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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Title
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Authors
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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
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K. John Pasi, Savita
Rangarajan, Benjamin Kim, Will Lester, David Perry, Bella Madan,
Fatemeh Tavakkoli, Ke Yang, Glenn F. Pierce and Wing Yen
Wong
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Poster Presentations
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Title
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Authors
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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
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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
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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
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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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Contact:
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Investors:
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Media:
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Traci McCarty
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Debra
Charlesworth
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BioMarin
Pharmaceutical Inc.
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BioMarin
Pharmaceutical Inc.
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(415)
455-7558
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(415)
455-7451
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SOURCE BioMarin Pharmaceutical Inc.