SAN RAFAEL, Calif., Dec. 6, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company will present data in a late breaking abstract session at the 59th American Society of Hematology (ASH) Annual Meeting & Exposition being held December 9-12, 2017 in Atlanta, Georgia. The presentation will report interim results from a Phase 1/2 study of valoctocogene roxaparvovec, an AAV5-FVIII Gene transfer in severe hemophilia.
Listing of Posters and Presentations Related to BioMarin Products and Programs at the 59th American Society of Hematology Annual Meeting & Exposition
Oral Presentation – Late Breaking Abstract Session
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Achievement of Normal Circulating Factor VIII Activity Following BMN 270 AAV5-FVIII Gene Transfer: Interim, Long-Term Efficacy and Safety Results from a Phase 1/2 Study in Patients with Severe Hemophilia A
Presentation: December 11 at 7:30 AM |
K. John Pasi, Savita Rangarajan, Benjamin Kim, Will Lester, David Perry, Bella Madan, Fatemeh Tavakkoli, Ke Yang, Glenn F. Pierce and Wing Yen Wong |
Poster Presentations
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Impact of Pre-Existing Immunogenicity to AAV on Vector Transduction By BMN 270, an AAV5-Based Gene Therapy Treatment for Hemophilia A
Presentation: December 10 at 6:00-8:00 PM Poster/Presentation: #3332 |
Brian Long, Krystal Sandza, Jennifer Holcomb, Juli Pherarolis, Lucy Crockett, Lillian Falese, Greg Hayes, Jeremy Arens, Charles A. O'Neill, Nancy Pryer, Carlos Fonck, Stephen Zoog and Christian Vettermann |
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Interim Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in a Phase-1/2 Clinical Study of BMN 270, an AAV5-Mediated Gene Therapy for Hemophilia A
Presentation: December 11 at 6:00-8:00 PM Poster/Presentation: #4611 |
Brian Long, Benjamin Kim, Wing Yen Wong, Ke Yang, Christian Vettermann, Nancy Pryer, Romain Hardet, Klaudia Kuranda, Philippe Veron, Federico Mingozzi, Glenn F. Pierce and Becky Schweighardt |
About Hemophilia A
Hemophilia A is a genetic disease caused by the deficiency of clotting factor VIII. It is the most common type of hemophilia and occurs much more frequently in males; incidence is estimated to be 1 in 4,000-5,000 male births. People born with hemophilia produce little or no clotting factors. The two main types of hemophilia are A and B. People with hemophilia A are missing or have low levels of clotting factor VIII.
About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com.
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Investors: |
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Traci McCarty |
Debra Charlesworth |
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BioMarin Pharmaceutical Inc. |
BioMarin Pharmaceutical Inc. |
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(415) 455-7558 |
(415) 455-7451 |
SOURCE BioMarin Pharmaceutical Inc.