P-type ATPase agonist (CD3)

The P-type ATPase agonist program is a preclinical drug discovery initiative by the Leuven Centre for Drug Design and Discovery (CD3) focused on developing small molecule therapies for Parkinson's disease. The program specifically targets P-type ATPases, most notably ATP13A2 (also known as PARK9), which is a lysosomal transporter essential for maintaining cellular proteostasis and ion homeostasis. Dysfunction or genetic mutations in ATP13A2 are linked to impaired lysosomal clearance of alpha-synuclein, a hallmark of Parkinson's disease. By pharmacologically activating these ATPases, the small molecules aim to restore lysosomal function, promote the degradation of toxic protein aggregates, and provide neuroprotective effects. This approach represents a potential disease-modifying strategy for Parkinson's and other neurodegenerative disorders characterized by lysosomal failure.

Other names

Leuven Centre for Drug Design and Discovery-p-type ATPase-small molecule-ParkinsonP-type ATPase agonistsATP13A2 activatorsATP-13A2 activatorsATP 13A2 activators