Generics Bulletin is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction

Spark Therapeutics, Inc.

https://sparktx.com/

Latest From Spark Therapeutics, Inc.

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

Regenxbio: On The Cusp Of Becoming A Commercial Gene Therapy Player

Partnered with AbbVie on a wet AMD gene therapy, Regenxbio is also advancing wholly owned candidates for Duchenne muscular dystrophy and MPS I and MPS II.

Gene Therapy Business Strategies

Re-Thinking Rare Disease

Germany is drastically squeezing orphan drug pricing while new US laws protect rare diseases drugs from price negotiations. These contrasting moves fuel the longstanding debate over whether orphans still deserve special incentives.

Cost Effectiveness Rare Diseases
See All

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)
UsernamePublicRestriction

Register