tranScrip’s experts utilise their expertise in therapeutic areas to develop products in the areas of rare diseases and advanced therapeutic medicinal products (ATMPs). tranScrip provides specialist support for strategic, translational, clinical development, regulatory and the complexity of commercial delivery challenges for clients seeking to develop treatments for rare diseases or novel ATMPs.

tranScrip’s Rare Diseases and Advanced Therapeutics team has supported design and delivery of large, complex, cross-functional orphan and ATMP programmes, across a large spectrum of therapy areas and diseases (e.g. haematological and solid malignancies, metabolic disorders, graft vs host disease, etc), including gene therapy (viral and non-viral vectors), cell therapy and tissue engineering (including combined IMP and device technologies). The team has strategic and tactical experience of the complex clinical operational considerations, unique to ATMPs. tranScrip has a deep understanding of the unique ATMP regulatory pathways and strategies to execute expeditious clinical development.


Optimal design and executiona 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.