Drug intelligence / Profile preview

IPS101A

Development stage
Phase 1
Lead developer
Innopeutics
Modality
Gene Therapies
Administration
Intracerebral
01

Overview

IPS101A is an adeno-associated virus (AAV) gene therapy candidate developed by Innopeutics Corporation for the treatment of advanced Parkinson's disease. It is designed to deliver the gene encoding human aromatic L-amino acid decarboxylase (AADC) directly into the substantia nigra of the midbrain via stereotactic injection. AADC is the key enzyme that converts levodopa into dopamine. In advanced Parkinson's disease, the loss of dopaminergic neurons leads to a deficiency of this enzyme, reducing the effectiveness of oral levodopa therapy. By restoring AADC expression, IPS101A aims to enhance dopamine synthesis and improve motor function in patients with severe functional impairment (Hoehn & Yahr stages 4-5) who are refractory to conventional medical management.

02

Targets

DDC (Aromatic L-amino acid decarboxylase)

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