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HER-096 is an engineered peptidomimetic molecule (~1000 Da retro-inverso-isomerized peptide) designed to mimic the activity of cerebral dopamine neurotrophic factor (CDNF), a human protein that promotes cell survival and functional recovery of neurons[1][5][6]. Unlike CDNF, which requires intracranial delivery, HER-096 can cross the blood-brain barrier after subcutaneous administration, enabling more convenient dosing[1][6]. Its mechanism involves modulation of the unfolded protein response (UPR) pathway and protection against endoplasmic reticulum (ER) stress-induced neuronal death. Preclinical studies have shown that HER-096 provides significant neuroprotection in models of synucleinopathy by reducing alpha-synuclein aggregation, microgliosis, and ER stress markers in dopaminergic neurons[1]. The drug also alleviates inflammation in affected brain areas. Clinical trials have demonstrated good safety and tolerability profiles as well as efficient BBB penetration in healthy volunteers; ongoing clinical development focuses on Parkinson’s disease with the aim to provide a disease-modifying therapy rather than symptomatic relief[2][3][4][5].
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