CRISPR Therapeutics Highlights Strategic Priorities and 2024 Outlook - Seite 3

CRISPR Therapeutics plans to initiate a clinical trial of CTX112 in systemic lupus erythematosus (SLE) in the first half of 2024, with the potential to expand into additional autoimmune indications in the future. Early clinical studies have shown that CD19-directed autologous CAR T therapy can produce long-lasting remissions in multiple autoimmune indications.

CRISPR Therapeutics is expanding trials of CTX131 into hematologic malignancies, including T- and B-cell malignancies, in addition to the ongoing clinical trial in solid tumors.

In Vivo

• CRISPR Therapeutics is advancing a pipeline of in vivo gene editing programs using lipid nanoparticle (LNP) delivery of Cas9 mRNA and a guide RNA (gRNA) to the liver. Its first two in vivo programs, CTX310 and CTX320, each aim to reduce expression of a validated target for cardiovascular disease. Beginning with these programs, CRISPR Therapeutics aims to transform the treatment paradigm for cardiovascular indications and beyond with potential one-time therapies that could recapitulate the proven benefit of targets validated by natural human genetics and other therapeutic modalities.
• A Phase 1 clinical trial is ongoing for CTX310, targeting angiopoietin-like 3 protein (ANGPTL3). In humans, naturally occurring loss-of-function variants in ANGPTL3 are associated with reduced levels of serum lipids and reduced risk of atherosclerotic cardiovascular disease. The Phase 1 trial is enrolling patients with mixed dyslipidemias, homozygous familial hypercholesterolemia, and severe hypertriglyceridemia.

• CRISPR Therapeutics has initiated a Phase 1 clinical trial for CTX320, an investigational program targeting lipoprotein(a) (Lp(a)). Elevated Lp(a), which is associated with an increased risk of atherosclerotic cardiovascular disease, is present in approximately one in five people in the United States and around the world.

• CRISPR Therapeutics expects to nominate additional in vivo programs targeting both rare and common diseases this year, to be disclosed in mid-2024.
  

Regenerative Medicine

• CRISPR Therapeutics announced today that ViaCyte, Inc. (a subsidiary of Vertex Pharmaceuticals, Inc.) has elected to opt-out of the collaboration with CRISPR Therapeutics for the co-development and co-commercialization of gene-edited stem cell therapies for the treatment of diabetes. Per the opt-out terms, the on-going collaboration assets will now be wholly owned by CRISPR Therapeutics, subject to a royalty on future sales owed to ViaCyte. The opt-out will become effective in early February. The ViaCyte collaboration assets include CTX211 (formerly VCTX211), an allogeneic, gene-edited, immune-evasive, stem cell derived product candidate that is transplanted into patients in a device and intended to produce insulin in a glucose-dependent manner. CRISPR Therapeutics continues to advance a Phase 1 clinical trial for CTX211 for the treatment of Type 1 Diabetes (T1D). CRISPR Therapeutics remains committed to its goal of developing a beta-cell replacement product that does not require chronic immunosuppression.
• Separate from the ViaCyte collaboration, Vertex continues to have non-exclusive rights to certain CRISPR Therapeutics’ CRISPR/Cas9 technology to accelerate development of potentially curative cell therapies for T1D. Vertex paid $170 million to CRISPR Therapeutics in upfront and milestone payments in 2023 as part of that licensing agreement, and CRISPR Therapeutics remains eligible for an additional $160 million in research and development milestones and would receive royalties on any future products resulting from this agreement.

Manufacturing