Rare Diseases and Advanced Therapeutics

The tranScrip orphan drug consulting, rare disease and advanced therapeutics team has helped design and implement complex, cross-functional programmes across a wide spectrum of medical conditions.

Our comprehensive therapeutic expertise is clearly evident in the development of advanced therapeutic medicinal products (ATMPs), as well as products designed to treat issues such as haematological and solid malignancies, and even metabolic disorders.

We provide specialist support for clinical development, all regulatory requirements, and any conceivable commercial delivery challenge that might occur. Whatever happens along the way, we ensure your ideas become reality.


Our team are experts in gene therapy, cell therapy, tissue engineering, investigational medicinal products (IMP) and device technologies.

We also have strategic and tactical experience of the complex operational considerations, unique to ATMPs. Our specialists have a deep understanding of the ATMP regulatory pathways, and we have the decisive mindset required for rapid clinical development.

How can we support you?

We assist with the design of clinical development programmes, covering pre-clinical, proof of concept work, pivotal studies, and any other development needs.

Our team develops biomarker strategies, then reviews and interprets study results to ensure everything is proceeding as planned. Our experts also supply good manufacturing practice (GMP) data to make certain that products remain stable within the clinical setting.

Beyond that, we help prepare orphan drug designation applications, and we’re on hand for all your most pivotal meetings. You’ll have our support in every consultation from pre-investigational new drug (IND), to Food and Drug Administration (FDA) and European Medicines Agency (EMA) applications and anything pertaining to national scientific advice.

We also represent your interests in independent safety boards and ethical review boards.

Capability Overview

09 May 23

Contact our team of experts

We have teams of life science and R&D experts to help achieve your goals and seize opportunities. Whatever your project requires, we have the people to meet your needs.