Abstract: An isolated nucleic acid sequence including at least two copies of a miRNA target site of the miR183 family having a sequence as set forth in SEQ ID NO: 1, SEQ ID NO: 21, or SEQ ID NO: 24, or a sequence having at least 90% identity with any one of SEQ ID NO: 1, SEQ ID NO: 21, or SEQ ID NO: 24. Also a vector including a regulatory element having at least one copy of this miRNA target site of the miR183 family. The isolated nucleic acid sequence and the vector may be particularly useful for controlling the expression of a gene of interest, for example when designing and developing gene therapies.

Abstract: Azasetron or an analog thereof, or a pharmaceutically acceptable salt and/or solvate thereof, for treating severe or profound sudden sensorineural hearing loss (SSNHL) in a subject in need thereof. In particular, azasetron or an analog thereof, or a pharmaceutically acceptable salt and/or solvate thereof, for treating severe or profound SSNHL in a subject suffering from severe or profound SSNHL associated with (i) a hearing threshold at baseline corresponding to a pure tone audiometry (PTA) at baseline equal to or greater than 70 dB, preferably equal to or greater than 80 dB, more preferably equal to or greater than 90 dB; (ii) a hearing loss at baseline equal to or greater than 91 dB corresponding to a profound hearing loss according to the American Speech-Language-Hearing Association (ASHA) classification; (iii) a hearing loss at baseline affecting frequencies equal to or lower than 2000 Hz; and/or (iv) the presence of vertigo at baseline.

Abstract: Disclosed is a method for treating ear disorders, including the administration of (+)-azasetron, or a pharmaceutically acceptable salt and/or solvate thereof.

Abstract: Disclosed is an inhibitor of calcineurin of the setron family for use for treating hearing loss in a subject in need thereof.

Abstract: The therapeutic use of azasetron or an analogue thereof for treating and/or preventing a lesion in the central auditory nervous system (CANS) in an individual. The treatment of lesions in the CANS with azasetron also prevents, inhibits, and/or reduces the loss of the central auditory neuron cells in the brainstem.

Abstract: A process for manufacturing 2-isobutyl-6-(3-(methylamino)azetidin-1-yl)pyrimidin-4-amine of Formula (I): including starting from 6-chloro-2-isobutylpyrimidin-4-amine and tert-butyl azetidin-3-yl(methyl)carbamate, or another N-protected N-methylazetidin-3-amine, and performing the following steps: (a) coupling reaction of both compounds in dimethylsulfoxide in presence of potassium carbonate to afford an intermediate protected compound; and (b) deprotection of the protected compound to afford 2-isobutyl-6-(3-(methylamino)azetidin-1-yl)pyrimidin-4-amine. Also, a process for manufacturing the intermediate protected compound, wherein deprotection step (b) is omitted, and the compounds obtained from the processes.

Abstract: A process for manufacturing 2-isobutyl-6-(3-(methylamino)azetidin-l-yl)pyrimidin-4-amine of Formula (I): including starting from 6-chloro-2-isobutylpyrimidin-4-amine and tert-butyl azetidin-3-yl(methyl)carbamate, or another N-protected N-methylazetidin-3-amine, and performing the following steps: (a) coupling reaction of both compounds in dimethylsulfoxide in presence of potassium carbonate to afford an intermediate protected compound; and (b) deprotection of the protected compound to afford 2-isobutyl-6-(3-(methylamino)azetidin-1-yl)pyrimidin-4-amine. Also, a process for manufacturing the intermediate protected compound, wherein deprotection step (b) is omitted, and the compounds obtained from the processes.

Abstract: The present invention relates to a calcineurin inhibitor for use in the treatment of a lesional vestibular disorder.

Abstract: Disclosed is an inhibitor of calcineurin of the setron family for use for treating hearing loss in a subject in need thereof.

Abstract: The invention relates to Histamine type 4 receptor (H4R) inhibitors for treating tinnitus.