Collaboration with Genomics Medicine Company on X-Linked CGD Gene Therapy 

Our charity partnered with Ensoma, a genomic medicines company advancing the development of a groundbreaking gene therapy for X-linked chronic granulomatous disease (X-CGD). This collaboration underscores our commitment to championing innovative solutions that address the unique challenges faced by individuals living with rare genetic conditions like CGD. 

Our role in the clinical development process 

In alignment with our mission to amplify patient voices and ensure that therapies meet real-world needs, we organized a patient advisory panel to provide balanced feedback on the ongoing clinical trial. The panel consisted of five individuals directly affected by CGD, either as patients or close family members. Their insights were instrumental in shaping a patient-centred approach to this promising therapy. 

The patient advisory panel 

The panel fostered open dialogue between participants and a representative from Ensoma. Key areas of discussion included: 

• Treatment expectations: Understanding what patients hope to achieve from the therapy. 

• Risks and concerns: Identifying potential challenges, from side effects to accessibility. 

• Quality of life factors: Exploring how the therapy might impact daily living, independence, and overall well-being. 

The panel’s feedback was insightful, and deeply valuable. Participants emphasized the importance of transparency about potential risks, the need for robust support systems during the trial, and the desire for clear communication regarding the therapy’s long-term implications. 

Moving forward

This collaboration marks a significant step forward in bridging the gap between cutting-edge science and patient needs. By integrating the perspectives of those most impacted, we are helping to shape a therapy that is not only effective but also aligned with the lived experiences of the CGD community. 

As the trial progresses, we remain dedicated to advocating for patients and ensuring that their voices continue to guide the development and implementation of this therapy. We are optimistic that this initiative will lead to transformative outcomes for individuals with X-CGD and their families. 

Acknowledgments 

We extend our deepest gratitude to the patient advisory panel members for their courage, honesty, and invaluable contributions. Their input has provided a clearer path forward and reinforced the importance of placing patients at the heart of medical innovation. 

Latest news on Ensoma’s lead program for X-Linked CGD 

Ensoma announced U.S. Food and Drug Administration (FDA) clearance of the Investigational New Drug (IND) application for its lead program EN-374 in X-linked CGD.

This is a pivotal moment for gene therapies targeting CGD, and our charity has been actively collaborating with Ensoma to provide valuable patient insights for this trial. We eagerly anticipate continued partnership in the future. 

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