Rare Diseases and Advanced Therapeutics Featured Image

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.

Contact our team of experts

Our physicians, scientists and service experts can help to maximise the value of your products. Whatever your project requires, we have the people to meet your needs.