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Manufacture Advanced Medicine, Standards & Regulation

The World’s First CRISPR-Based Therapeutic Approval

The FDA set a target action date of December 8 for a decision on whether to approve Vertex and CRISPR Therapeutics’ CRISPR-Cas9 gene edited therapy for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), but the UK has beaten it to the punch.

The UK Medicines and Healthcare products Regulatory Agency has conditionally authorized the therapy. “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,” said Reshma Kewalramani, Chief Executive Officer and President of Vertex, in a statement.

Casgevy (exagamglogene autotemcel [exa-cel]) has been approved for patients aged 12 years and over 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.”

In a clinical trial for SCD, 28 out of 29 patients were free of severe pain crises for at least 12 months after treatment. For TDT, 39 out of 42 patients did not need a red blood cell transfusion for at least 12 months following treatment, with the other patients seeing a reduction of more than 70 percent in the need for red cell transfusions. The MHRA says it will monitor the safety of the treatment through real-word safety data and post-authorization safety studies to be conducted by Vertex and CRISPR Therapeutics.

“Both sickle cell disease and β-thalassemia are painful, life-long conditions that in some cases can be fatal. To date, a bone marrow transplant – which must come from a closely matched donor and carries a risk of rejection – has been the only permanent treatment option,” said Julian Beach, Interim Executive Director of Healthcare Quality and Access at the MHRA.

It is thought that around 2000 patients in the UK could benefit from the therapy. However, the treatment will need to be approved by the UK’s drug cost watchdog, NICE, which will evaluate the benefits to patients versus the cost of the treatment to the country’s national health service.

In the US, the FDA recently completed an advisory committee meeting for the therapy, noting that it was highly effective at preventing the pain episodes associated with SCD; many experts predict an approval on December 8. The therapy is also under review by the EMA.

“The MHRA’s authorisation of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ gene editing therapy, Casgevy (exagamglogene autotemcel), is a seminal moment in the history of biotechnology. This makes it the first approval globally of a gene-editing medicine,” said Tim Hunt, CEO of the Alliance for Regenerative Medicine, in an emailed statement. “The therapy is transformative for patients with sickle cell disease and transfusion-dependent beta-thalassaemia, and is positioned to reduce the ongoing burden of chronic care for these diseases in the UK. This is a remarkable step forward for Advanced Therapy Medicinal Products and for those living with two rare, serious genetic diseases.”

You can read more CRISPR coverage in the following articles:

Manufacture Advanced Medicine

Manipulating the Fabric of Life

| 20 min read

A selection of experts using genome editing and CRISPR/Cas9 for drug development purposes offer their views on the field.

Discovery & Development Drug Delivery

Creating CRISPR Chickens

| Jamie Irvine | 4 min read

Researchers genetically modify chickens to build bird-flu resistance – read more here.

Discovery & Development Drug Discovery

CRISPR With Caution

| Rob Coker | 3 min read

Deputy editor Rob Coker recalls his trip to the labs of Caszyme, Vilnius, where he spoke with CEO Monika Paule about the potential of genome editing

Discovery & Development Drug Discovery

Enhancing CRISPR-Cas9

| Stephanie Vine | 3 min read

Is safeguard sgRNA the key to reducing the off-target effects of gene editing, while increasing its potential applications?

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Stephanie Vine

Making great scientific magazines isn’t just about delivering knowledge and high quality content; it’s also about packaging these in the right words to ensure that someone is truly inspired by a topic. My passion is ensuring that our authors’ expertise is presented as a seamless and enjoyable reading experience, whether in print, in digital or on social media. I’ve spent fourteen years writing and editing features for scientific and manufacturing publications, and in making this content engaging and accessible without sacrificing its scientific integrity. There is nothing better than a magazine with great content that feels great to read.

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