Affiliation: Bayer HealthCare
- Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapyHaiyan Jiang
Bayer HealthCare Pharmaceuticals, 800 Dwight Way, Berkeley, CA 94710, USA
Blood 108:3321-8. 2006..These findings enable a clinical study to assess the effects of immunomodulation on long-term FIX expression in patients with hemophilia B...
- Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogsHaiyan Jiang
Avigen, Almeda, CA, USA
Blood 108:107-15. 2006..In summary, this is the first report demonstrating multiyear therapeutic efficacy and safety of multiple AAV-cFVIII vectors in hemophilia A dogs and provides the basis for human clinical studies...
- Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia BHaiyan Jiang
Avigen, Inc, Alameda, CA 94502, USA
Mol Ther 14:452-5. 2006..These results demonstrate, for the first time, multiyear FIX expression by AAV2 vector in humans and suggest that improved muscle delivery provides effective treatment for protein deficiencies or muscle-specific diseases...
- Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID miceCiaran D Scallan
Avgen, Alameda, CA, USA
Blood 107:1810-7. 2006..Therefore, novel nonprimate AAV vectors or compartmentalized delivery may offer more consistent therapeutic effects in the presence of pre-existing AAV neutralizing antibodies...
- Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIIICiaran D Scallan
Avigen, Inc, 1201 Harbor Bay Parkway, Alameda, CA 94502, USA
Blood 102:3919-26. 2003..This strategy may potentially be useful for other large therapeutic proteins that contain functionally distinct domains...
- Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatmentBaisong Mei
Global Biological Development, Bayer HealthCare LLC, Berkeley, CA, USA
Blood 116:270-9. 2010..In summary site-specifically PEGylated FVIII has the potential to be a long-acting prophylactic treatment while being fully efficacious for on-demand treatment for patients with hemophilia A...
- Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patientsJerry S Powell
University of California, Davis, Sacramento, CA, USA
Blood 119:3031-7. 2012..Thus, rFVIIIFc may offer a viable therapeutic approach to achieve prolonged hemostatic protection and less frequent dosing in patients with hemophilia A. This trial was registered at www.clinicaltrials.gov as NCT01027377...
- Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liverFederico Mingozzi
Division of Hematology and Center for Cellular and Molecular Therapeutics, The Children s Hospital of Philadelphia, PA 19104, USA
Blood 110:2334-41. 2007....
- Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune responseCatherine S Manno
The Children s Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania, 19104, USA
Nat Med 12:342-7. 2006..We conclude that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels of F.IX, but that future studies in humans may require immunomodulation to achieve long-term expression...
- Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vectorCiaran D Scallan
Avigen, Inc, 1201 Harbor Bay Parkway, Alameda, CA 94502
Blood 102:2031-7. 2003..These data support the use of AAV2 vectors in human clinical trials to treat hemophilia A patients...
- CD8(+) T-cell responses to adeno-associated virus capsid in humansFederico Mingozzi
The Children s Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA
Nat Med 13:419-22. 2007..In addition, we show that AAV-2 induced human T cells proliferate upon exposure to alternate AAV serotypes, indicating that other serotypes are unlikely to evade capsid-specific immune responses...
- The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII geneLingxia Chen
Department of Pediatrics, University of Pennsylvania Medical Center and The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
Mol Ther 15:1856-62. 2007..In sum, enhancing the secretion of the FVIII HC polypeptide may require the proper association of the FVIII LC polypeptide in cis or in trans. These results may be helpful in designing new strategies to improve FVIII gene delivery...