The finalists in the Big Idea category of the Xconomy Awards San Diego are pursuing ambitious goals in pursuit of better ways of improving health. Their approaches range, from a new way to deliver insulin to patients with Type 1 diabetes to an innovative method to detect brain trauma that could reduce the need for medical imaging. Here are brief profiles of the finalists.
This is the last in a series of articles about the finalists for the Xconomy Awards San Diego. We’ve written about the CEO, Commitment to Diversity, Innovation at the Intersection, Digital Trailblazer, X of the Year, and Startup and Secret Weapon finalists. We’ve also profiled our Lifetime Achievement Award winner, Ivor Royston. Winners will be announced at the Awards Gala on May 29.
Surgery isn’t just about fixing a medical problem, but also about preserving function after the procedure. Alume Biosciences is developing a “precision surgery” tool for physicians to improve their ability to visualize, and avoid harming, a patient’s nerves during an operation. The fluorescently-labelled molecule that underlies Alume’s core technology binds to human nerves, essentially making them glow under fluorescent light. The technology was developed as a collaboration between founder and CEO Quyen Nguyen, a UC San Diego surgery professor, and the late Roger Tsien, a Nobel laureate in chemistry.
A patient would get an injection with the agent that would make it easier for surgeons to spot nerves that would otherwise likely be hidden during an operation. Tagging the nerves could help surgeons better protect the hard-to-spot fibers, and reduce the incidence of nerve injury caused by surgery. The company says it hopes to start clinical trials in 2020.
American Gut Project
A lab at the UC San Diego School of Medicine is home to the world’s largest crowdsourced microbiome research project, launched in 2012 by researchers Rob Knight, Jeff Leach, and Jack Gilbert. Called the American Gut Project, the program interweaves big data and science to pinpoint new insights about the effects of the microbiome on human health. It has collected more than 15,000 stool samples from people around the world.
The project sends out a sample kit to volunteers who provide a stool sample as well as mouth and skin swabs. In Knight’s lab, the bacterial DNA in each sample is isolated and sequenced to identify the bacterial species.
Last year the team published the first major results from the project, finding correlations between more diverse gut microbiomes in people who ate more than 30 different plant types per week compared to those who ate 10 or fewer types of plants per week, regardless of the type of diet they followed.
Banyan Biomarkers has developed a new way to determine whether a patient has suffered brain trauma. The test measures levels of certain proteins in the blood to evaluate whether the brain is functioning normally following an injury.
Patients with traumatic brain injury can appear healthy, even as they suffer from symptoms such as headache, confusion, and sensitivity to light. Diagnosis today often involves a CT scan but concussions, as milder forms of traumatic brain injury (TBI) are termed, aren’t always detectable that way. The scans are also expensive, and expose patients to radiation. The test is intended to help clinicians determine the need for a head CT scan, hopefully reducing the need for neuroimaging in cases of suspected TBI. Results of the blood test can be available within three to four hours.
Last year the FDA approved Banyan’s Brain Trauma Indicator to aid in the evaluation of concussion in adults—the first blood test the agency approved for that purpose. It is also of interest to healthcare providers for use in areas where access to scanners is limited. The U.S. Department of Defense, for example, worked with the FDA to expedite approval of Banyan’s test for use as a diagnostic for members of the military.
In October, the DOD, which said finding a better way to evaluate such injuries had been a “top priority” for more than a decade, signed a contract with Banyan to use its test to diagnose soldiers at military installations.
The first CAR-T therapies, which use a patient’s own engineered immune cells to fight cancer, came on the market last year. Fate Therapeutics is working on an approach to cancer treatment that deploys stem cell science to design “off-the-shelf” versions of such treatments. Last year, Fate received the FDA’s OK to move its lead candidate into the clinic.
Derived from stem cells, such therapies could be more easily scaled and therefore less expensive than the highly personalized CAR-Ts in use today. The biotech’s pipeline includes drugs it is developing from cells from healthy donors, but the potential game changer is its investigational drugs derived from induced pluripotent stem cells (iPSC). The company is using those cells to create “master iPSC lines”—a potential renewable resource for manufacturing a wide array of cell types via what Fate calls an “industrialized” production.
The company is working with collaborators, including Ono Pharmaceuticals and Memorial Sloan-Kettering Cancer Center, and investors have backed it with hundreds of millions in financing, including a $144 million round raised in September.
Gene Yeo, University of California, San Diego
Gene Yeo’s research on RNA led his lab to come up with a new CRISPR-based approach to treating genetic disease. Yeo, a UC San Diego professor, has demonstrated a way to use CRISPR gene editing, typically targeted at DNA, to instead manipulate specific RNA molecules in various ways.
Yeo’s approach was initially developed to enable basic researchers to track and follow the fate of RNA in living cells. Then, in 2017, his lab showed how, in principal, a CRISPR system that targets RNA could reverse molecular and cellular defects related to repetitive RNAs that cause myotonic dystrophy type 1, a form of adult-onset muscular dystrophy, and similar genetic diseases. The technology also allows scientists to edit single nucleotides in RNA, increase protein production, and to correct RNA splicing defects.
The discovery provided the foundation for Locana, a biotech Yeo co-founded to advance CRISPR-based RNA targeting for human medicines. Eliminating faulty RNAs could be a safer way to deploy CRISPR, which, when aimed at DNA, can cause unwanted genetic cuts that could lead to serious side effects.
ViaCyte is developing a cell therapy that could replace the insulin-producing cells that die in patients with type 1 diabetes. The company is working on a way to “hide” the replacement cells from the immune system—a potential “functional” cure for those with the condition. The hope is that the cell therapy would allow patients to reduce or even eliminate the need for insulin injections.
To achieve its aims, ViaCyte is developing two products, PEC-Direct and PEC-Encap. Both are devices that are filled with stem cells and implanted under the skin. The cells are meant to mature into the pancreatic cells that produce insulin. PEC-Direct would require the patient to also take immunosuppressants. Viacyte anticipates that patients treated with PEC-Encap wouldn’t require such drugs, which can cause a range of unwelcome side effects. The PEC-Encap device uses semi-permeable materials meant to protect the implanted cells from immune attack, while still allowing nutrients to flow through. The device is being designed in collaboration with W. L. Gore & Associates, the makers of Gore-Tex. Both products are in clinical testing.
The company has also teamed up with CRISPR Therapeutics to develop a gene-edited cell therapy for diabetes that would avoid triggering an immune response.