CRISPR Therapeutics Expands Beyond Blood Disorders

- CRISPR Therapeutics is advancing its *in vivo* programs which edit genes directly inside the body, using lipid nanoparticles (LNPs) to deliver the gene-editing components to the liver. The company has two key cardiovascular disease programs using this approach: CTX310, which targets the gene ANGPTL3, and CTX320, which targets a gene called LPA. - A Phase 1 trial of CTX310, a one-time treatment, showed it could safely reduce "bad" cholesterol and triglycerides. The therapy works by switching off the ANGPTL3 gene in the liver, which is known to regulate lipid levels. People naturally born with a non-working copy of this gene have a lower lifetime risk of cardiovascular disease. - The company is also moving into immuno-oncology, developing "off-the-shelf" CAR-T cell therapies from healthy donors' cells instead of a patient's own. Their next-generation candidates, CTX112 and CTX131, include additional gene edits intended to boost the potency and persistence of the cancer-fighting cells. - Beyond cancer, CRISPR Therapeutics is exploring its CAR-T cell therapy, CTX112, for autoimmune diseases. A clinical trial is planned to treat systemic lupus erythematosus (SLE), with the potential to expand to other autoimmune conditions. This approach could offer a scalable and more cost-effective treatment compared to therapies that require using a patient's own cells. - In 2025, CRISPR Therapeutics partnered with Sirius Therapeutics to co-develop and co-commercialize small interfering RNA (siRNA) therapies, a different method for altering gene expression. Their lead program under this collaboration is CTX611 (also known as SRSD107), which targets a factor involved in blood clotting and is being studied for the prevention of thromboembolic disorders. - A career in developing these therapies on the tech side, such as a computational biologist, involves writing code (often in Python or R), analyzing large biological datasets like DNA sequences, and building computational tools to interpret experimental results. This path typically requires at least a bachelor's degree in a field like bioinformatics or genetics, with many roles requiring a master's or Ph.D. - In contrast, a patient-facing role like a genetic counselor involves direct interaction with individuals and families to help them understand genetic testing and hereditary risk factors. This career requires a master's degree from an accredited genetic counseling program and often involves coursework in human genetics, psychosocial counseling, and bioethics, alongside clinical experience.