BioMarin to Present Innovative Pipeline at R&D Day on September 12th in New York
Live Audio Webcast to Begin at 8:00 a.m. Eastern Time
SAN RAFAEL, Calif., Sept. 11, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, will update the investor community on the company's research and development portfolio, including early-stage product candidates and new indication opportunities with VOXZOGO and ROCTAVIAN. In addition, external clinicians from leading global institutions will participate in panel discussions.
"We look forward to sharing progress across our earlier-stage, next generation product candidates from the most diverse and high-potential pipeline in our company's history," said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin.
Pipeline programs to be presented:
Therapeutic Focus | Candidate | Technology | Condition |
Musculoskeletal/ Metabolic | BMN 255 | Small molecule | Hyperoxaluria in nonalcoholic fatty liver disease |
BMN 351 | Exon 51 oligonucleotide | Duchenne muscular dystrophy | |
Cardiovascular | BMN 293 | AAV gene therapy | Myosin binding protein C3 hypertrophic cardiomyopathy |
BMN 365 | AAV gene therapy | Plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy | |
BMN 355 | Monoclonal antibody | Long QT syndrome types 2 and 3 | |
Other | BMN 349 | Small molecule | Alpha-1 antitrypsin deficiency |
BMN 331 | AAV gene therapy | Hereditary angioedema |
ROCTAVIAN panel discussion on severe hemophilia A and new indications:
Professor Johnny Mahlangu, MBBCh, MMed, FCPath – Director Haemophilia Comprehensive Care, Charlotte Maxeke Johannesburg Academic Hospital, Professor of Haematology, University of the Witwatersrand and National Health Laboratory Service, Johannesburg
Professor Amit Nathwani, MBChB, FRCP, RCPath, Ph.D.- Director of the Katharine Dormandy Hemophilia Centre, Royal Free Hospital, London
Guy Young, M.D. – Director, Hemostasis and Thrombosis Center and Clinical Coagulation Laboratory, Children's Hospital; Professor of Pediatrics, University of Southern California's Keck School of Medicine, Los Angeles
VOXZOGO panel discussion on achondroplasia and new indications:
Andrew Dauber, M.D. - Chief of Endocrinology, Children's National Hospital, Washington, D.C.
Melita Irving, M.B.B.S. - Consultant in Clinical Genetics, Guy's and St. Thomas' NHS Foundation Trust, Evelina Children's Hospital, London, UK
Bradley Miller, M.D., Ph.D. - Director, Division of Endocrinology, M Health Fairview Masonic Children's Hospital; Professor, Department of Pediatrics, University of Minnesota Medical School, Minneapolis, Minnesota
Live audio webcast
An audio webcast of the presentations will be available live. You can access the webcasts at: https://investors.biomarin.com/. An archived version of the remarks will also be available through the Company's website for a limited time following the event.
About BioMarin
Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of the genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit www.biomarin.com.
Contacts: | |
Investors | Media |
Traci McCarty | Andrew Villani |
BioMarin Pharmaceutical Inc. | BioMarin Pharmaceutical Inc. |
(415) 455-7558 | (628) 269-7393 |
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SOURCE BioMarin Pharmaceutical Inc.
