The present disclosure provides certain compositions and methods that may be useful in the treatment and/or prevention of a malignant, neurodegenerative, cardiovascular, metabolic, inflammatory, autoimmune, or viral disease or disorder, such as carcinomas, Alzheimer's and Parkinson's disease, multiple sclerosis, Paget's disease, or other aspects of aging, such as atherosclerosis or type-2 diabetes. In some such embodiments, compositions are provided that contain at least one cyclodextrin active agent, such as alpha-cyclodextrin, or an analogue or derivative thereof. In some embodiment the composition is a clathrate of HP-aCD and sodium caprate or caprylate.

The present disclosure provides certain compositions and methods that may be useful in the treatment and/or prevention of a malignant, neurodegenerative, cardiovascular, metabolic, inflammatory, autoimmune, or viral disease or disorder, such as carcinomas, Alzheimer's and Parkinson's disease, multiple sclerosis, Paget's disease, or other aspects of aging, such as atherosclerosis or type-2 diabetes. In some such embodiments, compositions are provided that contain at least one cyclodextrin active agent, such as alpha-cyclodextrin, or an analogue or derivative thereof. In some embodiment the composition is a clathrate of HP-aCD and sodium caprate or caprylate.

Concentrated, low-viscosity, low-volume liquid pharmaceutical formulations of polysaccharides and nucleic acids have been developed. Such formulations can be rapidly and conveniently administered by subcutaneous or intramuscular injection, rather than by lengthy intravenous infusion. These formulations include low-molecular-weight and/or high-molecular-weight polysaccharides and nucleic acids, and viscosity-lowering agents.

Concentrated, low-viscosity, low-volume liquid pharmaceutical formulations of polysaccharides and nucleic acids have been developed. Such formulations can be rapidly and conveniently administered by subcutaneous or intramuscular injection, rather than by lengthy intravenous infusion. These formulations include low-molecular-weight and/or high-molecular-weight polysaccharides and nucleic acids, and viscosity-lowering agents.

In some embodiments, the present disclosure provides certain compositions and methods that may be useful in the treatment and/or prevention of malignant, neurodegenerative, vascular, metabolic, inflammatory, autoimmune, pulmonary, fibrotic, hepatic, lysosomal storage, or viral disease, disorder, or condition, such as carcinomas, Alzheimer's and Parkinson's disease, multiple sclerosis, Paget's disease, or other aspects of aging, such as atherosclerosis or type-2 diabetes. In some such embodiments, compositions are provided that contain at least one cyclodextrin active agent, such as alpha-cyclodextrin, or an analogue or derivative thereof. In some embodiments, the composition is a clathrate of hydroxypropyl-alpha-cyclodextrin and sodium caprate.

In some embodiments, the present disclosure provides certain compositions and methods that may be useful in the treatment and/or prevention of malignant, neurodegenerative, vascular, metabolic, inflammatory, autoimmune, pulmonary, fibrotic, hepatic, lysosomal storage, or viral disease, disorder, or condition, such as carcinomas, Alzheimer's and Parkinson's disease, multiple sclerosis, Paget's disease, or other aspects of aging, such as atherosclerosis or type-2 diabetes. In some such embodiments, compositions are provided that contain at least one cyclodextrin active agent, such as alpha-cyclodextrin, or an analogue or derivative thereof. In some embodiments, the composition is a clathrate of hydroxypropyl-alpha-cyclodextrin and sodium caprate.

This disclosure provides mixtures of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, for use as a pharmaceutically active ingredient; methods of making such mixtures; methods of qualifying such mixtures for use in a pharmaceutical composition suitable for intrathecal or intracerebroventricular administration; pharmaceutical compositions suitable for intrathecal or intracerebroventricular administration comprising such mixtures; and methods of using the pharmaceutical compositions for treatment of Niemann-Pick disease Type C.

This disclosure provides mixtures of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, for use as a pharmaceutically active ingredient; methods of making such mixtures; methods of qualifying such mixtures for use in a pharmaceutical composition suitable for intrathecal or intracerebroventricular administration; pharmaceutical compositions suitable for intrathecal or intracerebroventricular administration comprising such mixtures; and methods of using the pharmaceutical compositions for treatment of Niemann-Pick disease Type C.

The present invention provides processes for the preparation of sugammadex: (I) In one aspect, there is provided a process for the preparation of sugammadex from 8-per-deoxy-8-bromo-γ-cyclodextrin and 3-mercaptopropionic acid. In another aspect, there is provided an alternative process for the preparation of sugammadex from 8-per-deoxy-8-bromo-γ-cyclodextrin and disodium 3 -mercaptopropionate. In another aspect, there is provided a process for the preparation of 8-per-deoxy-8-bromo-γ-cyclod extrin, which may be used in the production of sugammadex. In one such aspect, there is provided a process for the preparation of 8-per-deoxy-8-bromo-y-cyclodextrin from γ-cyclodextrin and a brominating agent. In another such aspect, there is provided a process for the preparation of 8-per-deoxy-8-bromo-γ-cyclodextrin comprising, inter alia, reacting γ-cyclodextrin with an electrophilic brominating agent, a deoxygenating agent, and an acid in the presence of an organic solvent. ##STR00001##

The present disclosure generally relates to methods and formulations for treating central nervous system (CNS) disorders. The present disclosure involves intranasal delivery of at least one antioxidant compound, allowing for effective treatment of a central nervous system disorder such as traumatic brain injury or stroke.