Drug intelligence / Profile preview

PER303

Development stage
Preclinical
Lead developer
Perseo Pharma
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Nanoparticles → Drug Delivery Systems
Administration
Oral
01

Overview

PER303 is an oral enzyme replacement therapy (ERT) being developed by Perseo Pharma for the treatment of Phenylketonuria (PKU). The drug utilizes a proprietary nanotechnology platform that encapsulates the enzyme Phenylalanine Ammonia Lyase (PAL) within a 'nano-shield.' This protective coating is designed to prevent the degradation of the enzyme by gastric acid and proteases in the digestive tract, while also promoting its localization within the intestinal mucus layer. By maintaining the enzyme's activity in the gut, PER303 enables the metabolism of dietary phenylalanine into non-toxic metabolites, such as trans-cinnamic acid and ammonia, before it can be absorbed into the systemic circulation. This mechanism aims to reduce blood phenylalanine levels and provide a more convenient oral alternative to systemic ERT for PKU patients.

02

Targets

PAL (Phenylalanine ammonia-lyase)

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