The CRISPR RevolutionCRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a recently discovered tool for genetic editing. It allows for very precise and directed gene editing, and its discoverers have won the 2020 Nobel Prize.The initial CRISPR system discovered was CRISPR-Cas9, and many modified CRISPR systems have been discovered or created since. You can read more about the technical details of CRISPR in our article “What Is CRISPR-Cas12a2? Why Does It Matter?”CRISPR is at the forefront of the genomic revolution, with the first gene therapies using it now getting approved for blood diseases, something we explored in depth in “How CRISPR Companies Target Sickle Cell Anemia”.Almost every month that passes seems to bring a brand new revolutionary CRISPR therapy or application. The latest in date is curing a rare form of blindness.Curing Blindness With CRISPR?In a scientific paper titled “Gene Editing for CEP290-Associated Retinal Degeneration”, researchers and doctors at the Universities of Harvard Medical School, Pennsylvania, Michigan, Miami, Oregon Health and Science, as well as Perelman School of Medicine and Editas Medicine have seen remarkable results in treating a disease called Leber Congenital Amaurosis (LCA)LCA causes the degradation of the vision in the first early months of life. There is currently no treatment for LCA, and affects an estimated 50,000 people in the USA and 180,000 people worldwide.After a break in 2022,  Editas Medicine announced in May 2024 that the clinical trial for EDIT-101 has seen 79% of the 14 clinical trial participants experience measurable improvement after receiving the experimental gene therapy.“One of our trial participants has shared several examples, including being able to find their phone after misplacing it and knowing that their coffee machine is working by seeing its small lights.While these types of tasks might seem trivial to those who are normally sighted, such improvements can have a huge impact on quality of life for those with low vision.” – Mark Pennesi, M.D., Ph.D. – Oregon Health Science University’s lead scientistNext Steps For EDIT-101Now that the efficiency of the treatment is proven, and there were no serious side effects, the next step is determining the “ ideal dosing, whether a treatment effect is more pronounced in certain age groups such as younger patients, and include refined endpoints to measure impacts on activities of daily living.”“Our hope is that the study will pave the road for treatments of younger children with similar conditions and further improvements in vision.This trial represents a landmark in the treatment of genetic disease, in specific genetic blindness, by offering important alternative treatment when traditional forms of therapy, such as gene augmentation, are not an option.” – Tomas S. Aleman, M.D

Pictured: Illustration of a gloved hand holding tweezers and editing DNA/iStock, LadadikArtThe era of CRISPR gene-editing technology has arrived. With the approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exa-cel) in two indications, the question now is, what’s next?In short, the answer to that question is not much right now. While the cell and gene therapy sector as a whole is expecting continued growth in terms of approvals, CRISPR-based therapies are predominately early stage.CRISPR Pipeline Skews Toward DiscoveryThe December and January approvals of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia represent a landmark for gene therapy. Casgevy is the first approved treatment that uses CRISPR/Cas9 gene-editing technology. The one-time treatment can be curative for these serious, life-altering blood disorders.But Casgevy will not open any immediate floodgates to CRISPR therapy approvals, as most potential CRISPR-based treatments are still in the earlier stages of development. According to a December 2023 report from GlobalData, discovery and preclinical assets account for 88% of the CRISPR drug candidates currently under investigation, with 24 CRISPR-based therapeutics in Phase II trials and one other than Casgevy in Phase III.Thus, despite the milestone approval of Casgevy, GlobalData analyst Jasper Morley believes the industry is “unlikely to see another [CRISPR] drug approval in the near future.”While hematological drugs are the most advanced, out of 262 active drugs in the discovery and preclinical stage, oncology dominates—accounting for some 25%—Morley told BioSpace in an email