Vertex and CRISPR Therapeutics Announce MHRA Marketing Authorisation Application Validation for CRISPR/Cas9 GeneEdited Therapy, exagamglogene autotemcel (exa-cel

pharmafile | January 30, 2023 | News story | Business Services  

London, UK and ZUG, Switzerland, January 27, 2023 – Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics (NASDAQ: CRSP) today announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has validated the Marketing Authorisation Application (MAA) of exa-cel for the treatment of sickle cell disease (SCD) and transfusiondependent beta thalassaemia (TDT) in Great Britain. The submission is supported by two global Phase 3 studies investigating exa-cel as a potential one-time therapy for people with SCD or TDT.

“Today marks a significant milestone in our efforts to bring a new one-time therapy to people living with sickle cell disease or transfusion-dependent beta thalassaemia,” said Nia Tatsis Ph.D., Executive Vice President, Chief Regulatory and Quality Officer. “We look forward to working with the MHRA on our application, the first for a CRISPR-based therapy for a genetic disease.”

Vertex has requested Orphan Drug Designation as part of the MAA.

About exagamglogene autotemcel (exa-cel)

Exa-cel, formerly known as CTX001™, is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients with SCD or TDT, in which a patient’s own hematopoietic stem cells are edited to produce high levels of foetal haemoglobin (HbF; haemoglobin F) in red blood cells. HbF is the form of the oxygencarrying haemoglobin that is naturally present during foetal development, which then switches to the adult form of haemoglobin after birth. The elevation of HbF by exa-cel has the potential to reduce or eliminate painful and debilitating vaso-occlusive crises (VOCs) for patients with SCD and alleviate transfusion requirements for patients with TDT. Earlier results from these ongoing trials were published in The New England Journal of Medicine in January of 2021 and presented at the European Hematology Association Congress in June 2022.

About CLIMB-111 and CLIMB-121

The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 12 to 35 years with TDT or with SCD, respectively. The trials are now closed for enrollment.

About Sickle Cell Disease

Sickle cell disease (SCD) is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” blood cells. People with SCD experience several complications in addition to pain crises, including strokes and anaemia. People with SCD often have spleen damage, which puts them at risk for bacterial infections. Most often, treatment is focused on relieving pain and minimising organ damage, requiring medication and sometimes monthly blood transfusions and frequent hospital visits. The only cure for SCD today are stem cell transplants, but these options are only available to a small fraction of people living with SCD. SCD requires a lifetime of treatment and can result in a reduced life expectancy.

About Transfusion-Dependent Beta Thalassaemia

Transfusion-dependent beta thalassaemia (TDT) is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. A lack of red blood cells, known as anemia, is the primary manifestation of TDT. Because of this anemia, people living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart complications; misshapen bones; and delayed puberty. People with TDT need regular blood transfusions to deliver healthy donated blood to their body. This requires many hospital visits and can also lead to an unhealthy buildup of iron. The amount and frequency of blood transfusions is personalised and depends on the severity of disease each person experiences. The only cure for TDT today are stem cell transplants, but these options are only available to a small fraction of people living with TDT. TDT requires a lifetime of treatment and can result in a reduced life expectancy.

About the Vertex and CRISPR Collaboration

Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exa-cel represents the first potential treatment to emerge from the joint research programme. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialisation of exa-cel and splits programme costs and profits worldwide 60/40 with CRISPR Therapeutics.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, lifethreatening genetic disease — and has several ongoing clinical and research programmes in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassaemia, APOL1-mediated kidney disease, pain, type 1 diabetes and alpha-1 antitrypsin deficiency.

Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters is now located in Boston’s Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognised as one of the industry’s top places to work, including 13 consecutive years on Science magazine’s Top Employers list and one of Fortune’s Best Workplaces in Biotechnology and Pharmaceuticals and Best Workplaces for Women.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. For more information, please visit

CRISPR THERAPEUTICS® word mark and design logo and CTX001™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners

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