Celebrating a decade of CRISPR gene editing research and development, the Innovative Genomics Institute (IGI) remarks upon just how far this groundbreaking DNA-editing tool has come by presenting some of the latest studies to use it. In addition to closely following the creation of new CRISPR-based therapies, IGI tracked the progress of ongoing clinical trials.
In recent years, news stories related to CRISPR gene editing have been dominated by clinical research into serious blood disorders such as sickle cell anemia and beta thalassemia. Building on modalities that date back to 2019, researchers in Europe and Canada are currently using CRISPR to treat beta thalassemia with remarkable results. With some subjects more than a year into treatment, none require blood transfusions, and all have normal to near-normal hemoglobin levels. In other studies, scientists are using CRISPR-based therapies to target leukemia, lymphoma, and other forms of blood cancer.
Blood diseases and disorders are now just the tip of the iceberg when it comes to clinical CRISPR research. Perhaps most significantly, researchers are now using CRISPR gene editing to address inherited blindness. LCA (Leber Congenital Amaurosis) is the most common cause of genetic childhood blindness, and LCA10 is the most common type of LCA. Currently underway, a new trail promises to address LCA10 by delivering CRISPR-derived treatment to one eye of several patient cohorts, leaving the other eye temporarily untreated to serve as a test control.
Although researchers have yet to share any data about this study, it has already broken new ground by using CRISPR to edit genes within the body of the subject. While previous blood disorder studies have edited genetic material outside of the body (ex vivo editing), the LCA10 study employs in vivo editing for the first time.
Elsewhere (specifically, in Canada), scientists are putting CRISPR gene editing to work in the fight against diabetes. An inherited condition, type 1 diabetes (T1D) occurs when pancreatic beta cells are destroyed by an autoimmune reaction or another form of endocrine disfunction. A new T1D clinical trial, sponsored by CRISPR Therapeutics and ViaCyte, is using CRISPR to edit the specific immune-related genes that cause the disease. Although results are currently uncertain, researchers hope to produce new pancreatic cells that the immune system will not attack.
The vast potential of CRISPR to treat inherited disease has been confirmed by new or ongoing studies into deadly conditions such as hereditary transthyretin amyloidosis (hATTR) and hereditary angioedema (HAE). But 2022 marks a significant milestone in the evolution of CRISPR gene editing to address infectious as well as genetic disease. In fact, CRISPR researchers are addressing two infectious diseases in 2022: chronic urinary tract infections (UTIs) and HIV/AIDS.
Triangle Park, North Carolina’s Locus Biosciences recently completed phase one of their CRISPR-driven UTI clinical study and are moving forward with a phase 2/phase 3 combined trial that began recruiting patients in June of 2022. By integrating phase 2 and phase 3 into a single initiative, the company hopes to put its trial therapeutic on the fast track to approval from the US Food and Drug Administration (FDA). Designed to act upon the genome of the three strains of E. coli that cause roughly 95 percent of UTIs, this therapeutic is a cocktail of three bacteriophages mixed with CRISPR-Cas3.
To support the fight against AIDS, Excision Biotherapeutics has launched the first experimental CRISPR treatment to target the DNA sequence of the HIV (human immunodeficiency virus) stored in the host cell genome of the patient. The company hopes to eliminate HIV from the cell by surgically excising most of the HIV genome. The first trail for this revolutionary treatment approach is currently enrolling patient volunteers.