Imperial College London has recently unveiled an innovative gene therapy known as Casgevy, offering promising prospects as a potential treatment for two inherited blood disorders.
Casgevy, the novel therapy, harnesses the power of CRISPR gene editing. This groundbreaking treatment is eligible for patients aged 12 and above following meticulous evaluations ensuring its safety, quality, and effectiveness.
Casgevy functions by precisely editing the flawed gene within a patient's bone marrow stem cells, enabling the production of functional haemoglobin in the body. The treatment process involves extracting stem cells from the patient's blood, a procedure that accesses bone marrow-derived cells for the subsequent gene editing process.
In a laboratory setting, the CRISPR gene editing technology is employed as a precise set of molecular scissors targets and cuts a specific segment of DNA, effectively deactivating the faulty gene. Subsequently, the edited cells undergo infusion back into the patient's body, facilitating the production of functional haemoglobin.
Casgevy (exagamglogene autotemcel) stands as a revolutionary treatment for sickle-cell disease and transfusion-dependant beta thalassaemia. This groundbreaking therapy relies on the precise gene-editing capabilities of CRISPR technology.
According to regulatory bodies, the clinical evidence supports the efficacy of the new treatment in restoring normal haemoglobin production among patients and alleviating the associated disease symptoms.
These inherited blood disorders profoundly affect the lives of many within our local community. This authorisation presents a fresh opportunity for eligible patients eagerly awaiting innovative therapies, potentially providing a new avenue of hope and care.
The primary permanent treatment for these blood conditions involves a bone marrow transplant from a closely matched donor, but this approach has limitations. Finding a compatible donor can be difficult, and even when a match is found, there are risks of rejection and complications associated with the transplant process. Additionally, this option is accessible to only a fraction of individuals affected by these conditions due to the challenge of finding suitable donors.
Casgevy offers as a new treatment for sickle cell disease and beta thalassaemia.
