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CRISPR Therapeutics (CRSP) investor relations material

CRISPR Therapeutics Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary12 Nov, 2025

Portfolio and financial highlights

  • CASGEVY launch progressing well, with over $100M revenue expected in 2025 and significant growth in 2026 as nearly 300 patients have started treatment.

  • Pipeline advancing, including CTX310 (ANGPTL3) showing 55% triglyceride and 50% LDL reduction, with strong KOL support.

  • Cell therapy updates for oncology and autoimmune disease expected by year-end, with initial CTX112 autoimmune data and higher dose oncology data forthcoming.

  • Factor XI program highlighted at AHA; TKA study readout expected in the next 12 months, potentially de-risking a major cardiovascular target.

  • $1.94 billion in cash reserves, supporting execution of strategic initiatives.

Factor XI program strategy and differentiation

  • Targeting patient populations at risk for clots but not on anticoagulation due to bleeding risk, aiming for specific, potent inhibition with limited bleeding.

  • Focus on stroke prevention, AF, cancer-associated thrombosis, and post-procedure prevention where standard of care is lacking.

  • Differentiation through single-shot siRNA therapy with prolonged activity, improving compliance and offering reversibility in case of bleeding.

  • Safety advantage over biologics and orals due to easier reversibility of siRNA effects.

  • Phase 2 TKA study readout expected in H2 2026, providing key de-risking data.

Competitive positioning and pricing

  • Positioned to benefit from being 12–18 months behind competitors, allowing benchmarking against Milvexian and Abelacimab for both efficacy and pricing.

  • Differentiated product profile in durability and reversibility expected to support market entry and pricing strategy.

  • Ability to observe competitor strategies before finalizing own approach.

Factor XI: How will durability/reversibility drive market share
CTX320: Horizon data impact on dosing & population
Gene editing safety: Differentiate from Intellia events
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Frequently asked questions

Transforming medicine

CRISPR-Cas9 had its initial description ten years ago when a study revealed the potential of exploiting the system for RNA-programmable gene editing. It allows scientists to change the molecules that carry the instructions to produce proteins without changing the original DNA code.

The technology has its foundation in the natural defense mechanism of bacteria and single-celled microorganisms. When mentioned, CRISPR often links to Cas9, the protein that helps target and cut the genetic code to be edited. You can therefore see Cas9 as the genetic scissors cutting the DNA at a specific point, and once the gene is cut, there are two possible outcomes:

  1. The cell repairs itself.

  2. The researchers insert a new DNA template for the repair.

A true gene editing enabler?

CRISPR technology is transforming medicine, enabling us to treat and prevent many diseases. It also has the potential to be used to make precise changes, such as replacing faulty genes – true gene editing.

We have seen remarkable progress within this technology in the first ten years. As new editing technologies develop, we can insert larger DNA fragments more precisely, enabling us to tackle more of the 75,000 genetic variants associated with human disease. Some of CRISPR’s main competitors include Intellia Therapeutics, Denali Therapeutics, Vaxcyte, and Ginkgo Bioworks.

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