CRISPR Therapeutics Highlights Strategic Priorities and 2024 Outlook - Seite 2

Completed enrollment in two global Phase 3 studies of CASGEVY in patients 5 to 11 years of age with SCD or TDT.

Vertex is engaging with experienced hospitals to establish a network of authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to patients. Nine ATCs have been activated in the U.S. and three in Europe, with the goal of activating approximately 50 ATCs in the U.S. and 25 in the EU. Additionally, Vertex announced an agreement with a major medication contracting organization, Synergie Medication Collective, which covers nearly 100 million lives in the U.S., to provide access to CASGEVY through an outcomes-based contract.

Hemoglobinopathies

• CRISPR Therapeutics has two next-generation research focuses that each have the potential to expand the addressable population with SCD and TDT significantly. First, the Company continues to advance its internal targeted conditioning program, an anti-CD117 (c-Kit) antibody-drug conjugate (ADC), through preclinical studies.

• Second, the Company has ongoing research efforts to enable in vivo editing of hematopoietic stem cells. This work, supported in part by a $14.5 million grant from the Bill & Melinda Gates Foundation, could obviate the need for conditioning altogether, expand geographic reach, and enable the treatment of multiple additional other diseases beyond SCD and TDT.
  

Immuno-Oncology and Autoimmune Disease

• CRISPR Therapeutics’ next-generation allogeneic CAR T candidates reflect the Company’s mission of innovating continuously to bring potentially transformative medicines to patients as quickly as possible. Clinical trials are ongoing for CRISPR Therapeutics’ next-generation CAR T product candidates, CTX112 and CTX131, targeting CD19 and CD70, respectively. Emerging pharmacology data, including pharmacokinetics, indicate that the novel potency gene edits in CTX112 and CTX131 can lead to significantly higher CAR T cell expansion and functional persistence in patients compared to the first-generation candidates. Focusing efforts on these candidates will enable the Company to advance these potentially best-in-class CAR T therapies more efficiently and rapidly. The Company expects to provide a clinical update in 2024 for these next-generation candidates.