Drug intelligence / Profile preview

PRO289

Development stage: Discontinued
Lead developer: BioMarin Pharmaceutical
Modality: Small Molecules
Administration: Oral, Buccal, Sublingual, Rectal, Inhalation, Intranasal, Topical, Transdermal, Subcutaneous, Intramuscular, Intravenous, Intradermal, Vaginal

Overview

**PRO289** is an investigational small molecule developed as a **Huntingtin inhibitor** for the treatment of **Huntington's disease**. It was initially pursued by **Prosensa Therapeutics**, a company later acquired by **BioMarin Pharmaceutical**. The drug targeted the underlying pathology of Huntington's disease by inhibiting the mutant Huntingtin protein, which causes neuronal damage through toxic gain-of-function mechanisms. However, development has been **discontinued** at the preclinical stage, with no further advancement reported.

Targets

HTT (Huntingtin protein)

Beyond the preview

Go deeper on PRO289.

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 PRO289.

Explore the full profile