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Rare Diseases and Advanced Therapeutics

30 Mar 23

Challenge

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

Solution

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

Result

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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Our experienced physicians and scientists, together with our regulatory, clinical and commercial experts can support you to maximise the value of your products. Whatever your project requires, we have the expertise to meet your needs.