It is a remarkable time for the development of CRISPR-based therapies. In late 2023, we saw the first-ever approval of CRISPR-based medicine: Casgevy, a cure for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The IGI has tracked the progress of CRISPR clinical trials since they first began, and this milestone has been anticipated for some time, but its speed is still noteworthy.
With one CRISPR-based therapy across the finish line, we’re entering a new phase in the clinical development of genome editing in medicine. The enthusiasm in the field still has to face some significant real-world hurdles. Market forces have reduced venture capital investment in biotechnology, leading to layoffs in a number of CRISPR-focused companies and a narrowing of focus on their furthest developed products.
BLOOD DISORDERS Sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) were among the first targets of CRISPR-based treatments. The approval of Casgevy for the treatment of SCD and TDT in patients aged 12 by the UK’s Medicines and Healthcare Products Regulatory Agency and the US Food and Drug Administration (FDA) marks a significant milestone.
CHRONIC BACTERIAL INFECTION A treatment currently in clinical trials uses a cocktail of three bacteriophages combined with CRISPR-Cas3 to attack the genome of the three strains of E. coli responsible for about 95% of UTIs.
PROTEIN-FOLDING DISEASE Hereditary transthyretin amyloidosis (hATTR) is being targeted with CRISPR-Cas9 tools to reduce the amount of faulty TTR protein the body makes.
INFLAMMATORY DISEASE Hereditary angioedema (HAE) is being treated with CRISPR-Cas9 tools to reduce the amount of an inflammatory protein the body makes.
CANCERS CRISPR is being used to enhance CAR-T immunotherapy, with researchers hoping to make it even more powerful by creating allogeneic CAR-T cells from healthy donors.
CARDIOVASCULAR DISEASE Verve Therapeutics began a trial for a subtype of familial hypercholesterolemia using a base editor delivered by lipid nanoparticle (LNP).
HIV/AIDS A US trial using CRISPR to treat HIV by targeting the HIV DNA sequence stored in the host cell genome is underway.
DIABETES A clinical trial using pancreatic cells made from stem cells, edited with CRISPR to avoid immune system rejection, aims to help control or even cure type-1 diabetes without immunosuppressants.
AUTOIMMUNE DISEASE CRISPR Therapeutics is initiating a trial treating patients with systemic lupus erythematosus (SLE) with their next-generation CD19-targeting CAR-T cells.
As the IGI has tracked CRISPR clinical trials each year, we’ve seen both an expansion in number and also in the disease areas being investigated, all on a remarkably short time scale. New approaches to regulating platform technologies could also benefit rare disease patients.