Quality and experience inked in years of recognition by top experts:

Supporting FDA IND submission

  •  Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, Zelenaia O, Mingozzi F, Hui D, Chung D, Rex TS, Wei Z, Qu G, Zhou S, Zeiss C, Arruda VR, Acland GM, Dell'Osso LF, High KA, Maguire AM, Bennett J. Mol Ther. 2008 Mar;16(3):458-65. Epub 2008 Jan 22.
  • AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models. Vasireddy V, Mills JA, Gaddameedi R, Basner-Tschakarjan E, Kohnke M, Black AD, Alexandrov K, Zhou S, Maguire AM, Chung DC, Mac H, Sullivan L, Gadue P, Bennicelli JL, French DL, Bennett J. PLoS One. 2013 May 7;8(5):e61396. doi: 10.1371/journal.pone.0061396. Print 2013.

Unparallel high yield for large animal studies

  • Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC. Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20.
  • The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy. Finn JD, Nichols TC, Svoronos N, Merricks EP, Bellenger DA, Zhou S, Simioni P, High KA, Arruda VR. Blood. 2012 Nov 29;120(23):4521-3. doi: 10.1182/blood-2012-06-440123. Epub 2012 Aug 23.
  • Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.Mingozzi F, Chen Y, Murphy SL, Edmonson SC, Tai A, Price SD, Metzger ME, Zhou S, Wright JF, Donahue RE, Dunbar CE, High KA. Mol Ther. 2012 Jul;20(7):1410-6. doi: 10.1038/mt.2012.84. Epub 2012 May 8.

Ability to manufacture various capsid serotypes

  • Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses. Ciesielska A, Hadaczek P, Mittermeyer G, Zhou S, Wright JF, Bankiewicz KS, Forsayeth J. Mol Ther. 2013 Jan;21(1):158-66. doi: 10.1038/mt.2012.167. Epub 2012 Aug 28.
  • Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. Jiang H, Lillicrap D, Patarroyo-White S, Liu T, Qian X, Scallan CD, Powell S, Keller T, McMurray M, Labelle A, Nagy D, Vargas JA, Zhou S, Couto LB, Pierce GF. Blood. 2006 Jul 1;108(1):107-15. Epub 2006 Mar 7.
  • Adeno-associated virus (AAV) capsid genes isolated from rat and mouse liver genomic DNA define two new AAV species distantly related to AAV-5. Lochrie MA, Tatsuno GP, Arbetman AE, Jones K, Pater C, Smith PH, McDonnell JW, Zhou SZ, Kachi S, Kachi M, Campochiaro PA, Pierce GF, Colosi P. Virology. 2006 Sep 15;353(1):68-82. Epub 2006 Jun 30.
  • Novel caprine adeno-associated virus (AAV) capsid (AAV-Go.1) is closely related to the primate AAV-5 and has unique tropism and neutralization properties.Arbetman AE, Lochrie M, Zhou S, Wellman J, Scallan C, Doroudchi MM, Randlev B, Patarroyo-White S, Liu T, Smith P, Lehmkuhl H, Hobbs LA, Pierce GF, Colosi P. J Virol. 2005 Dec;79(24):15238-45.

Used in an array of tissue targets





20 years of proven experience

Robust ZFN-mediated genome editing in adult hemophilic mice. Anguela XM, Sharma R, Doyon Y, Miller JC, Li H, Haurigot V, Rohde ME, Wong SY, Davidson RJ, Zhou S, Gregory PD, Holmes MC, High KA. Blood. 2013 Oct 1. [Epub ahead of print]

Versatile adeno-associated virus 2-based vectors for constructing recombinant virions. Nahreini P, Woody MJ, Zhou SZ, Srivastava A. Gene. 1993 Feb 28;124(2):257-62.