CRISPR technology has appeared in thousands of scientific publications, capturing the attention of researchers worldwide. Laboratories across the globe are striving to optimize its molecular mechanisms, refining its components for a wide range of applications. Despite its promise, challenges remain, particularly in making therapeutic applications more efficient and accessible.
Biotechnology and CRISPR
Currently, many gene therapies using viral-free vectors rely on ex vivo editing. This involves removing a patient’s cells, editing them in a lab, and reintroducing them into the body. While effective, this approach has significant drawbacks, including lengthy hospital stays and exorbitant costs, limiting accessibility. For example, the FDA-approved CRISPR-Cas9-based therapy Casgevy, designed to treat sickle cell disease, provides life-changing potential but comes at a staggering one-time cost of $2.2 million.
Jennifer Doudna, Nobel laureate and co-recipient of the 2020 Nobel Prize in Chemistry for her work on CRISPR-Cas9, emphasizes the importance of new biotechnology in lowering these costs. “Innovative delivery methods and improved manufacturing processes will be key to making gene-editing therapies more affordable,” she notes.
At the Innovative Genomics Institute (IGI), Doudna’s lab is exploring ways to bypass the ex vivo cell engineering step. One promising avenue is the development of enveloped delivery vehicles (EDVs). Dr. Jennifer Hamilton, a CRISPR researcher in Doudna’s lab, initially demonstrated how the outer envelope of an emptied HIV-1 virus could be repurposed to edit T cells ex vivo, transforming them into CAR T cells. Over time, these virus-like particles (VLPs) were refined into EDVs.
EDVs hold significant potential due to their ability to target specific cells with precision. By coating EDVs with antibody fragments, researchers can enhance their binding capacity and specificity, opening new doors for efficient and targeted therapies. However as research progresses, CRISPR continues to move closer to reshaping the landscape of modern medicine.
