FDA Approval, Genotype, In-Vivo and Trials - Clinical Research Informer

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

XTalks

APRIL 24, 2023

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.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

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

Pharmaceutical Technology

AUGUST 18, 2022

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 The open-label, single-arm, 24-month Phase III trials enrolled 41 subjects of the age four to 34 years with non-?0/?0 0 genotypes.

Gene Therapy

Gene Therapy Gene Genotype Genetics

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

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

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