Establishing the Scientific Stories of Novel Gene Therapies

Scenario.

• Client had a wide array of innovative gene therapy candidates being explored in the cardiology and neurology spaces, featuring a distinct mechanism of action and utilizing cutting-edge vector technology.
• Two of these candidates were in early phase 1 clinical development for treatment of rare cardiac disorders, with robust pre-clinical data demonstrating promising results in animal models.
• Despite strong data, the team was small, and there was a lack of clear scientific narratives for both of these assets.
• Moreover, the emergence of competitors in these disease areas added pressure to expedite data publication and articulate their unique scientific narrative.

The Ask.

• There was a need for the development of clear and strategic scientific narratives for their two major assets.
• Client was also looking to publish their bolus of pre-clinical data as well as present at a variety of major gene therapy and cardiology conferences. With a goal being to begin laying down the ground-work for their scientific narratives, as well as increase awareness for trial recruitment.

Our Solution and Value Delivered.

Given Client was new to the gene therapy space and was ramping up engagement with key stakeholders, it was critical that there was internal alignment when describing their assets and scientific narratives to external audiences. Additionally, it was crucial to articulate their unique MoA, as well as differentiate themselves within an evolving market. 

1. Scientific narratives & Lexicon – We developed fit-for-purpose scientific narratives that were cross-functionally validated, scientifically robust, high-impact, and aspirational as well as supportive of Client’s clinical and corporate strategies. Additionally, due to internal variability, we identified the opportunity to develop a scientific lexicon, to support more cohesive internal and external communication, and ensure the scientific narratives can be adapted appropriately to resonate with different audiences (HCPs, Investors, Patients / Caregivers). This work is currently ongoing with our team.
2. Scientific Posters / Oral Presentations – We developed scientific posters & oral presentations for several key congresses and meetings. Our team partnered with various external authors as well as Client’s med affairs, clinical development, and pre-clinical development teams to craft over 12 posters & oral presentations. These efforts have not only allowed for effective communication of the data but also supported clinical trial recruitment efforts in these rare therapeutic areas.
3. Publications – In parallel to the congress materials we developed, we also supported Client in the development of two manuscripts – one of which reported the results of in vivo studies evaluating the gene therapy in animals, as well as another examining the comparison of their novel vector technology vs the standard gene therapy. This work is currently ongoing with our team.

Meet The Authors.

Kwamaa Duah 
Principal, Medical Communications