Hemophilia

FDA Rejects Biomarin’s Application for its Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

SAN RAFAEL, Calif., Aug. 19, 2020 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A on August 18, 2020.  The FDA issues a …

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CSL Behring to Pay $450 Million to Aquire uniQure’s Late-Stage Hemophilia B Gene Therapy

KING OF PRUSSIA, Pa., – 24 June 2020 – Global biotherapeutics leader CSL Behring announced today that it has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of hemophilia B from uniQure (NASDAQ: QURE), a leading gene therapy company. The AMT-061 …

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Regeneron and Intellia Therapeutics Expand Collaboration to Develop CRISPR/Cas9-Based Treatments for Hemophilia

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Intellia Therapeutics, Inc. (NASDAQ: NTLA) announced an expansion of their existing collaboration to provide Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic targets and for the companies to jointly develop potential products for the treatment of hemophilia A and B. Regeneron also receives non-exclusive rights to independently develop and …

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HEMA Biologics Announces FDA Approval of SEVENFACT for Treatment of Bleeding Episodes in Hemophilia A and B Patients with Inhibitors

HEMA Biologics, LLC, (“HEMA Biologics”) today announced that the U.S. Food and Drug Administration (FDA) approved SEVENFACT® [coagulation factor VIIa (recombinant)-jncw] as the first new bypassing agent in over 20 years for adults and adolescents (12 years of age and older) with hemophilia A and B with inhibitors. “Through a joint …

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FDA Accepts and Grants Priority Review to Biomarin’s BLA for Valoctocogene Roxaparvovec for Patients with Hemophilia A

SAN RAFAEL, Calif., Feb. 20, 2020 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.  This …

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Light Chain Bioscience Achieves Milestone Payment for Successfully Completing Factor VIII Mimetic Bispecific Antibody Discovery for Takeda

Light Chain Bioscience, a company developing bispecific antibodies with a pipeline focused in immuno-oncology, announced that it has achieved a milestone and received a payment under its research and collaboration agreement with Takeda Pharmaceutical Company Limited (Takeda). The milestone was achieved upon the successful completion of the discovery phase and …

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Biomarin Submits Valoctocogene Roxaparvovec BLA to the FDA for Treatment of Hemophilia A

SAN RAFAEL, Calif., Dec. 23, 2019 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.  Subject to completion of the …

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Bayer Collaborates with Children’s Hospital of Philadelphia to Research Novel Small Molecule Therapies for Oral Treatment of Hemophilia

WHIPPANY, N.J., Dec. 5, 2019 /PRNewswire/ — Bayer announced today that it has entered into a three-year collaboration agreement with Children’s Hospital of Philadelphia (CHOP) for the discovery and development of small molecules (SMOLs) to develop a first-in-class oral non-replacement therapy (NRT) for the treatment of hemophilia A and B. …

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Takeda and Enzyre to Develop Diagnostic Device Allowing Hemophilia Patients to Determine their Coagulation Status at Home

NIJMEGEN, Netherlands, Dec. 5, 2019 /PRNewswire/ — Enzyre, which is developing breakthrough ambulant diagnostic technology for coagulation testing, today announced that it has entered into a research collaboration agreement with Takeda Pharmaceutical Company Limited (Takeda) to develop a diagnostic device that will enable hemophilia patients to determine their coagulation status …

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bluebird bio and Novo Nordisk Partner to Develop in vivo Genome Editing Candidates for Hemophilia and Other Severe Genetic Diseases

CAMBRIDGE, Mass. and BAGSVÆRD, Denmark–(BUSINESS WIRE)–bluebird bio, Inc. (Nasdaq: BLUE) and Novo Nordisk A/S (NVO) today announced that they have entered into a research collaboration to jointly develop next-generation in vivo genome editing treatments for genetic diseases, including hemophilia. During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development …

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