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What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers

FDA Law Blog

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials.

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Blood Donor Day 2023: Experts weigh inĀ 

Drug Discovery World

One example we are following with anticipation are the two FDA approvals for the first gene therapies for sickle cell disease. Besides looking at donor characteristics, our platform, which we call Excellos 360TM, includes the assessment of metabolic and effector potential of cells, and genetic analysis.