The recent approval of Casgevy represents the first official success of gene editing-based therapies. The treatment for sickle cell anemia and thalassemia came in record time, only 11 years after CRISPR was invented. “Two diseases down, 5,000 to go,” commented Fyodor Urnov, Director of Technology & Translation at the Innovative Genomics Institute. Among the many diseases awaiting a cure, what will be the next to benefit from CRISPR? At what rate can we expect new treatments to arrive? The periodic update published by IGI is a must-read to navigate through hope and hype, papers and press-releases. The picture is overwhelmingly positive, but there is also some cause for disappointment. Here is an excerpt from the introduction:

“The enthusiasm in the field still has to face some significant real-world hurdles. Overall, the CRISPR medicine landscape has shifted significantly since our last update and Spring 2023. Market forces have reduced venture capital investment in biotechnology. Investors want to see returns, which means companies are hyper-focused on clinical trials and getting new products to market as quickly as possible to return on investment. Combined with the high price of clinical trials, financial pressure has led to layoffs in a number of CRISPR-focused companies and a narrowing of focus on their furthest developed products, rather than developing their pipeline of new treatments and expanding disease areas. This year, we see a trial in only one new disease area: autoimmunity.”