The Rett Syndrome Research Trust Funds Herophilus’ Rett Syndrome Lead Candidate Studies

Excerpt from the Press Release:

SAN FRANCISCO–(BUSINESS WIRE)–Herophilus announced today that in vivo studies underway of its lead candidate HRP-12975 are funded in part by the Rett Syndrome Research Trust (RSRT). HRP-12975 is the first small molecule therapy for Rett Syndrome with the potential to reverse the root cause of the disease, MECP2 deficiency.

HRP-12975 is the first novel disease-modifying chemical entity discovered with any brain organoid discovery platform and thus represents an industry first and validation for the approach of human-led in vitro experimental drug discovery. HRP-12975 causes reactivation of the MECP2 gene on the silenced X chromosome in brain cells of Rett patients. This strategy avoids the risk of MECP2 overexpression inherent with many proposed gene therapies. MECP2 overexpression is known to cause MECP2 duplication syndrome, an extremely severe and often fatal neurodevelopmental disease.

HRP-12975 was discovered by screening a highly curated chemical library of 12,000 target-annotated compounds for MeCP2 reactivation in neurons and astrocytes using 3D Rett patient organoid models on Herophilus’ discovery platform.

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