VECTORS OF FVIII FACTOR BASED ON ADENO-ASSOCIATED VIRUSES, CORRESPONDING VIRUS PARTICLES AND THERAPEUTIC COMPOSITIONS CONTAINING THEM Russian patent published in 2021 - IPC A61K39/12 C07K14/755 

Abstract RU 2742352 C2

FIELD: biotechnology.

SUBSTANCE: described is a group of inventions comprising a pharmaceutical composition for treating haemophilia A, comprising a recombinant virus AAB5-FVIII-SQ in a concentration of about 1E12 vg/ml to about 2E14 vg/ml, method of treating a subject suffering from hemophilia A, a method for reducing bleeding time in a haemorrhage episode in a subject suffering from haemophilia A, a method for increasing expression of factor VIII protein in a subject, method of treating a subject suffering from hemophilia A, comprising steps of determining the absence of antibodies to the AACB capsid in the serum of said subject and administering the above pharmaceutical composition, and a method of treating a subject suffering from hemophilia A, comprising the steps of administering to said subject a pharmaceutical composition and, after administering said pharmaceutical composition, determining the absence or presence of the antibodies to the AACB capsid in the serum of said subject.

EFFECT: invention extends the range of products for treating haemophilia A.

26 cl, 49 dwg, 1 tbl, 8 ex

Similar patents RU2742352C2

Title Year Author Number
VECTORS OF EXPRESSION OF VIII FACTOR BASED ON ADENO-ASSOCIATED VIRUS 2014
  • Kolosi Piter Kemeron
  • Natvani Amit
  • Makintosh Dzhenni
  • Taddenkhem Edvard
RU2710307C2
FACTOR VIII EXPRESSION VECTORS BASED ON AN ADENO-ASSOCIATED VIRUS 2014
  • Colosi, Peter, Cameron
  • Nathwani, Amit
  • Mcintosh, Jenny
  • Tuddenham, Edward
RU2777372C2
NON-VIRAL DNA VECTORS AND OPTIONS FOR THEIR USE FOR EXPRESSION OF THERAPEUTIC AGENT BASED ON PHENYLALANINE HYDROXYLASE (PAH) 2020
  • Kerr, Duglas Antoni
  • Samajoa, Fillip
  • Silver, Nataniel
  • Chiokko, Mettyu
RU2814137C2
NON-VIRAL DNA VECTORS AND OPTIONS FOR THEIR USE FOR EXPRESSION OF THERAPEUTIC AGENT BASED ON FACTOR VIII (FVIII) 2020
  • Kerr, Duglas, Antoni
  • Klatte, Debra
  • Samajoa, Fillip
  • Silver, Nataniel
RU2812852C2
NON-VIRAL DNA VECTORS AND THEIR USE FOR THE PRODUCTION OF ANTIBODIES AND FUSION PROTEINS 2019
  • Alkan, Ozan
  • Kerr, Duglas Entoni
  • Kotin, Robert Majkl
  • Klatte, Debra
  • Liu, Lea
  • Silver, Nataniel
RU2800914C2
CLOSED-ENDED DNA VECTORS OBTAINED BY CELL-FREE SYNTHESIS, AND METHOD OF PRODUCING CEDNA VECTORS 2019
  • Alkan, Ozan
  • Kotin, Robert Majkl
  • Stanton, Metyu
  • Kerr, Duglas Entoni
  • Pelleter, Karolin
RU2820586C2
STABLE EXPRESSION OF VECTORS BASED ON ADENO-ASSOCIATED VIRUS IN MINOR PATIENTS 2019
  • Bunting, Stuart
RU2792432C2
CONTROLLED EXPRESSION OF TRANSGENES USING CLOSED-END DNA VECTORS (ceDNA) 2019
  • Kerr, Duglas A.
  • Stanton, Metyu Dzh.
  • Chiokko, Mett
  • Angelino, Mark D.
  • Kotin, Robert M.
  • Samajoa, Fillip
RU2816871C2
METHODS FOR TREATMENT OF PHENYLKETONURIA 2019
  • Berguig, Dzheffri
  • Makhimkar, Radzhiv
  • Akife, Khassibulla
  • Kolozi, Piter
RU2788484C2
GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A 2017
  • Wang, Lili
  • Wilson, James M.
  • Sidrane, Jenny Agnes
RU2762257C2

RU 2 742 352 C2

Authors

Bunting, Stuart

Colosi, Peter, Cameron

Pungor, Erno

Dates

2021-02-05Published

2016-09-23Filed