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Rare Diseases and Advanced Therapeutics

30 Mar 23


A small company with novel an X ATMP for Graft vs Host Disease (GvHD) (cell therapy for the treatment and prevention of GvHD).


tranScrip worked with the company to:

  • Successfully file an Orphan Drug designation
  • Confirm phase 2 design with EMA
  • Identify CRO for clinical work
  • Seek funding


Orphan Drug designation for prevention of GvHD was achieved Agreement with EMA on phase 2 design and major design elements for phase 3 was reached.

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