The latest clinical advances in CRISPR/Cas9 gene editing therapy

On December 5, CRISPR and Vertex jointly announced the latest data for CRISPR/Cas9 Gene Editing Therapy (CTX001) at the ASH conference, showing a consistent and consistent response from 10 patients treated with CTX001.
7 patients with blood transfusion dependence β TDT), including 3 patients with the b0/b0 genotype, were treated with CTX001 and did not rely on blood transfusions during the most recent follow-up.
3 cases of sickle cell disease (SCD) patients were treated with CTX001 and none of them had vascular ococcysts (VOCs).
CTX001 is currently in phase I/II clinical studies and is expected to be a potential treatment for TDT and severe SCD.
CTX001 is a gene editing therapy in the study of self-study CRISPR/Cas9, which aims to increase the level of fetal hemoglobin (HbF) in red blood cells by editing hematopoietic stem cells in patients with TDT and SCD.
HbF is an oxygen-carrying hemoglobin that is naturally present at birth and then transforms into an adult form of hemoglobin.
based on the current progress of the project, CTX001 treatment of TDT and SCD adaptations has been certified by the FDA Advanced Therapy for Regenerative Medicine (RMAT), Fast Track, Orphan Medicine, and Rare Pediatric Diseases.
13 TDT patients in the ongoing Phase I/II clinical trial, CLIMB-111.
7 of them were followed for at least 3 months after CTX001 treatment, and efficacy analysis could be carried out.
7 patients were no longer dependent on blood transfusions during the 3 to 18 months of follow-up, and the total hemoglobin value at the last follow-up reached normal or near-normal levels.
total hemoglobin levels were 9.7-14.1 g/dL, of which fetal hemoglobin accounted for 40.9%-97.7%.
safety data of 7 patients were basically consistent with innard stem cell transplantation and myelin.
1 patient reported 4 CTX001 treatment-related serious adverse events (SAEs), including headache, haemophageal syndrome (HLH), acute respiratory distress syndrome, and idiopathic pneumonia syndrome, most of which were mild and moderate.
6 SCD patients were included in the ongoing Phase I/II clinical trial, CLIMB-121.
3 of them were followed for at least 3 months after CTX001 treatment.
3 to 15 months of follow-up, 3 patients did not show the risk of vascular closure, the last follow-up of hemoglobin levels reached normal or close to the normal range.
total hemoglobin level was 11.5-13.2 g/dL, of which fetal hemoglobin accounted for 31.3% to 48.0%.
data from 3 patients were basically consistent with autobiographic stem cell transplantation and myelin.
no CTX001 treatment associated with SAEs, most adverse reactions are mild and moderate.
CRISPR-Cas9 gene editing technology is through the artificial design of sgRNA (guide RNA) to identify the target genome sequence, and guide Cas9 protease to effectively cut THE DNA double strands, forming a double-stranded fracture, post-injury repair will cause gene knock-out or knock-in, and so on, and ultimately achieve the purpose of genome DNA modification.
Emmanuelle Charpentier of the Max Planck Institute for Pathogens in Germany and Jennifer A. Doudna of the University of California, Berkeley, were awarded the 2020 Nobel Prize in Chemistry for their technology.
Kewalramani, CEO of
Vertex, said: 'This is the first published result of CRISPR/Cas9 therapy in the treatment of people with genetic disorders, an important milestone in medicine and an important milestone in our collaboration with CRISPR.
through clinical proof-of-concept and 19 patient dose studies in TDT and SCD patients, we will continue to work hard to bring our therapies to TDT and SCD patients as soon as possible.
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