Drug Profile

Research programme: phenylketonuria therapeutics - iECURE

Alternative Names: iECURE-PKU

Latest Information Update: 24 Jun 2022

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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

24 May 2022 Early research in Phenylketonuria in USA (Parenteral) as of May 2022 (iECURE pipeline, May 2022)
10 Sep 2021 iECURE in-licenses PCSK9-Directed ARCUS Nuclease technology from Precision Biosciences for ornithine transcarbamylase deficiency, citrullinemia type 1 (CTLN1) and phenylketonuria (PKU) as of September 2021

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