The pharmaceutical industry has experienced a notable uptick in licensing agreements for innovator drugs incorporating clustered regularly interspaced short palindromic repeats (CRISPR)-based technology over the past five years. These agreements, primarily concentrated in oncology, immunology, and central nervous system therapeutics, have collectively accumulated an impressive $21 billion in deal value. Furthermore, the period from 2020 to 2022 witnessed a remarkable surge in deal worth, particularly in the domain of hematological disorders, reaching a substantial total deal value of $1.8 billion, reveals GlobalData, a leading data and analytics company.

Ophelia Chan, Business Fundamentals Analyst at GlobalData, comments: “This underscores the increasing significance of CRISPR advancements in the development of therapies for hematological disorders.”

The FDA’s approval of Casgevy in December 2023 marked a significant breakthrough in gene therapy. Developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy is the first CRISPR and CRISPR-associated protein 9 (Cas9) genome editing therapy for sickle cell disease and beta thalassemia. This therapy precisely edits DNA in blood stem cells, by taking the patient’s bone marrow stem cells and enhancing the expression of fetal hemoglobin to restore healthy hemoglobin production when reintroducing these edited stem cells back into the patient, thus alleviating symptoms in patients.

Chan adds: “Innovator drugs harnessing CRISPR technologies saw 182% growth in total licensing agreement deal value from $5.6 billion in 2020 to $15.8 billion in 2022. Among the top three therapy areas, oncology represented over half of the total deal value with $11.9 billion, followed by immunology with $6.7 billion, and central nervous system with $2.2 billion.”

Lily and Sanofi have recently collaborated with CRISPR-based technology companies. Last year, Lily’s subsidiary, Prevail Therapeutics secured exclusive rights to Scribe Therapeutics’s CRISPR X-Editing (XE) technologies in a deal potentially worth more than $1.57 billion. This agreement, aimed at advancing in vivo therapies for specific targets known to cause serious neurological and neuromuscular diseases, marked the largest CRISPR-based deal of the year.

Sanofi also expanded its collaboration with Scribe in July 2023, with a deal worth up to $1.24 billion, focusing on utilizing Scribe’s XE genome editing technologies to develop in vivo therapies, particularly for sickle cell disease and other genomic disorders.

Furthermore, Lily’s expertise in cardiometabolic diseases led to a collaboration with Beam Therapeutics in October last year. This $600 million agreement included acquiring Beam’s rights in Verve Therapeutics, a gene-editing company focusing on single-course therapies for cardiovascular disease. This includes Verve’s programs targeting PCSK9 and ANGPTL3, both set for clinical initiation this year.

Chan concludes: “CRISPR technology is transforming targeted gene therapies for diverse unmet diseases by precisely targeting diverse genomic sites, promising tailored treatments and improved patient outcomes. The increasing presence of CRISPR-based therapies in clinical trials is anticipated to fuel further advancements in precision medicine.”

Note: Includes all announced and completed deals for target companies headquartered globally from 2019-24 year to date. Includes deals where at least one drug involved is an innovator for Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system across Marketed, Pre-registration, Phase III, Phase II, Phase I, Preclinical, and Discovery development stages. Displays top 6 therapy areas. A deal may have multiple drugs that may be present across multiple therapy areas depending on the drugs involved in the deal.