CRISPR Therapeutics and Vertex Pharmaceuticals announce FDA RMAT designation granted to CTX001 to treat severe hemoglobinopathies

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“RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies,” said Samarth Kulkarni, Ph.D., Chief

The post CRISPR Therapeutics and Vertex Pharmaceuticals announce FDA RMAT designation granted to CTX001 to treat severe hemoglobinopathies appeared first on Pharmaceutical Business review.

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