CRISPR Therapeutics (CRSP) Goldman Sachs 45th Annual Global Healthcare Conference summary

Portfolio strategy and pipeline prioritization

• Focuses on areas where gene editing can be most transformative, balancing rare and common diseases for maximum impact.

• Pipeline includes cancer, ex-vivo non-cancer, regenerative medicine, and in-vivo gene editing, with high entry standards for new programs.

• Leverages core competencies in cell handling and technology to expand into CAR-T therapies and regenerative medicine.

• In-vivo gene editing is expanding beyond liver to other organs, aiming for leadership in the space.

• Portfolio is managed with a risk-reward approach, focusing on programs that can significantly change disease landscapes.

Innovation and technology adaptation

• Established CRISPR-X group to drive next-generation gene editing and incorporate new effector proteins.

• Applies a practical approach to technology adoption, selecting the best tools for each disease.

• Invests in both internal development and external innovations to maintain leadership.

Delivery technology advancements

• Shifted focus from viral vectors to lipid nanoparticles (LNPs) for safer, more controllable delivery.

• LNPs now being used for multiple organs, including eye and lung, with ongoing work to improve targeting and durability.

• Regulatory preference and safety profile of LNPs support their adoption.