Title:
SYNTHETIC PRODUCTION OF SINGLE-STRANDED ADENO ASSOCIATED VIRAL DNA VECTORS
Document Type and Number:
WIPO Patent Application WO/2021/011842
Kind Code:
A9
Abstract:
The present application discloses methods for synthetic production and cell-free synthesis of single stranded adeno-associated virus (AAV) vectors, for delivery and expression of a transgene in host cells. The present invention also relates to an in vitro process for production of closed-ended DNA vectors and corresponding single stranded AAV DNA vector products synthesized from the closed-ended DNA vectors having nicks.
Inventors:
ALKAN OZAN (US)
KOTIN ROBERT (US)
KERR DOUGLAS (US)
MONDS RUSSELL (US)
PELLETIER CAROLYN (US)
STANTON MATTHEW (US)
KOTIN ROBERT (US)
KERR DOUGLAS (US)
MONDS RUSSELL (US)
PELLETIER CAROLYN (US)
STANTON MATTHEW (US)
Application Number:
PCT/US2020/042449
Publication Date:
March 25, 2021
Filing Date:
July 17, 2020
Export Citation:
Assignee:
GENERATION BIO CO (US)
International Classes:
A61K48/00; C12N15/09; C12N15/64; C12N15/66
Attorney, Agent or Firm:
CLARKE, Marcie, B. et al. (US)
Download PDF:
Previous Patent: COMPOSITIONS AND METHODS FOR THE TREATMENT OF SANFILIPPO DISEASE AND OTHER DISORDERS
Next Patent: AXIAL PUMP ASSEMBLIES
Next Patent: AXIAL PUMP ASSEMBLIES