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Vertex announces first CRISPR gene-editing therapy

Published on 23 November 2023

Milton Park occupier Vertex has announced the world’s first regulatory authorisation of a CRISPR-based gene-editing therapy.

Vertex Pharmaceuticals and CRISPR Therapeutics announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorisation for CASGEVY™. A CRISPR/Cas9 gene-edited therapy, it’s designed for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

CASGEVY has been authorised 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. There are an estimated 2,000 patients eligible for CASGEVY in the UK.

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

“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 Chief Executive Officer 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. Once achieved, these benefits are potentially expected to be life-long. 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.

“This authorisation 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.

In the UK, exa-cel was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA, and Vertex is already working closely with national health authorities to secure access for eligible patients as quickly as possible.

Vertex has also just announced that the European Medicines Agency (EMA) has validated a Type II variation application to the Marketing Authorisation for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor. If approved, c. 2,800 people with cystic fibrosis in the European Union ages 2 and above could receive a medicine that treats the underlying cause of their disease for the first time.

Vertex Pharmaceuticals

Find out more about Vertex in our company spotlight series.

Company Spotlight: Vertex Pharmaceuticals

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