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Neurodon rare disease program

Development stage: Preclinical
Lead developer: Neurodon
Modality: Small Molecules

Overview

Neurodon's rare disease program focuses on the development of small molecule activators of the Sarco/Endoplasmic Reticulum Ca2+-ATPase (SERCA) enzyme. These compounds are designed to restore intracellular calcium homeostasis and alleviate endoplasmic reticulum (ER) stress, which are fundamental pathological features in several neurodegenerative and skin disorders. By accelerating the activity of SERCA, the therapy aims to correct aberrant calcium signaling. The program is currently in the lead optimization stage of preclinical development, targeting indications such as Huntington's disease, amyotrophic lateral sclerosis (ALS), and Darier disease.

Other names: Rare Disease Program (unnamed)

Targets

ATP2A (Sarcoplasmic reticulum Ca2+-ATPase)

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