Opsona Therapeutics Ltd.

Experts Highlight Key Trends and Challenges in Rare Disease Drug Development for 2024

a year ago

• The rare disease therapeutics landscape shows promising momentum in 2024, with three orphan drug approvals already achieved and approximately 50% of approved drugs targeting rare conditions. • The historic FDA approval of CRISPR gene therapy in late 2023 marks a significant milestone, with in-vivo CRISPR therapies and dual AAV gene therapy approaches showing promise for future treatments. • Clinical trial recruitment remains a major challenge for rare disease research due to small patient populations, geographic dispersion, and new FDA diversity requirements, though patient advocacy groups are helping bridge these gaps.