Ucl Business Plc

Royal Holloway and Bedford New College

Vitaflo International Ltd

Matthew Charles Walker

Robin Simon Brooke Williams

Stephanie Schorge

Dimitri Michael Kullmann

Dennis Kaetzel

Simon Eaton

Simon Heales

Patricia Rutherford

Robin Williams

Aziza Khabbush

Maura Louise ODonnell

Elodie Chabrol

Albert Snowball

Robin Simon Williams

Dimitri M Kullmann

A compound having the formula (I) R1-COOH. R1 is an alkyl or alkenyl group having a Cbackbone, optionally branched with a Calkyl group at any C position in the backbone, or a pharmaceutically acceptable salt, amide or ester thereof. The backbone of the alkyl or alkenyl group, and/or the branched alkyl groups, are optionally interrupted by one or more heteroatoms, provided that when R1 is an alkyl group having a Cbackbone, the branching does not consist only of a hexyl group at the a carbon of R1, or only of a methyl group at the γ carbon of R1, or of only single methyl groups at both the β and ω-1 carbons of R1, and provided that when R1 is an alkyl group having a Cor Cbackbone, the branching does not consist only of a propyl group at the a carbon of R1.

Therapeutic use of compounds

The invention provides expression vectors, nucleic acids, vector particles and methods of treatment involving these vector particles, comprising an engineered KCNA1 gene encoding an edited Kv1.1 potassium channel, as well as methods of confirming the presence of engineered KCNA1 mRNA in a cell. The features of the engineered KCNA1 gene combine to advantageously enhance the translation and activity of the Kv1.1 protein and improve detection of KCNA1 gene expression in a cell and can be used for example in the treatment of epilepsy and similar neurological disorders.

Expression vectors comprising engineered genes

A compound having the formula (I) R1-COOH. R1 is an alkyl or alkenyl group having a Cbackbone, optionally branched with a Calkyl group at any C position in the backbone, or a pharmaceutically acceptable salt, amide or ester thereof. The backbone of the alkyl or alkenyl group, and/or the branched alkyl groups, are optionally interrupted by one or more heteroatoms, provided that when R1 is an alkyl group having a Cbackbone, the branching does not consist only of a hexyl group at the a carbon of R1, or only of a methyl group at the &#x3b3; carbon of R1, or of only single methyl groups at both the &#x3b2; and &#x3c9;-1 carbons of R1, and provided that when R1 is an alkyl group having a Cor Cbackbone, the branching does not consist only of a propyl group at the a carbon of R1.

A composition comprising a decanoic acid to octanoic acid ratio of 70:30 to 90:10 wt/wt for use in treating epilepsy or controlling epileptic seizures.

Composition for use in the treatment of epilepsy

A compound having the formula (I) R1-COOH wherein R1 is an alkyl or alkenyl group having a Cbackbone, optionally branched with a Calkyl group at any C position in the backbone, or a pharmaceutically acceptable salt, amide or ester thereof, wherein the backbone of the alkyl or alkenyl group, and/or the branched alkyl groups, are optionally interrupted by one or more heteroatoms, provided that when R1 is an alkyl group having a Cbackbone, the branching does not consist only of a hexyl group at the a carbon of R1, or only of a methyl group at the &#x3b3; carbon of R1, or of only single methyl groups at both the &#x3b2; and &#x3c9;&#x2212;1 carbons of R1, and provided that when R1 is an alkyl group having a Cor Cbackbone, the branching does not consist only of a propyl group at the a carbon of R1, for use in the treatment or prevention of a disease or a biomedical condition selected from seizure-related disorders, bipolar disorders, mania, depression, migraine, attention deficit hyperactivity disorders, latent HIV infection, Alzheimer's disease, chorea, schizophrenia, ischemia, cancer and fatal blood loss.

The invention provides methods and materials for treating a seizure disorder such as epilepsy in a patient which employ a vector encoding a modified receptor, the so-called 'DREADD' receptor being characterised by (i) a decreased responsiveness to its endogenous activating ligand (ii) a retained or enhanced responsiveness to an exogenous agonist. The modified receptor is expressed in neurons of a seizure focus in brain of the patient, and an exogenous agonist is administered which activates the modified receptor to reversibly alters the excitability of the neurons in the seizure focus leading to synaptic silencing or other inhibition.

Combined use of a vector encoding a modified receptor and its exogenous agonist in the treatment of seizures

The invention relates to pharmaceutical uses of compounds of formula (II). Particular aspects of the invention relate to the use of those compounds in treating, preventing or ameliorating a seizure-related disorder, bipolar disorder, mania, migraine, Alzheimer's disease, Parkinson's disease or stroke.

Growing community of inventors

London, United Kingdom

Matthew Charles Walker