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CRISPR Therapeutics (CRSP) investor relations material
CRISPR Therapeutics Citi’s 2026 Virtual Oncology Leadership Summit summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Oncology program strategy and innovation
Focus on zugo-cel, an allogeneic CAR-T therapy with advanced edits for potency and persistence, targeting CD19 in hematologic malignancies.
Key edits include CAR insertion into the TCR locus, beta-2M knockout for immune evasion, and proprietary Regnase-1 and TGF-beta receptor 2 knockouts to enhance durability and cytotoxicity.
No HLA matching required, enabling broad patient applicability and redosing without immune response issues.
Standard lymphodepletion regimen is used, matching autologous CAR-T protocols, with improved cell expansion and efficacy.
Combination with BTK inhibitor pirtobrutinib is being explored to further improve durable complete response rates.
Autoimmune program expansion and vision
Rapidly enrolling and expanding indications in autoimmune diseases, including lupus, myositis, scleroderma, ITP, and WAIHA.
Allogeneic CAR-T offers cost, scalability, and convenience advantages over autologous therapies, with sub-$10,000 cost of goods.
Early data in SLE patients show complete remission and immune reset, with zero disease activity and sustained B-cell depletion.
Active trials in ITP and WAIHA, targeting high unmet need and less competitive indications for potential first-mover advantage.
Dose of 100 million cells used across indications, with ongoing evaluation for optimal dosing.
Regulatory and commercialization strategy
Parallel development in oncology and autoimmune, with regulatory discussions planned to determine registration paths.
Potential for single-arm trials in relapsed/refractory oncology settings and small trials for autoimmune indications.
Pricing strategy aims for $200K or lower per treatment, significantly undercutting autologous CAR-Ts and increasing access.
Plans for lower-cost manufacturing in Asia and other regions to further expand global access.
Commercial focus includes niche oncology populations and broad autoimmune indications, with potential for $2B+ in revenue.
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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:
The cell repairs itself.
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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