Cell and gene therapy (CGT) is revolutionizing the treatment of rare diseases by targeting the root causes of conditions rather than merely managing symptoms.

These therapies, including gene augmentation, gene editing, and cell replacement, promise one-time interventions that can deliver long-lasting effects, contrasting sharply with conventional treatments that necessitate ongoing care.

Dr. Kristian Johnson from Bayer Pharmaceuticals noted that CGTs hold the potential to repair single-gene mutations responsible for many rare diseases.

However, patient support programs for CGT must be specifically tailored, as these therapies often involve complex pre-treatment processes that differ from the lifelong treatments patients are used to.

Incorporating patient perspectives early in the CGT development process is crucial for building trust and ensuring that treatments align with patient needs.

Building trust through honest communication with patients, caregivers, and healthcare professionals is essential for the acceptance of CGT, as emphasized by the panelists.

Lynda Chang highlighted the importance of education and transparency to dispel misconceptions about CGT, particularly fears surrounding DNA alterations and unrealistic expectations of cures.

The panel expressed optimism about the future of CGT, recognizing the potential of AI and big data to enhance patient experiences and outcomes.

Despite the promise of CGTs, clinical trial recruitment faces challenges due to small patient populations and the need for comparisons against placebos, which can deter patient participation.

Financial challenges in the CGT field necessitate innovative approaches that leverage existing infrastructures and shared resources across various rare disease communities.