The Craig Martin Lab in chemistry recently received an Acorn Award from the Massachusetts Technology Transfer Center (MTTC), one of 13 innovation seed-funding $15,000 grants given statewide intended “to support the demonstration of the viability of a technology developed at Massachusetts research universities.”
Martin and colleagues will work to advance what he calls “a wide variety of new RNA therapeutics that are on the horizon today,” such as mRNA-based therapeutics, RNA-guided technologies such as CRISPR and RNA “logic gate” smart therapeutics.
“The RNA molecule, the central component of life’s ‘central dogma,’ has significant advantages over current approaches,” he says. “I am delighted that the Commonwealth has chosen to invest in our proposal and am excited by prospects for enabling an exciting new direction in therapeutics.”
Martin is an expert with more than 30 years’ experience working with ribonucleic acid (RNA) model systems. RNA, found in all living cells, usually serves as a messenger bringing instructions from DNA to controlprotein synthesis. He says in recent years RNA has become “poised to revolutionize the treatment and prevention of a wide variety of disorders and diseases, but deficiencies in its laboratory synthesis are holding back applications such as RNA vaccines and other therapeutic drugs.”
He and colleagues have steadily built upon recent work such as their 2018 study, where they established a foundation for breaking through a major roadblock that has held back researchers who wanted to make large quantities of RNA for biomedical and biotech studies. Now, two years later, the difficulties center on impurities in synthetic RNA that are limiting the full application of many of these systems, the lab director points out.
He says, “The development of dramatically improved RNA preparations will remove these barriers, allowing these new technologies to offer new and improved benefits across a wide spectrum of the biomedical landscape. This Acorn Award is focused on one such approach with the aim of demonstrating a path forward to high-quality, high-yield RNA.”
DNA sequences encode the production of everything in most living cells, Martin explains, while RNA molecules, copied from DNA, are the temporary workhorse sequences that actually drive and sometimes control that process. “In genetic diseases, something is wrong in a person’s code.Scientists are working on ways to correct faulty code during this process or to add code to prevent diseases. This work requires very pure RNA so that the instructions are clear. We are developing new approaches toward making such RNA in the laboratory,” he says.
MTTC Acorn Awards are funded by the Commonwealth to enable public and private research universities and medical centers in the state “to lead the nation in translating basic research to the market, creating jobs and spurring economic development,” the organization states.
This year’s recipients, selected from among 24 applicants, were chosen for their technical merit, commercial viability, project plan and strength of team, says Vinit Nijhawan, MTTC interim executive director. “The strength of the selected projects demonstrates that Massachusetts leads the nation in translating basic research to the market,” he adds.
Martin says, “Clearly mRNA therapeutics companies are very interested” in this work, along with firms developing CRISPR gene-editing technologies. “But I’ve also had interest from more traditional small-molecule drug companies that target key biological RNAs,” he adds. “For example, alternative splicing complexes are a huge future target. In this case, the purity of their target is key to success in screening candidates. The future of these technologies is virtually unlimited!”