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Spark Therapeutics, Inc.

https://sparktx.com/

Latest From Spark Therapeutics, Inc.

Pfizer’s Gene Therapy Asset Chases UniQure/CSL’s Hemgenix After Pivotal Hemophilia B Success

Pfizer’s fidanacogene elaparvovec has hit the mark in a Phase III hemophilia B trial just a month after rival UniQure’s Hemgenix won US approval, with Pfizer’s plans for regulatory action raising the stakes for Hemgenix.

Clinical Trials Blood & Coagulation Disorders

Value-Based Agreements On The Cards For UniQure/CSL’s $3.5m Hemophilia B Gene Therapy

While Hemgenix has courted criticism since its $3.5m price tag was unveiled, a drug pricing expert has told Scrip discounts and value-based agreements could make the therapy more palatable to payers.

Pricing Strategies Blood & Coagulation Disorders

Vision Possible: Why Eyestem Is 'Bullish' About Its Dry AMD Candidate

Eyestem founder and CEO talks to Scrip about progress made by the firm’s experimental treatment for dry age-related macular degeneration, cell and gene therapy pricing considerations and the wider competitive landscape. The company is also developing treatments for retinitis pigmentosa and idiopathic pulmonary fibrosis.

Research & Development Regenerative Medicine

Round Two: BioMarin Resubmits Hemophilia A Gene Therapy Roctavian In US

BioMarin maintains its lead in the race to win approval for a hemophilia A gene therapy in the US by resubmitting Roctavian two years after the FDA issued a CRL in response to the original BLA.

Gene Therapy Strategy
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Company Information

  • Industry
  • Pharmaceuticals
    • Drug Delivery
    • Drug Delivery
      • Macromolecule
      • Site Specific
  • Biotechnology
    • Gene Therapy, Cell Therapy
  • Other Names / Subsidiaries
    • Genable Technologies Limited
    • Roche Holding AG (RHHBY)
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