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1. WO2020102667 - VECTORS COMPRISING A NUCLEIC ACID ENCODING LYSOSOMAL ENZYMES FUSED TO A LYSOSOMAL TARGETING SEQUENCE

Publication Number WO/2020/102667
Publication Date 22.05.2020
International Application No. PCT/US2019/061701
International Filing Date 15.11.2019
IPC
C12N 15/86 2006.01
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 15/864 2006.01
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
864Parvoviral vectors
C07K 14/65 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
575Hormones
65Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
A61K 38/30 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
22Hormones
30Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
Applicants
  • ASKLEPIOS BIOPHARMACEUTICAL, INC. [US]/[US]
Inventors
  • O'CALLAGHAN, Michael W.
  • FRANCOIS, Achille
Agents
  • BENN, Susanna C.
  • EISENSTEIN, Ronald I.
  • BRAICH, Ravinderjit
  • FITZGERALD, Mark J.
  • RESNICK, David S.
  • CROSBY, David F.
  • DE REGE THESAURO, Francesco
  • HUFF, Shayne Y.
  • KLING, Nicole D.
  • PTASHKA, Teresa A.
Priority Data
62/768,64516.11.2018US
62/769,69720.11.2018US
62/778,70612.12.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) VECTORS COMPRISING A NUCLEIC ACID ENCODING LYSOSOMAL ENZYMES FUSED TO A LYSOSOMAL TARGETING SEQUENCE
(FR) VECTEURS COMPRENANT UN ACIDE NUCLÉIQUE CODANT POUR DES ENZYMES LYSOSOMALES FUSIONNÉES À UNE SÉQUENCE DE CIBLAGE LYSOSOMALE
Abstract
(EN)
Vectors including viral vectors comprising a genome comprising a heterologous nucleic acid encoding a lysosomal targeting sequence, fused to a lysosomal storage enzyme, enabling the lysosomal enzyme to be targeted to the lysosomes. Particular embodiments relate to a recombinant viral vector, e.g., rAAV vector encoding a lysosomal enzyme, having a lysosomal targeting IGF2(V43) sequence that binds human cation-independent mannose-6-phosphate receptor (CI-MPR) or to the IGF2 receptor, permitting proper subcellular localization of the lysosomal enzyme polypeptide to lysosomes. Also encompassed are therapeutic fusion proteins encoded by the viral vector, non-viral vectors, cells, and methods to treat a glycogen storage disease, e.g., those listed in Table 4A or Table 5A with the viral vector.
(FR)
L'invention concerne des vecteurs comprenant des vecteurs viraux présentant un génome comprenant un acide nucléique hétérologue codant pour une séquence de ciblage lysosomale, fusionnés à une enzyme de stockage lysosomale, permettant à l'enzyme lysosomale d'être ciblée sur les lysosomes. Des modes de réalisation particuliers concernent un vecteur viral recombinant, par exemple, un vecteur rAAV codant pour une enzyme lysosomale, ayant une séquence IGF2(V43) de ciblage lysosomale qui se lie au récepteur humain mannose-6-phosphate indépendant des cations (CI-MPR) ou au récepteur IGF2, permettant une localisation subcellulaire appropriée du polypeptide enzymatique lysosomal dans des lysosomes. L'invention concerne également des protéines de fusion thérapeutiques codées par le vecteur viral, des vecteurs non viraux, des cellules, ainsi que des procédés pour traiter une maladie de stockage du glycogène, par exemple celles figurant dans le tableau 4A ou le tableau 5A avec le vecteur viral.
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