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.

Discovery & Preclinical

Set a clear product strategy, define a credible clinical and regulatory path, select the right asset and get ready for first-in-human trials with confidence

Early Clinical​

Implement first-in-human trials, interpreting emerging data and adapting plans as needed. Ensure robust decisions, including clear proof-of-concept

Late Clinical​

Design medically-endorsed pivotal programs that stand up to global regulatory and commercial scrutiny. Navigate issues in implementation​

Registration & Approval

Interpret data, assess benefit/risk and secure regulatory approvals, successfully bring your medicine to market. Ensure strong lifecycle management​

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

Get in touch to see how our senior cross-functional experts can help you reduce risk, protect value, and move forward with confidence.