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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

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FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia 0 genotypes. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.