Rachel Haurwitz, Co-founder and CEO of Caribou Biosciences, discusses using CRISPR to engineer human immune cells to fight cancer. Topics include the journey of CRISPR from breakthrough to FDA approval, enhancing CRISPR's effectiveness, balancing affinity in CRISPR editing, challenges in CAR T-cell therapies for cancer, and reflections on career shifts and gene complexities.
Enhancing CRISPR for CAR T-cell therapy development to broaden accessibility and efficiency in cancer treatment.
Developing CRISPR technologies like Chardonnay to improve editing accuracy and minimize genetic off-target effects.
Deep dives
Overview of CRISPR Technology and Approvals
CRISPR, a revolutionary gene-editing tool, has advanced to the stage of clinical drug approvals, with the first FDA-approved CRISPR-produced therapy for sickle cell and beta thalassemia. Scientists aim to enhance CRISPR for more treatment options. The field has rapidly progressed from foundational research to approved therapies within a remarkably short timeframe.
Challenges and Innovations in CAR T-cell Therapies
In the realm of CAR T-cell therapies, which engineer immune cells to combat cancer, challenges such as high costs and customization limitations exist. Companies like Caribou Biosciences are developing off-the-shelf versions to broaden accessibility and efficiency. The aim is to create CAR T-cell therapies that can be administered more widely without the need for individual customization, enhancing patient treatment opportunities.
Addressing Specificity in Gene Editing with CRISPR
One key focus area for CRISPR advancements is enhancing specificity in genome editing to ensure precise modifications. Caribou Biosciences' Chardonnay technology, incorporating hybrid guides of RNA and DNA, significantly improves editing accuracy by decreasing genetic off-target effects. The technology strives to target specific genetic sequences effectively while minimizing unintended alterations.
Future Prospects and Enhancements in CRISPR Applications
The field of CRISPR continues to evolve, with ongoing efforts to refine editing techniques and optimize therapeutic outcomes. Companies like Caribou Biosciences are pushing the boundaries with multiple genome edits and novel approaches to improve therapeutic efficacy. Research advancement aims to strike a delicate balance between editing efficiency, cellular functionality, and treatment longevity to enhance the effectiveness of CRISPR-based therapies.
Last year, the FDA approved a treatment for sickle cell disease using a revolutionary new gene editing technology called CRISPR. Rachel Haurwitz conducted pioneering research on CRISPR as a graduate student. Now she’s the co-founder and CEO of Caribou Biosciences. Rachel's problem is this: How can you improve CRISPR and use it to engineer human immune cells to fight cancer?