Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) have shattered scientific barriers today as the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) grants conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), marking the first-ever regulatory approval of a CRISPR-based gene-editing therapy globally. CASGEVY is set to transform the treatment landscape for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

CASGEVY Granted Unprecedented Approval for Revolutionary Treatment

This historic authorization opens new horizons in medicine, with CASGEVY being authorized for eligible patients aged 12 and older with SCD experiencing recurrent vaso-occlusive crises (VOCs) or TDT, where a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available. The estimated 2,000 eligible patients in the U.K. now have access to this groundbreaking therapy.

Leadership Perspectives on the Revolutionary Moment

Vertex's Chief Executive Officer and President, Reshma Kewalramani, expressed the historic significance of the day, stating, "Today is a historic day in science and medicine: this authorization of CASGEVY in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world." Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics, echoed this sentiment, envisioning a future where CRISPR technology benefits patients with serious diseases globally.

Life-Changing Results and Global Collaboration

Global clinical trials of CASGEVY in SCD and TDT have demonstrated remarkable outcomes, meeting primary endpoints of freedom from severe VOCs or transfusion independence for at least 12 consecutive months. The safety profile, consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant, offers promise for long-term benefits.

Accelerated Access for Eligible Patients

Professor Josu de la Fuente, Principal Investigator in the CLIMB-111 and CLIMB-121 studies, emphasized the urgency of providing eligible patients with innovative therapies, expressing excitement about the newfound treatment option. In the U.K., exa-cel was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP), and Vertex is collaborating closely with national health authorities to expedite access for eligible patients.

The approval of CASGEVY marks a transformative moment in medical history, ushering in a new era of CRISPR-based gene-editing therapies with the potential to revolutionize the treatment of genetic diseases globally.