EMD-201

EMD-201 is a preclinical gene modulator therapy developed by Emendo Biotherapeutics. It utilizes a gene-editing approach to target SARM1 (Sterile Alpha and TIR Motif Containing 1), a protein expressed in neuronal and retinal tissues that regulates axonal degeneration. SARM1 is normally auto-inhibited but becomes activated under cellular stress, leading to Wallerian degeneration—a form of programmed axon self-destruction associated with neurodegenerative diseases. By employing biallelic knockout of the SARM1 gene, EMD-201 aims to provide neuroprotection by rescuing or postponing the death of retinal ganglion cells (RGCs). The initial therapeutic focus for EMD-201 is glaucoma, where it seeks to prevent RGC loss and preserve vision.