Drug Profile

Research programme: phenylketonuria therapeutics - iECURE

Alternative Names: iECURE-PKU

Latest Information Update: 04 Oct 2026

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At a glance

Originator University of Pennsylvania
Developer iECURE; University of Pennsylvania
Class Gene therapies
Mechanism of Action Gene transference; Phenylalanine hydroxylase replacements

Orphan Drug Status

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

No
New Molecular Entity No

28 Sep 2026 Early research is ongoing in the USA (iECURE pipeline, September 2026)
28 Jun 2026 No recent reports of development identified for research development in Phenylketonuria in USA (Parenteral)
24 May 2022 Early research in Phenylketonuria in USA (Parenteral) as of May 2022 (iECURE pipeline, May 2022)

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