Optimal design and execution of studies in the area of orphan diseases and ATMPs require both cross-functional approach and in-depth scientific expertise.

The services that tranScrip provides include, but are not limited to:

  • Design of clinical development programmes, from preclinical through to proof of concept and pivotal studies
  • Review and interpretation of study results
  • Review of GMP data for assessment of stability of products within the clinical setting
  • Acting as member of independent safety boards and ethical review boards
  • Preparation for Pre-IND meetings and FDA, EMA and national scientific advice meetings
  • Preparation of Orphan Drug designation applications
  • Biomarker strategies

Case Study

This case study outlines the role which tranScrip has played in development of a product for an orphan disease/ATMP.


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.