Commercializing CRISPR/Cas9 Therapy: FDA Approval of Exa-Cel Marks Milestone in Treatment Development

Pharmacy Practice in Focus: Health SystemsMay 2024
Volume 13
Issue 3

These developments highlight the dynamic landscape of health care innovation and the collaborative efforts driving progress in disease management and treatment.

Gene-editing technology -- Image credit: vchalup |

Image credit: vchalup |

Clustered regularly interspaced short palindromic repeats (CRISPRs)/ CRISPR-associated protein 9 (Cas9) therapy is a revolutionary treatment approach that allows scientists to selectively modify a living organism’s DNA.1 The gene-editing technology involves 2 essential components: a guide RNA to match a target gene and Cas9, which is an endonuclease that causes a double-stranded DNA break that allows modifications to the genome.1,2 With this system, scientists are finding new potential treatments for genetic disorders and diseases.

In the cover feature on page 11, author Sophia Humphreys, PharmD, MHA, BCBBS, discusses 2 gene therapy agents that the FDA recently approved for sickle cell disease: exagamglogene autotemcel (exa-cel; Casgevy; Vertex Pharmaceuticals Incorporated) and lovotibeglogene autotemcel (lovo-cel; Lyfgenia; bluebird bio, Inc). Exa-cel is the first cell-based therapy using CRISPR/Cas9 therapy, marking the beginning of commercializing this gene-editing technique. The approval of exa-cel and lovo-cel also marks a significant advancement in treating sickle cell disease, offering a potential cure beyond bone marrow transplants. However, the wholesale acquisition cost for 1 course of exa-cel is $2.2 million, and lovo-cel’s single course costs $3.1 million.

In the peer-reviewed case description on page 14, Allison Brunson, PharmD, BCPS, and Dawn Waddell, PharmD, BCPS, discuss the implementation and outcomes of a transitions-of-care (TOC) program focused on improving care and reducing 30-day readmissions for patients with chronic obstructive pulmonary disease (COPD). By addressing gaps in medication adherence and discharge planning, the program successfully lowered the 30-day readmission rate for patients with COPD from 25% to an average of 16.2% over fiscal years 2020 to 2023.

In the peer-reviewed literature review on page 18, Azhar Hussain, DHA, MBA, and Syed Ahsan discuss the importance of medication adherence in chronic disease management and the role of artificial intelligence (AI)–integrated reminders in improving patient adherence. The authors highlight various AI-powered reminder systems tailored to individual patient needs, including personalized alerts, chatbot interfaces, and gamified approaches to promote adherence. The authors alsounderscore the potential of AI technology to revolutionize health care outcomes by enhancing patient engagement and motivation, ultimately improving both adherence and treatment effectiveness.

The transformative impact of CRISPR/Cas9 therapy has begun to alter treatment opportunities for patients with sickle cell disease with an option such as exa-cel becoming available. Although exa-cel represents a groundbreaking advancement, its high cost underscores the ongoing challenges in ensuring equitable access to cutting-edge treatments. Moreover, innovative approaches in TOC programs and AI-integrated interventions demonstrate the pivotal role of pharmacists and technology in enhancing patient outcomes and shaping the future of health care. Together, these developments highlight the dynamic landscape of health care innovation and the collaborative efforts driving progress in disease management and treatment.


1. CRISPR. National Human Genome Research Institute. Updated April 19, 2024. Accessed April 22, 2024.,editing%20systems%20found%20in%20bacteria
2. Redman M, King A, Watson C, King D. What is CRISPR/Cas9? Arch Dis Child Educ Pract Ed. 2016;101(4):213-215. doi:10.1136/archdischild-2016-310459
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