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Why Tessera Believes Its Gene Writing Platform Can Go Way Beyond CRISPR

As the first CRISPR-based therapies draw closer to approval, the Flagship-backed biotech believes its gene writing tech is the ‘definitive’ genetic medicine platform.

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Vertex/CRISPR Prepare To Take Exa-Cel To Regulators For SCD, TDT

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The Real World Is Not Enough: Comparability Is Common Obstacle For RWE In US FDA Decisions

Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.

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Commercializing Rare Disease Therapies Is Getting Tougher

For an industry increasingly dominated by these drugs, commercial programs need to take on the risk once reserved for development.

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Company Information

  • Industry
  • Pharmaceuticals
    • Drug Delivery
      • Macromolecule
  • Biotechnology
    • Gene Therapy, Cell Therapy
    • Large Molecule
  • Other Names / Subsidiaries
    • Genetix Pharmaceuticals
    • Precision Genome Engineering, Inc. (Pregenen)
    • Oncology Newco
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