The business of CRISPR-based gene editing has seen its share of ups and downs. Once the darling of biotech investors, the technology’s initial hype was followed by a period of hard reckoning. Yet, with regulatory approvals rolling in and commercial applications expanding, the gene-editing industry appears poised for a new era of profitability.
The approval of Casgevy, the first CRISPR-based therapy, is a milestone that showcases both the potential and the financial realities of this technology. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy has effectively cured patients with sickle-cell disease and beta-thalassemia—two painful, life-shortening blood disorders. At $2.2 million per patient, the treatment is not cheap, but with global demand and public-payer deals already in place, analysts project multi-billion-dollar revenues.
However, CRISPR’s long-term business viability hinges on more than just rare-disease therapies with steep price tags. The challenge is twofold: scalability and accessibility. Currently, Casgevy requires a complex and costly bone marrow transplant process, limiting its reach. The next phase of CRISPR’s commercialization must involve in vivo (inside-the-body) editing, which could slash treatment costs and open the door to widespread applications.
This shift is already underway. Verve Therapeutics, for example, is developing an in vivo base-editing therapy to lower cholesterol by silencing the PCSK9 gene. The market potential is staggering—cardiovascular disease affects hundreds of millions globally. If successful, this would mark CRISPR’s move beyond rare conditions into common, high-burden diseases, dramatically expanding revenue streams.
Despite its promise, CRISPR still faces regulatory and technical hurdles. Gene-editing firms have had to consolidate, abandoning some early ventures in favor of high-value targets. Additionally, concerns remain about delivery methods—lipid nanoparticles (LNPs), while promising, have encountered safety issues in trials. The industry is racing to refine these technologies, with alternative delivery vehicles, like virus-like particles, gaining traction.
From an investment perspective, the climate is improving. With falling interest rates and the FDA’s move to streamline approvals, capital is flowing back into biotech. Moreover, corporate partnerships—such as UC Berkeley’s non-profit collaboration with Danaher—are emerging to address the unmet needs of patients with ultra-rare genetic diseases.
CRISPR’s potential extends far beyond its Nobel Prize-winning origins. The next five years will determine whether it remains a niche treatment for rare disorders or evolves into a transformative force in mainstream medicine. For businesses in the space, the opportunity is vast—but only for those willing to push the science, and the economics, forward.