New Publication: Cell and Gene Therapy for Rare Diseases
The NEED Sub-team’s latest paper has been published in Therapeutic Innovation & Regulatory Science:
Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases, by DahShu Innovative Design Scientific Working Group (IDSWG)
This paper represents the third major publication from the NEED (Nature and Extent of Evidence Needed for Rare Diseases) Sub-team, co-led by Mercedeh Ghadessi (Bayer) and Sammi Tang (Astellas). It follows earlier publications on historical controls (2020) and decentralized clinical trials (2023).
The paper addresses clinical development considerations specific to cell and gene therapy in rare disease settings, covering design approaches, regulatory landscape, and operational challenges.
Read the paper, doi:10.1007/s43441-025-00853-4