Home » Therapeutics – MRC

We manage a wide range of Medical Research Council technologies that are available for licensing or further collaborative development.

Therapeutic pipeline

Molecule name
Therapeutic area
Potential indication
Stage
Modality
Source
R15B Inhibitors
Neuroscience
Neurodegenerative and Endoplasmic Reticulum (ER) – related stress diseases
Preclinical Development
Small molecules
MRC

R15B Inhibitors

Modality

Small molecules

Therapeutic area

Neuroscience

Advantages

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

Background info

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).

Indication

Neurodegenerative and Endoplasmic Reticulum (ER) – related stress diseases

Intellectual property

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

SiR-A novel vector for CRISPR delivery into the CNS
Neuroscience
Central nervous system disorders
Early Discovery
Advanced therapies
MRC

SiR-A novel vector for CRISPR delivery into the CNS

Modality

Advanced therapies

Therapeutic area

Neuroscience

Advantages

The vector allows for a highly site specific level of infection at a very low concentration. Moreover, retrograde movement of Rabies virus means the vector could be delivered at the spinal cord or peripheral motor neurones, before tracing to a specific cell type deeper in the CNS.

Background info

A neurotropic, attenuated Rabies virus vector that can be used to deliver a payload to the CNS before self-inactivating, thereby allowing for gene editing.

Indication

Central nervous system disorders

Intellectual property

Granted patents and pending patent applications that entered their respective national stage from WO2018203049

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