First CRISPR/Cas9 Gene-Edited Therapy Authorized by the UK MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

“This authorization offers a new option for eligible patients who are waiting for innovative therapies, and I look forward to patients having access to this therapy as quickly as possible,” said Professor Josu de la Fuente, Principal Investigator in the CLIMB-111 and CLIMB-121 studies, Professor of Practice (Cellular & Gene Therapy) at Imperial College London, and Consultant Haematologist at Imperial College Healthcare NHS Trust.

CASGEVY™, a CRISPR/Cas9 gene-edited therapy, is indicated for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The therapy has been authorized for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available.

“Today is a historic day in science and medicine: this authorization of CASGEVY in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world,” expressed Reshma Kewalramani, M.D., CEO and President of Vertex.

There are an estimated 2,000 patients eligible for CASGEVY in the U.K.

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“I hope this represents the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases,” said Samarth Kulkarni, Ph.D., Chairman and CEO of CRISPR Therapeutics.”

In two global clinical trials of CASGEVY in SCD and TDT, the trials met their respective primary outcome of becoming free from severe VOCs or transfusion independent for at least 12 consecutive months. These benefits are potentially expected to be life-long once achieved. The safety profile of 97 SCD and TDT patients treated to date with CASGEVY in these ongoing studies is generally consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.

In the U.K., exa-cel was granted an Innovation Passport under the Innovative Licensing and Access Pathway from the MHRA. Additionally, Vertex is presently working closely with national health authorities to secure access for eligible patients as efficiently as possible.

Source: Vertex Therapeutics Press Release

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