The gene therapy market in the United States is growing rapidly, fueled in part by new gene editing technologies such as CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). Scaling gene therapy will require a number of new technologies for both researchers and clinicians. Currently, there is a growing unmet need for Cavidi’s expertise in Reverse Transcriptase (RT) Technology, specifically Cavidi’s long experience quantifying infectivity in a class of retroviruses called lentiviruses, such as HIV. These are the viruses that are most often used as delivery mechanisms (called viral vectors) in gene therapy using CRISPR.
“There is a clear role for Cavidi to play in this growing segment and we will be using the ALMI grant to develop a strategy that will get us to market and allow us to contribute in a meaningful way,” says Cavidi CEO John Reisky.
Viral vectors are the tools commonly used by molecular biologists to deliver genetic material into human cells. They are derived from existing strains of viruses found in nature. Among the most effective of these natural delivery systems are a class of viruses called lentiviruses. This class of viruses has evolved to be highly effective at transporting their genetic payload into the cells they infect. In gene therapy, the payload is replaced to include beneficial genomes. But even among a class of viruses as evolved as lentiviruses, there is a broad range of quality. In this case, quality refers to the viruses infectivity — the ability of the virus to successfully deliver its beneficial payload into the target cells.
A lentivirus has several components that all must be working for it to succeed in its delivery task. Since the virus is an organic entity and highly susceptible to its environment, many viruses in any given sample will have damage to one or more of the components that will prevent it from infecting its target. If enough of these duds are present, it can result in treatment failure or even adverse side effects for the patient.
Right now it is possible for the people who work with lentiviruses to see how much virus is present in any given sample. But what is far more difficult to see is infectivity, which shows how capable those vectors are of doing their job, specifically to infect other cells. Measuring infectivity of lentiviruses is a core technology at Cavidi and an area it has championed for decades in the fight against HIV.
The growing interest in lentiviruses with developments like CRISPR has significant implications for Cavidi. Its expertise with RT technology could have implications across a number of life science applications from basic research and bio-manufacturing to new vaccines and gene therapies for a broad range of conditions. One goal of the new project co-funded by the ALMI grant will be to define these opportunities and develop a path to market.