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CRISPR Therapeutics (CRSP) investor relations material
CRISPR Therapeutics Morgan Stanley 23rd Annual Global Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Morgan Stanley 23rd Annual Global Healthcare Conference summary9 Sep, 2025
Strategic priorities and pipeline focus
Emphasis on advancing a diversified pipeline in cardiovascular, autoimmune, oncology, and diabetes, with multiple clinical readouts expected in the next 12 months.
Cardiovascular programs target ANGPTL3, Lp(a), and factor XI, with in vivo gene editing and siRNA approaches under development.
Autoimmune and oncology focus on CTX112, a CD19 allogeneic CAR T, aiming for best-in-class expansion and scalability across indications.
Diabetes program leverages hypoimmune islet cell transplantation, aiming for competitive positioning in the space.
Strong balance sheet supports ongoing development and potential partnerships, especially in cardiovascular.
Business development and partnership outlook
Increased pharma interest in partnerships, particularly for cardiovascular assets, due to resource and bandwidth considerations.
Ongoing discussions with pharma, with decisions on partnerships expected as new data emerges over the next six months.
External data, such as Novartis' Horizon trial for Lp(a), will influence positioning and partnership strategies.
Regulatory and policy environment
FDA leadership is supportive of genetic medicines, with ongoing efforts to streamline guidelines and reduce early CMC requirements.
Regulatory framework for cell and gene therapy is being clarified, with a task force addressing overlapping guidelines.
The next few months are expected to provide more clarity on regulatory direction.
What drives current pharma interest in partnerships?
What are key dynamics impacting CASGEVY throughput?
How is FDA approach to genetic medicines evolving?
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Next CRISPR Therapeutics earnings date
Q3 20254 Nov, 2025
Next CRISPR Therapeutics earnings date
Q3 20254 Nov, 2025
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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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