Preclinical Studies of LM22A-4 in Mouse Models of Rett Syndrome

David Katz, PhD | Case Western Reserve University

$272,000 AWARDED

Studies have identified Brain Derived Neurotrophic Factor (BDNF) and its receptor, TrkB, as promising therapeutic targets for the treatment of Rett.

Studies in the Katz laboratory demonstrated that respiratory abnormalities are reversed in the Rett animal model following systemic treatment with LM22A-4, a compound that boosts TrkB activity.  As a next step towards a potential clinical development path for LM22A-4 for the treatment of Rett, RSRT is supporting the generation of a robust preclinical package of PK/PD (pharmacokinetic/pharmacodynamic), efficacy and safety data in support of a subsequent application for funding of Investigational New Drug (IND) – enabling studies.

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