Consensus-driven target product profiles for curative sickle cell disease gene therapies

Area of Science:

• Hematology
• Gene Therapy
• Drug Development

Background:

• Sickle cell disease (SCD) treatment innovation is advancing, with gene therapy offering potential cures.
• SCD disproportionately affects populations globally, necessitating globally relevant therapeutic strategies.
• Target product profiles (TPPs) are crucial for guiding the development of new medicines.

Purpose of the Study:

• To establish consensus-driven TPPs for ex vivo and in vivo gene therapies for SCD.
• To define minimal and optimal criteria for novel SCD gene therapy products.
• To ensure new therapies meet the diverse needs of individuals with SCD worldwide.

Main Methods:

• A multidisciplinary expert group was convened.
• A modified Delphi methodology was employed.
• Virtual workshops supplemented the Delphi process.

Main Results:

• Developed TPPs with 20 minimal and optimal criteria for SCD gene therapies.
• Criteria categorized into scope, performance/safety, manufacturing, and administration.
• Ex vivo and in vivo gene therapy TPPs showed differences in manufacturing and administration criteria.

Conclusions:

• The generated TPPs provide a framework for developing curative SCD gene therapies.
• These TPPs aim to facilitate the creation of safe, effective, and practical treatments.
• The criteria support the development of durable therapies for global SCD populations.