Date: 4th March 2020
Article in brief:
The gene-editing field is waiting with hope as the first wave of CRISPR-trials start to release patient data. Now Allergan and Editas Medicine have announced the treatment of the first patient in the BRILLIANCE clinical trial of AGN-151587 (EDIT-101), in a first ever attempt to edit the genome in vivo in humans.
To date all clinical trials involving CRISPRs have performed the gene editing steps in vitro, and then transplanted the edited cells back into the patient.
In September last year we saw an attempt to use CRISPR genome-edited donor cells to treat a HIV patient with blood cancer for the first time.
Then in November, came the release of patient data following clinical trials from CRISPR Therapeutics and Vertex Pharmaceuticals. Similarly, this trial used CRISPR-edited HSPCs, this time for the treatment of transfusion-dependent β thalassemia (TDT), and sickle cell disease (SCD).
The most recent report from last month saw scientists from the University of Pennsylvania and Stanford University School of Medicine, using multiplex CRISPR-Cas9 editing to engineer T cells in three patients with refractory (unresponsive) cancer.
In vivo gene editing
The BRILLIANCE Phase 1/2 clinical trial is designed to assess the safety, tolerability, and efficacy of AGN-151587 in approximately 18 patients with Leber congenital amaurosis 10 (LCA10).
LCA10 is an inherited form of blindness caused by mutations in the centrosomal protein 290 (CEP290) gene. The disorder primarily affects the retina, and typically people affected have severe visual impairment beginning in infancy. It occurs in 2 to 3 per 100,000 of newborns and is one of the most common causes of blindness in children. Currently there are no approved treatment options so this announcement offers hope for thousands of patients.
AGN-151587 is designed to eliminate a common intron 26 mutation in the CEP290 gene that results in retinal degeneration. It is administered directly into the eye via a subretinal injection and delivers the gene editing machinery directly to photoreceptor cells.
This world’s first human study of an in vivo, or inside the body, CRISPR genome editing medicine will no doubt attract much interest over the coming months.
How will this in vivo editing compare to those that occur in a relatively stable, controllable and measurable in vitro environment? With current patient data showing mixed but promising results, will in vivo editing prove safe, and more reliable? We will be waiting with anticipation for the initial patient data to emerge…
For more details please see the press release from Editas
Additional details are available on www.clinicaltrials.gov
