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EHA Preview: Agios Takes Spotlight In Thalassemia

The company expects to have data from transfusion-dependent and non-transfusion dependent patients this year, with a potential expanded US approval in 2025 for Pyrukynd.

Companies Clinical Trials

Gene Therapy Q1 Roundup: The Struggle Is Real

Vertex and bluebird compete for sickle cell patients, BioMarin talks about divesting Roctavian, and Sarepta warned that sales of Elevidys will plateau with a narrow label.  

Gene Therapy Launches

Accelerated Approval: Withdrawing Gene Therapies Could Be Easier Than Small Molecules

The FDA would only withdraw a rare disease gene therapy for “pretty clear” reasons, such as when there is minimal benefit with extensive side effects. The one-and-done nature of administration also makes withdrawal easier, CBER Director Peter Marks says.

Gene Therapy Review Pathway

Pfizer Matches Beqvez Price To Hemgenix In Hemophilia B, Plans Warranty Program

The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.

Approvals Gene Therapy
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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