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1. WO2019213011 - COMPOSITIONS AND METHODS FOR HEMOGLOBIN PRODUCTION

Publication Number WO/2019/213011
Publication Date 07.11.2019
International Application No. PCT/US2019/029787
International Filing Date 30.04.2019
IPC
C07K 14/805 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
795Porphyrin- or corrin-ring-containing peptides
805Haemoglobins; Myoglobins
C12N 15/867 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
867Retroviral vectors
CPC
C07K 14/805
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
795Porphyrin- or corrin-ring-containing peptides
805Haemoglobins; Myoglobins
C12N 15/86
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
C12N 2740/16043
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2740Reverse Transcribing RNA Viruses
00011Reverse Transcribing RNA Viruses
10011Retroviridae
16011Human Immunodeficiency Virus, HIV
16041Use of virus, viral particle or viral elements as a vector
16043viral genome or elements thereof as genetic vector
Applicants
  • THE CHILDREN'S HOSPITAL OF PHILADELPHIA [US]/[US]
Inventors
  • RIVELLA, Stefano
  • BREDA, Laura
  • DONG, Alisa
  • LOURENCO, Silvia, Pires
  • GONZALEZ, Amaliris
Agents
  • NETTER, Robert, C. Jr.
  • HERRELL, Roger, W.
  • PACE, Vincent, T.
  • PIPER, Donald, R.
  • ELAND, Stephen, H.
Priority Data
62/664,78830.04.2018US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR HEMOGLOBIN PRODUCTION
(FR) COMPOSITIONS ET PROCÉDÉS DE PRODUCTION D'HÉMOGLOBINE
Abstract
(EN) In one aspect of the instant invention, lentiviral vectors are provided, particularly for treating hemoglobinopathies. Composition comprising the lentiviral vector are also encompassed by the instant invention. In accordance with another aspect of the instant invention, methods of inhibiting, treating, and/or preventing a hemoglobinopathy (e.g., sickle cell disease or thalassemia) in a subject are provided. In a particular embodiment, the method comprises administering a viral vector of the instant invention to a subject in need thereof hemoglobinopathy. In a particular embodiment, the subject has sickle cell anemia.
(FR) Selon un aspect, la présente invention concerne des vecteurs lentiviraux, en particulier pour le traitement d'hémoglobinopathies. La présente invention concerne également des compositions comprenant le vecteur lentiviral. Selon un autre aspect, la présente invention concerne des procédés d'inhibition, de traitement et/ou de prévention d'une hémoglobinopathie (par exemple, la drépanocytose ou la thalassémie) chez un sujet. Dans un mode de réalisation particulier, le procédé comprend l'administration d'un vecteur viral selon la présente invention à un sujet en ayant besoin d'hémoglobinopathie. Dans un mode de réalisation particulier, le sujet présente une drépanocytose.
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