COMPOSITIONS AND METHODS FOR ENHANCING N-ACETYLGALACTOSAMINE-6-SULFATASE ENZYME ACTIVITY AND TREATING MUCOPOLYSACCHARIDOSIS TYPE IVA

Carlos Javier Alméciga Díaz; Jacobo Cepeda Del Castillo; Esteban Guzmán; Oscar Alejandro Hidalgo Lanussa; Sergio Olarte Avellaneda; Edwin Alexander Rodriguez López; Wei Zheng

COMPOSITIONS AND METHODS FOR ENHANCING N-ACETYLGALACTOSAMINE-6-SULFATASE ENZYME ACTIVITY AND TREATING MUCOPOLYSACCHARIDOSIS TYPE IVA

Carlos Javier Alméciga Díaz (Inventor)
, Jacobo Cepeda Del Castillo (Inventor)
, Esteban Guzmán (Inventor)
, Oscar Alejandro Hidalgo Lanussa (Inventor)
, Sergio Olarte Avellaneda (Inventor)
, Edwin Alexander Rodriguez López (Inventor)
, Wei Zheng (Inventor)

National Center for Advancing Translational Science (NCATS) National Institutes of Health (NIH)

Research output: Innovation Products › Invention patent

Abstract

The present invention relates to the development of pharmacological chaperones that bind GALNS and compositions thereof. The present invention further relates to the development of methods for treating mucopoiesisaca ridosis type IVA, which comprise administering a therapeutically effective amount of compositions comprising a pharmacological chaperone that binds to GALNS and optionally human recombinant N-acetylgalactosamine-6-sulfatase (hrGALNS). Additionally, the present invention relates to a method for increasing the production of hrGALNS.

Original language	English
Publication/registration number	WO2021250620
International Classification	A61P 43/ 00 A I
Grant date	12/06/20
Filing date	10/06/21
State	In preparation - 16 Dec 2021

Cite this

@misc{8bf7fb8d78474dfca69a75cf1e8d15f1,

title = "COMPOSITIONS AND METHODS FOR ENHANCING N-ACETYLGALACTOSAMINE-6-SULFATASE ENZYME ACTIVITY AND TREATING MUCOPOLYSACCHARIDOSIS TYPE IVA",

abstract = "The present invention relates to the development of pharmacological chaperones that bind GALNS and compositions thereof. The present invention further relates to the development of methods for treating mucopoiesisaca ridosis type IVA, which comprise administering a therapeutically effective amount of compositions comprising a pharmacological chaperone that binds to GALNS and optionally human recombinant N-acetylgalactosamine-6-sulfatase (hrGALNS). Additionally, the present invention relates to a method for increasing the production of hrGALNS.",

author = "\{Alm{\'e}ciga D{\'i}az\}, \{Carlos Javier\} and \{Cepeda Del Castillo\}, Jacobo and Esteban Guzm{\'a}n and \{Hidalgo Lanussa\}, \{Oscar Alejandro\} and \{Olarte Avellaneda\}, Sergio and \{Rodriguez L{\'o}pez\}, \{Edwin Alexander\} and Wei Zheng",

year = "2021",

month = dec,

day = "16",

language = "English",

type = "Patent",

note = "WO2021250620; A61P 43/ 00 A I",

}

TY - PAT

T1 - COMPOSITIONS AND METHODS FOR ENHANCING N-ACETYLGALACTOSAMINE-6-SULFATASE ENZYME ACTIVITY AND TREATING MUCOPOLYSACCHARIDOSIS TYPE IVA

AU - Alméciga Díaz, Carlos Javier

AU - Cepeda Del Castillo, Jacobo

AU - Guzmán, Esteban

AU - Hidalgo Lanussa, Oscar Alejandro

AU - Olarte Avellaneda, Sergio

AU - Rodriguez López, Edwin Alexander

AU - Zheng, Wei

PY - 2021/12/16

Y1 - 2021/12/16

N2 - The present invention relates to the development of pharmacological chaperones that bind GALNS and compositions thereof. The present invention further relates to the development of methods for treating mucopoiesisaca ridosis type IVA, which comprise administering a therapeutically effective amount of compositions comprising a pharmacological chaperone that binds to GALNS and optionally human recombinant N-acetylgalactosamine-6-sulfatase (hrGALNS). Additionally, the present invention relates to a method for increasing the production of hrGALNS.

AB - The present invention relates to the development of pharmacological chaperones that bind GALNS and compositions thereof. The present invention further relates to the development of methods for treating mucopoiesisaca ridosis type IVA, which comprise administering a therapeutically effective amount of compositions comprising a pharmacological chaperone that binds to GALNS and optionally human recombinant N-acetylgalactosamine-6-sulfatase (hrGALNS). Additionally, the present invention relates to a method for increasing the production of hrGALNS.

UR - https://worldwide.espacenet.com/patent/search/family/073823700/publication/CO2020007242A1?q=CO20200007242

M3 - Invention patent

M1 - WO2021250620

Y2 - 2021/06/10

ER -

COMPOSITIONS AND METHODS FOR ENHANCING N-ACETYLGALACTOSAMINE-6-SULFATASE ENZYME ACTIVITY AND TREATING MUCOPOLYSACCHARIDOSIS TYPE IVA

Abstract

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