Drug intelligence / Profile preview

PTI-501

Development stage: Preclinical
Lead developer: Pierrepont Therapeutics
Modality: Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes

Overview

PTI-501, also known by its former code name ENTR-501, is a proprietary intracellular enzyme replacement therapy under development for the treatment of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). MNGIE is a rare, progressive, and fatal mitochondrial disease with no approved treatment options. The drug was originally developed by Entrada Therapeutics and is now exclusively licensed to Pierrepont Therapeutics, a company dedicated to developing targeted treatments for primary mitochondrial and other rare genetic diseases.

Targets

TYMP (Thymidine phosphorylase)

Beyond the preview

Go deeper on PTI-501.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on PTI-501.

Explore the full profile