Drug intelligence / Profile preview

TW002

Development stage: Preclinical
Lead developer: Treeway
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intrathecal, Parenteral

Overview

TW002 is an adeno-associated virus serotype 5 (AAV5)-mediated gene therapy candidate being developed by Treeway for the treatment of amyotrophic lateral sclerosis (ALS). The therapy is designed to deliver the gene for glial cell line-derived neurotrophic factor (GDNF) directly to the central nervous system. GDNF is a potent neurotrophic factor that supports the survival and function of motor neurons, which are progressively lost in ALS patients. By providing sustained neurotrophic support, TW002 aims to slow disease progression and preserve motor function. It is currently in the preclinical stage of development.

Targets

GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)

Beyond the preview

Go deeper on TW002.

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 TW002.

Explore the full profile