I joined Pfizer in 2019 to lead the gene therapy research efforts in the Rare Disease Research and Development Unit. Our gene therapy teams located at Cambridge, MA and Morrisville, NC work closely with colleagues within the Rare Disease Unit and partner lines to deliver novel rAAV therapeutic candidates.
I have a BS in Genetics and a BA in Chemistry from University of California, Davis and a PhD in Molecular Biology from University of California, Los Angeles, where I studied bioconjugate chemistry in Dr. David Sigman’s lab. I completed a post-doc fellowship at Genentech in Dr. Robert Lazarus’ lab.
Prior to joining Pfizer, I spent 5 years at Shire, overseeing research for all therapeutic modalities, including protein, antibody, gene therapy, and small molecules. Earlier in my career I spent 8 years each at Genzyme/Sanofi and Bayer leading the protein engineering function.
I am an inventor of Jivi, a marketed third generation FVIII product for the treatment of hemophilia. I have also led the CMC, pharmacology, toxicology, and clinical assay activities through a successful clinical PoC for the development of avalglucosidase alfa, which is currently in a pivotal phase 3 trial for the treatment of the Pompe disease. I have been a co-author on 46 research articles and reviews and a co-inventor on 35 issued US patents.
With a shared urgency and unwavering passion, we hope to unlock the potential of using genes as medicine to make a meaningful difference in patients’ lives—today and in the future. As a leader in researching and developing innovative therapies, Pfizer Rare Disease is focusing on gene therapy as one of the next significant developments in delivering potentially transformative medicines to people suffering from genetic diseases. Pfizer Rare Disease has been working for many years to explore the promise of gene therapy by leveraging our expertise and strategic investments, including manufacturing facilities, new partnerships and collaborations.
- Structure of the lipoprotein lipase-GPIHBP1 complex that mediates plasma triglyceride hydrolysis. Proc. Natl. Acad. Sci. USA 2019 Jan;116(5):1723-1732.Birrane G, Beigneux AP, Dwyer B, Strack-Logue B, Kristensen KK, Francone O, Fong LG, Mertens HD, Pan CQ, Ploug M, Young SG, Meiyappan M.
- Protein engineering on human recombinant follistatin: enhancing pharmacokinetic characteristics for therapeutic application. J Pharmacol Exp Ther 2018 Aug;366(2):291-302.Shen C, Iskenderian A, Lundberg D, He T, Palmieri K, Crooker R, Deng Q, Traylor M, Gu S, Rong H, Ehmann D, Pescatore B, Strack-Logue B, Romashko A, Baviello G, Gill J, Zhang B, Meiyappan M, Pan C, Norton AW.
- Site-Specific Antibody-Drug Conjugation through Glycoengineering.Bioconjug Chem 2014 Mar;25(3):510-520. Zhou Q, Stefano JE, Manning C, Kyazike J, Chen B, Gianolio D, Park A, Busch M, Bird J, Zheng X, Simonds-Mannes H, Kim J, Gregory R, Miller RJ, Brondyk BH, Dhal PK, Pan CQ.
- Carbohydrate-mediated PEGylation of Thyrotropin (TSH) improves its pharmacological properties. Endocrinology 2013 Mar;154(3):1373-1383. Park A, Honey D, Hou L, Bird J, Zarazinski C, Searles M, Braithwaite C, Kingsbury J, Kyazike J, Culm-Merdek K, Greene B Stefano J, Qiu, H, McPherson J, Pan CQ.
- High Affinity VEGF Antagonists by Oligomerization of a Minimal Sequence VEGF-Binding Domain.Bioconjug. Chem. 2012 Dec;23(12):2354-2364. Stefano J, Bird, J, Kyazike J, Cheng, A, Boudanova E, Dwyer M, Hou L, Qiu H, Matthews G, O'Callaghan M, Pan CQ.