Rare Diseases

Rare Disease development poses unique challenges and opportunities. Our experts develop insightful and innovative strategies to bring these ‘game changing’ products to patients within all therapeutic indications with a particular focus on the unmet needs within rare and orphan diseases.

Therapeutic Experience

Therapeutic Experience

In-depth Expertise

At tranScrip, we are helping our biotech and pharma clients realise the potential of their assets. tranScrip is helping shift the paradigm of therapeutic intervention and steer strategies to maximise efficient, expeditious and safe development.

We provide strategic expertise, therapeutic experience and operational excellence across the entire product lifecycle.

Case Study

Regulatory Success for a US-Based Gene Therapy Company in the EU

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Brochure

Rare Diseases Project Examples

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“I have worked with tranScrip on several occasions, in my previous capacity as Head of Development and Chief Medical Officer at Polyphor AG and in my previous role as Chief Medical Officer at Polyneuron AG.

Services tranScrip have provided in the therapeutic areas of oncology, infectious diseases, neurology and rare diseases have ranged from protocol writing, manuscript writing, drug safety, clinical pharmacology and microbiology. To simply state that they have delivered consistently and to a high standard of quality would badly understate the impact they have had on the performance and ability of the companies to execute and deliver on key investor and regulatory milestones. Strategic oncology input enabled us to design a winning oncology strategy, deliver a publication in a Key Journal in time for IPO and write the protocol for the pivotal study. tranScrip’s staff were able to integrate seamlessly and work (remotely) as a full value adding part of the team. This delivering as part of the team was also seen in the drug safety and clinical pharmacology support where tranScrip employees integrate to the team and add value from their general drug development experience, as well as their specific area of key focus, both of which are appreciated.

As well as proactive ongoing support, tranScrip have also demonstrated their effectiveness on “emergency interventions”, able to pull a team together at short notice for a deep dive into a problem after regulatory advice has been received. The quality of the technical input and contribution to problem solving is impressive, as well as the higher level drug development and safety expertise to participate in the development of overarching and comprehensive effective solutions.”

Debra Barker

Chief Medical Officer

Destiny Pharma

Contact Us

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.