AR108687A1 - GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A - Google Patents

GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A

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Publication number
AR108687A1
AR108687A1 ARP170100975A ARP170100975A AR108687A1 AR 108687 A1 AR108687 A1 AR 108687A1 AR P170100975 A ARP170100975 A AR P170100975A AR P170100975 A ARP170100975 A AR P170100975A AR 108687 A1 AR108687 A1 AR 108687A1
Authority
AR
Argentina
Prior art keywords
hemophilia
raav
aav
treatment
transthyretin
Prior art date
Application number
ARP170100975A
Other languages
Spanish (es)
Original Assignee
Univ Pennsylvania
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of AR108687A1 publication Critical patent/AR108687A1/en

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Se proporcionan composiciones y regímenes útiles en el tratamiento de la hemofilia A. Las composiciones incluyen virus adenoasociado recombinante (rAAV) con un potenciador de transtiretina y promotor que conduce la expresión de un Factor VIII humano. Reivindicación 1: Un virus adenoasociado recombinante (rAAV) útil como un agente terapéutico dirigido al hígado para la hemofilia A, donde dicho rAAV comprende una cápside de AAV y un genoma de vector allí empaquetado, donde dicho genoma de vector comprende: (a) una secuencia de repetición terminal invertida (ITR) 5’ AAV; (b) un potenciador de transtiretina (enTTR); (c) un promotor de transtiretina (TTR); (d) una secuencia codificadora que codifica un Factor VIII humano que tiene función de coagulación; (e) una ITR de 3’ AAV.Compositions and regimens useful in the treatment of hemophilia A are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter that drives the expression of a human Factor VIII. Claim 1: A recombinant adeno-associated virus (rAAV) useful as a liver-directed therapeutic agent for hemophilia A, wherein said rAAV comprises an AAV capsid and a packaged vector genome, wherein said vector genome comprises: (a) a inverted terminal repeat sequence (ITR) 5 'AAV; (b) a transthyretin enhancer (enTTR); (c) a transthyretin promoter (TTR); (d) a coding sequence encoding a human Factor VIII that has a coagulation function; (e) a 3 ’AAV ITR.

ARP170100975A 2016-04-15 2017-04-17 GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A AR108687A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662323336P 2016-04-15 2016-04-15

Publications (1)

Publication Number Publication Date
AR108687A1 true AR108687A1 (en) 2018-09-19

Family

ID=63667412

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP170100975A AR108687A1 (en) 2016-04-15 2017-04-17 GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A

Country Status (1)

Country Link
AR (1) AR108687A1 (en)

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