Title:
POTENCY ASSAYS FOR VIRAL VECTOR PRODUCTION
Document Type and Number:
WIPO Patent Application WO/2021/026461
Kind Code:
A3
Abstract:
The present disclosure provides sensitive and robust assays for determining the potency of payloads encoded by recombinant viral vectors. Particularly, the present disclosure provides assays to determine the potency of SMN polypeptide as expressed by recombinant viral vectors used for the treatment of spinal muscular atrophy. The present description encompasses, inter alia, methods for determining potency (e.g., biological activity), e.g., relative potency, of a recombinant viral vector.
Inventors:
FESCHENKO MARINA (US)
BERGELSON SVETLANA (US)
LEYME ANTHONY CHRISTOPHE (US)
BERGELSON SVETLANA (US)
LEYME ANTHONY CHRISTOPHE (US)
Application Number:
PCT/US2020/045423
Publication Date:
March 18, 2021
Filing Date:
August 07, 2020
Export Citation:
Assignee:
BIOGEN MA INC (US)
International Classes:
A61K48/00; A61P25/14; C12N15/864
Domestic Patent References:
| WO2019094253A1 | 2019-05-16 | |||
| WO2019236949A1 | 2019-12-12 | |||
| WO2019210137A1 | 2019-10-31 |
Other References:
DIMITRIADI MARIA, DERDOWSKI AARON, KALLOO GEETIKA, MAGINNIS MELISSA S., O’HERN PATRICK, BLISKA BRYN, SORKAÇ ALTAR, NGUYEN KEN C. Q: "Decreased function of survival motor neuron protein impairs endocytic pathways", PROC. NATL. ACAD. SCI. U.S.A, vol. 113, no. 40, 26 July 2016 (2016-07-26), pages E4377 - E4386, XP055803767
CHERRY JONATHAN J., KOBAYASHI DIONE T., LYNES MAUREEN M., NARYSHKIN NIKOLAI N., TIZIANO FRANCESCO DANILO, ZAWORSKI PHILLIP G., RUB: "Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy", ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES, vol. 12, no. 6, 22 August 2014 (2014-08-22), pages 315 - 341, XP055803772
HINDERER ET AL.: "Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN", HUMAN GENE THERAPY, vol. 29, no. 3, 1 March 2018 (2018-03-01), pages 285 - 298, XP055532829, DOI: 10.1089/hum.2018.015
CHERRY JONATHAN J., KOBAYASHI DIONE T., LYNES MAUREEN M., NARYSHKIN NIKOLAI N., TIZIANO FRANCESCO DANILO, ZAWORSKI PHILLIP G., RUB: "Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy", ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES, vol. 12, no. 6, 22 August 2014 (2014-08-22), pages 315 - 341, XP055803772
HINDERER ET AL.: "Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN", HUMAN GENE THERAPY, vol. 29, no. 3, 1 March 2018 (2018-03-01), pages 285 - 298, XP055532829, DOI: 10.1089/hum.2018.015
Attorney, Agent or Firm:
ADAMS, Melissa M. et al. (US)
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