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FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD

CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX-001, an investigational, autologous, Gene Editing hematopoietic stem cell therapy, for the treatment of severe Transfusion-Dependent β-Thalassaemia (TDT) and Sickle Cell Disease (SCD).

As with the FDA’s breakthrough therapy designation, cell or gene therapies that receive RMAT designation may benefit from early interactions with the FDA and be eligible for priority review and accelerated approval.

Preliminary data from the Phase 1/2 CLIMB-SCD-121 trial, assessing the safety and effectiveness of a single dose of CTX001 in patients with severe SCD, has shown that the therapy safely increased the levels of fetal haemoglobin in the first patient dosed, effectively preventing the occurrence of Vaso-Occlusive Crises (VOCs).

Positive early findings have been reported for the first TDT patient receiving CTX001 in the Phase 1/2 CLIMB-Thal-111 trial, for which patients are still being recruited.

“RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe haemoglobinopathies, Samarth Kulkarni, PhD, CEO of CRISPR Therapeutics, said in a press release.

“We expect to share additional clinical data on CTX-001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX-001,” he added.

Read the Full Press Release HERE.

Read More Information HERE.


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