CASGEVY: A Cure for Sickle Cell & Thalassemia Disorders.
Thornton, Canada, November 17, 2023, Casgevy by the Boston-based pharmaceutical company, Vertex is the first medicine to be licensed that uses the innovative genome-editing tool CRISPR, for which its inventors (Emmanuelle Charpentier of the Max Planck Unit for the Science of Pathogens and Jennifer Doudna of the University of California, Berkeley) were awarded the Nobel Prize in Chemistry in 2020
The inventors first showed that CRISPR—which stands for clustered regularly interspaced short palindromic repeats—could edit DNA in an in vitro system in a paper published in the 28 June 2012 issue of Science. Their discovery was rapidly expanded on by many others and soon made CRISPR a common tool in labs around the world.
Today, Vertex Inc. has revolutionized treatments of hereditary blood disorders by using the CRISPR technology to bring about a cure for sickle-cell disease and transfusion-dependent β-thalassemia; and the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has authorized the use of this treatment for patients aged 12 and over after a rigorous assessment of its safety, quality, and effectiveness.
Sickle Cell Thalassemia Disorders are two hereditary blood disorders that are debilitating, painful, and life-altering. They have resulted in lifelong complications and premature death for many of the affected. Globally, many of the affected people are not identified and those identified experience a life clouded with stigma, discrimination, and poor access to quality care and treatments.
According to the CEO of the Global Action Network for Sickle Cell & Other Inherited Blood Disorders, Lanre Tunji-Ajayi, M.S.M, CASGEVY is a welcome innovation for the hereditary blood disorders community especially those living with sickle cell and the thalassemia disorders. She advised that CASGEVY is a viable cure and that everything should be done to ensure that cost will not prohibit its access to people who need it, especially in low and medium-resource countries.
Lanre Tunji-Ajayi, M.S.M also reminisced in an interview with Canadian Broadcasting Corporation (CBC) that CASGEVY is one of the dreams of her brother, Engr. Sunday Afolabi (whom she lost to preventable complications of sickle cell disease in October 1998) spoke about when he said during his lifetime “ I looked forward to a day when no one will suffer from sickle cell disease”. On this note, she urges Vertex Inc. and other pharmaceuticals working on similar technologies to ensure that the price of this breakthrough therapy is affordable in order for more people to take advantage of it.
Vertex Inc, CEO Reshma Kewalramani advised the Global Action Network for Sickle Cell and Other Hereditary Blood Disorders that “As the first CRISPR/Cas9 gene-edited treatment to be approved, this is a momentous scientific achievement, not only for Vertex but for the patients whose lives we have the potential to transform”
Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning hemoglobin. To do this, stem cells are taken out of bone marrow, edited in a laboratory, and then infused back into the patient after which the results have the potential to be life-long.