Patent classifications
C07C229/10
AMINO ACIDS
In some embodiments, the present disclosure provides amino acid compounds that are useful for producing products such as peptides. In some embodiments, the present disclosure provides peptides comprising residues of provided amino acids.
A LIPID
The invention provides a lipid of Formula (I) and nanoparticle compositions containing the same. Nanoparticle compositions including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
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COMPOUNDS FOR THE TREATMENT OF NEUROLOGICAL OR MITOCHONDRIAL DISEASES
Compounds and methods are provided for the treatment of neurological or mitochondrial diseases, including epilepsy. In some embodiments, the compounds are substituted 1,4-naphthoquinones.
IONIZABLE CATIONIC LIPIDS FOR RNA DELIVERY
The present disclosure describes compounds of Formula (I) and pharmaceutically acceptable salts thereof:
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VACCINE ADJUVANTS AND METHODS OF SYNTHESIZING AND USING THE SAME
The disclosure provides compounds useful as adjuvants in vaccines, as well as methods of synthesizing such compounds and methods of using such compounds in the formulation of a vaccine. The disclosure also features methods of administering such vaccines to a subject (e.g., a mammalian subject, such as a human) in order to treat or prevent one or more diseases, such as a disease caused by a viral or bacterial infection.
SYNTHESIS OF NIROGACESTAT
The present disclosures are directed to processes for synthesizing (S)-2-(((S)-6,8-difluoro-1,2,3,4-tetrahydronaphthalen-2-yl)amino)-N-(1-(2-methyl-1-(neopentylamino)propan-2-yl)-1H-imidazol-4-yl)pentanamide (“nirogacestat”).
SYNTHESIS OF NIROGACESTAT
The present disclosures are directed to processes for synthesizing (S)-2-(((S)-6,8-difluoro-1,2,3,4-tetrahydronaphthalen-2-yl)amino)-N-(1-(2-methyl-1-(neopentylamino)propan-2-yl)-1H-imidazol-4-yl)pentanamide (“nirogacestat”).
Compounds for the treatment of neurological or mitochondrial diseases
Compounds and methods are provided for the treatment of neurological or mitochondrial diseases, including epilepsy. In some embodiments, the compounds are substituted 1,4-naphthoquinones.
SYNTHESIS OF NIROGACESTAT
The present disclosures are directed to processes for synthesizing (S)-2-(((S)-6,8-difluoro-1,2,3,4-tetrahydronaphthalen-2-yl)amino)-N-(1-(2-methyl-1-(neopentylamino)propan-2-yl)-1H-imidazol-4-yl)pentanamide (“nirogacestat”).
SYNTHESIS OF NIROGACESTAT
The present disclosures are directed to processes for synthesizing (S)-2-(((S)-6,8-difluoro-1,2,3,4-tetrahydronaphthalen-2-yl)amino)-N-(1-(2-methyl-1-(neopentylamino)propan-2-yl)-1H-imidazol-4-yl)pentanamide (“nirogacestat”).