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CRISPR Therapeutics
CRISPR Therapeutics (CRSP) investor relations material
CRISPR Therapeutics 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.44th Annual J.P. Morgan Healthcare Conference summary12 Jan, 2026
Strategic Progress and Therapeutic Franchises
Four franchises advancing: Heme, in vivo gene editing, CAR-T, and diabetes/T1D, with significant milestones and best-in-class results in each.
CASGEVY achieved global regulatory approvals for SCD and TDT, launched in 10 countries, exceeding $100M in revenue, and expanding into pediatric populations with ongoing regulatory submissions.
In vivo gene editing platform demonstrates best-in-class editing in hematopoietic stem cells and liver, with >50% editing in NHPs and durable results, plus promising data for CTX310 in hypercholesterolemia.
CAR-T platform (CTX112/zugo-cel) shows high response rates in oncology and autoimmune diseases, with 70% CR in DLBCL, durable remissions in SLE, and ongoing expansion into new indications.
Advancing islet cell therapies for type 1 diabetes, with proof-of-concept data and new candidates moving toward the clinic.
Commercial and Clinical Progress
Over 75 authorized treatment centers globally for CASGEVY, with rapid patient initiation and cell infusions.
Pediatric pivotal studies showed 100% of children with sufficient follow-up achieved primary endpoints for SCD and TDT.
Regulatory submissions for pediatric use expected in 1H 2026; included in FDA's CNPV program and CMMI pilot for Medicaid access.
Partnerships and potential out-licensing considered for pipeline assets as pharma interest in gene and cell therapy increases.
2026 positioned as a pivotal year, with key data readouts and regulatory clarity expected to define the next phase.
Innovation in Enabling Technologies
Targeted conditioning with antibody-drug conjugates for HSC depletion, minimizing off-target toxicity.
Proprietary delivery systems and optimized editing for durable, redosable gene therapies.
AI is leveraged in preclinical design, especially for protein and mRNA folding, with future clinical applications anticipated.
In vivo CAR-T platform advancing with non-viral, targeted LNP delivery and durable T-cell expression.
Allogeneic CAR-Ts and in vivo approaches aim to reduce costs, with pricing strategies targeting affordability and broad access.
CASGEVY pediatric expansion and market access
CTX310's 'one and done' market disruption potential
In Vivo CAR-T non-viral delivery advantages
A1AT: How will you achieve best-in-class differentiation?
How will HORIZON trial data impact LP(a) strategy?
How does zugo-cel overcome allogeneic CAR-T challenges?
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Next CRISPR Therapeutics earnings date
Q4 202511 Feb, 2026
CRISPR Therapeutics
Next CRISPR Therapeutics earnings date
Q4 202511 Feb, 2026
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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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