Andre Watson Shares What's Missing from the Gene Editing Industry — And how to Block the Coronavirus

Years ago, Andre Watson, co-founder and CEO of Ligandal, started to develop a standardized delivery tool that could bring gene-editing machinery to any cell in the body.

After graduating college in 2013, Watson started Ligandal to fill in what he sees as a crucial gap in the genetics industry: Scientists are finding more and more ways to treat disease or improve the human condition with CRISPR and other gene-editing tools, but there's no standardized platform or tool to get the gene-editing machinery where it needs to go within the body.

»We want to have the underlying technology in place to be able to direct to these payloads, like CRISPR, to any cellular address,« Watson says.

The company was making steady progress. Watson and his team developed synthetic peptides capable of targeting receptors on specific cell types, allowing them to shuttle gene-editing machinery to the exact organ or intended cellular target rather than merely injecting them and hoping it all worked out.

»We want to have a set of platforms for targeting all these different tissues that can be rapidly tailored,« Watson says.

Pivot to make SARS-COV-2 antidote

The company had shown impressive results in targeting T cells and the haematopoietic stem cells that act as progenitors to blood cells and much of the immune system, with plans to expand to other cell types. But then, just as it disrupted every other aspect of life, the coronavirus pandemic came in and took its toll — and Watson rapidly switched gears to help out.

In early August, Ligandal published a preprint paper describing SARS-BLOCK: an adaptation of the company's targeted delivery system that, instead of carrying molecules to a certain cell, binds to the ACE2 receptors favoured by the coronavirus to bar its entry.

»We went for a pivot in our short-term strategy and the first product we wanted to develop because we were able to utilise this synthetic peptide approach to, essentially in a matter of hours, model and predict how a small fragment of the virus could be printed out synthetically to serve as an active block of viral entry while also doubling as an immunomodulatory agency that's almost like a vaccine in the sense that it binds to neutralizing antibodies,« Watson says.

»That means that you could couple this sort of antidote with an expected prophylactic action and an expected immunomodulatory action. And so, we're commercializing what we are calling a prophylactic and therapeutic or, quote-unquote, an "antidote vaccine" that we're aiming to bring to market through the therapeutic pathway with the FDA,« Watson adds.

Room-temperature-stable nasal spray

Pivoting from gene-editing to SARS-BLOCK took all of five hours, Watson explains, because the underlying system is fairly similar. He and his team had already done the leg work of developing a computational screening platform that could model how synthetic peptides would fold and bind to different cell types or receptors. So rather than starting from scratch, developing an experimental coronavirus therapeutic was essentially no different from merely adding another application to the existing platform.

There's still a long way to go before anyone might take SARS-BLOCK to prevent or block a coronavirus infection. Since August, Ligandal has been speaking to federal agencies in the United States like the National Institutes of Health (NIH) and the National Institute of Allergy and Infectious Diseases (NIAID) and pharmaceutical companies that would be able to help mass-produce and distribute the product.

»Over the next several months, we'll be working with them as well as contractors to perform the preclinical studies that we need to complete to be able to file an emergency investigational new drug application with the FDA, and take it into a Phase I. Ideally, a combined Phase I/II study that measures both the safety and efficacy of the compound in reducing the severity of infection,« Watson says.