders released by Alltrna
09 Dec 2024 Preclinical trials in Inborn genetic disorders in USA (Parenteral)

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