Some content of this application is unavailable at the moment.
If this situation persist, please contact us atFeedback&Contact
1. (WO2018160582) ADENO-ASSOCIATED VIRUS (AAV) CLADE F VECTOR AND USES THEREFOR
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2018/160582 International Application No.: PCT/US2018/019992
Publication Date: 07.09.2018 International Filing Date: 27.02.2018
IPC:
C12N 7/00 (2006.01) ,A61K 35/76 (2015.01) ,A61K 48/00 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
7
Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35
Medicinal preparations containing material or reaction products thereof with undetermined constitution
66
Materials from micro-organisms
76
Viruses
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Applicants:
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, Suite 200 Philadaelphia, PA 19104, US
Inventors:
WILSON, James, M.; US
WANG, Qiang; US
GILES, April; US
TURNER, Kevin; US
Agent:
KODROFF, Cathy, A.; US
DUMINIAK, Robert, J.; US
BAK, William; US
BAK, Mary, E.; US
SCHALLER, Colleen, M.; US
SMITH, George, A.; US
Priority Data:
62/464,74828.02.2017US
62/591,00227.11.2017US
62/614,00205.01.2018US
Title (EN) ADENO-ASSOCIATED VIRUS (AAV) CLADE F VECTOR AND USES THEREFOR
(FR) VECTEUR DE CLADE F DE VIRUS ADÉNO-ASSOCIÉ (AAV) ET SES UTILISATIONS
Abstract:
(EN) A recombinant adeno-associated virus (rAAV) vector comprising an AAVhu68 capsid produced in a production system comprising a nucleotide sequence of SEQ ID NO: 1, or a sequence at least 75% identical thereto which encodes SEQ ID NO:2. The AAVhu68 capsid comprises subpopulations of highly deamidated asparagine residues in asparagine - glycine pairs in the amino acid sequence of SEQ ID NO: 2. Also provided are compositions containing the rAAV and uses thereof. Additionally, rAAV having an engineered AAV capsid comprising at least one subpopulation of vpl or vp2 proteins having a Val at amino acid position 157 with reference to the AAVhu68 vpl numbering are provided.
(FR) L'invention concerne un vecteur de virus adéno-associé recombinant (rAAV) comprenant une capside AAVhu68 produite dans un système de production comprenant une séquence nucléotidique SEQ ID NO: 1, ou une séquence au moins identique à 75 % à celle-ci qui code pour la SEQ ID NO: 2. La capside AAVhu68 comprend des sous-populations de résidus asparagine hautement désamidés dans des paires asparagine-glycine dans la séquence d'acides aminés SEQ ID NO: 2. L'invention concerne également des compositions comprenant le rAAV et des utilisations de celles-ci. De plus, l'invention concerne un rAAV ayant une capside AAV modifiée comprenant au moins une sous-population de protéines vpl ou vp2 ayant une Val à la position de l'acide aminé 157 par rapport à la numérotation de l'AAVhu68 vpl.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)