The present invention relates to compounds of formula (I) that are capable of modulating, e.g., inhibiting or activating, one or more kinases, especially LRRK2 and/or NUAK1 and/or TYK2 or mutants thereof. The compounds are useful for treating diseases, such as autoimmune diseases, inflammatory diseases, bone diseases, metabolic diseases, neurological and neurodegenerative diseases, cancer, cardiovascular diseases, allergies, asthma, Alzheimer's disease, Parkinson's disease, skin disorders, eye diseases, infectious diseases and hormone-related diseases. The present description discloses the synthesis and characterisation of exemplary compounds as well as pharmacological data thereof (e.g. pages 40 to 146; examples 1 to 63; compounds 1 to 248; tables 1 to 3). Preferred compounds are e.g. 1,4-dihydrobenzo[d]pyrazolo[3,4-f] [1,3]diazepine derivatives and related compounds. An exemplary compound is e.g. 5-(2,6-difluorophenyl)-8-methoxy-1,4-dihydrobenzo[d]pyrazolo[3,4-f] [1,3]diazepine (example 49). (Formula (II)).

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The present invention relates to compositions comprising one or more compounds and/or extracts which induce, promote and/or improve production/release/delivery/excretion of mucin from and/or in the cornea, and methods of using the compositions to treat the eye.

Methods of treating a subject having or at risk of developing a neurodegenerative disease or condition associated with demyelination, insufficient myelination, or underdevelopment of myelin sheath are described. The methods include administration of a therapeutically effective amount of sobetirome, or a pharmaceutically acceptable salt thereof.

A method is described in which treatment is carried out with compounds of tetrahydropyridoethers that are administered in form of a medication to treat AMD and to remove lipofuscin from a patient's eye.

The present invention relates to therapeutic use of calreticulin in treatment of angiogenic eye diseases. Different treatment methods and pharmaceutical compositions comprising calreticulin are disclosed.

Aspects of the disclosure relate to a recombinant adeno-virus encoding an anti-Vascular endothelial cell growth factor (VEGF) agent in a cell or subject (e.g., rAAV2.7m8-KH902). In some embodiments, compositions described herein are useful for treating subjects having diseases associated with angiogenesis or aberrant VEGF activity/signaling.

Active agents that bind to VEGF or a VEGF receptor and reduce the severity of a condition associated with BLB disruption and/or angiogenesis, for example anti-VEGF antibodies or tyrosine kinase inhibitor small molecules, can be locally, regionally or systemically administered to an individual with Meniere's Disease to alleviate symptoms of the disease, for example, due to edema and endolymphatic dysfunction. An effective amount of these compounds can be delivered by intratympanic or intracochlear administration. Other methods of administration include, but are not limited to, topical, parenteral, subcutaneous, intraperitoneal and intranasal. Formulations may be, for example, for immediate release, sustained release, or controlled release.

Embodiments of the disclosure encompass methods and compositions for the treatment of medical conditions in which cell regeneration is in need, including neural cells. In specific embodiments, the cell are retinal cells and include Müller glial cells. In particular embodiments, an individual with retinal regeneration is provided an effective amount of one or more histone acetylase inhibitors for the purpose of regenerating retinal cells, including retinal neurons.

The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.

Methods for treating eye diseases associated with inflammation and/or vascular proliferation in subjects are disclosed. The methods include administering therapeutically effective amounts of a tranilast compound, in particular (E)-2-[[3-(3-Methoxy-4-propargyloxy)phenyl)-1-oxo-2-propenyl]amino]benzoic acid or (E)-2-[[3,4-Bis(difluoromethoxy)phenyl)-1-oxo-2-propenyl]amino]benzoic acid or pharmaceutically acceptable salts or solvates thereof.