Novel target: phosphatase subunit R15B
Lead series of small molecules at late preclinical development stage
For the potential treatment of a broad spectrum of neurodegenerative diseases which are of high unmet clinical need, such as HD, ALS and PD or other ER-stress disorders (such as Wolcott-Ralisson syndrome)
Disease-modifying approach
Strong patent portfolio

The Bertolotti lab at the MRC Laboratory of Molecular Biology has developed a series of nanomolar inhibitors for the R15B eIF2α phosphatase with selective binding to R15B over R15A. The inhibitors showed efficacy in in vitro and in vivo mouse models of neurodegenerative diseases including Huntington’s Disease (HD), Amyotrophic Lateral Sclerosis (ALS) and Parkinson’s Disease (PD).

Neurodegenerative and Endoplasmic Reticulum (ER) – related stress diseases

WO2016162689A1 (inhibitors and their uses, Priority Date 8 April 2015) and WO2016162688A1 (methods for selecting phosphatase selective and non-selective phosphatase inhibitors, Priority Date 8 April 2015) patent families claim the inhibitors (composition-of-matter), derivatives thereof and their use. Patents are granted in Australia and Europe and are at examination stage in Brazil, Canada, China, Hong Kong, India, Japan, Korea, Mexico, Singapore and US