Gene therapy has the potential to treat a wide range of otherwise intractable diseases by making precise edits to the genome. But the most common method used to deliver gene-editing components involves adeno-associated viruses, which can result in unintended, “off-target” edits. Samagya Banskota, 32, has co-invented a more efficient delivery system using engineered virus-like particles. “We now have a system that can safely and effectively deliver genome editors to multiple tissue and organ types,” Banskota says. That should make it easier to develop therapies for a variety of genetic disorders.
Imagine if you could replace a vaccine jab or an insulin shot with a pill. Alex Abramson, 29, is developing a way to make it possible. Until now, pills haven’t been capable of delivering drugs based on proteins and nucleic acids, since these molecules are rapidly degraded by the enzymes in the gastrointestinal tract. Further, the biological molecules are too large to pass through the tissue wall of the stomach. Abramson’s innovation is a pill that falls to the bottom of the stomach and reorients itself, inserting the medicine directly into the stomach tissue.
Recent advances have identified the genetic risks behind many human diseases, but the next big step is to observe how these genetic variations lead to the illnesses, and how we might modify or even reverse the pathologies. Xin Jin, 34, has invented a method that allows simultaneous analysis of many genes in living organs. “Previously, genetic studies were done by analyzing one gene at a time, and typically one or a few cell types at a time,” she says. Her method allows scientists to examine dozens or hundreds of genes in each experiment, and it works for many different types of organs and cells. Jin is using it to better understand how genetic changes affect mental health.
Ovarian cancer kills more than 184,000 women worldwide every year. A better way to detect early-stage cases could greatly lower that number. Mijin Kim, 32, combined machine learning with a special sensor to detect a blood-based “fingerprint” of ovarian cancer. Kim hopes the benefits of her liquid biopsy don’t end with one illness. “This method could be rapidly adapted to the detection of many conditions,” she says. “The array could be used to train an algorithm to recognize nearly any disease when given enough data from the sensor.”
The gene-editing tool CRISPR has revolutionized research, but so far it’s been hard to use it to treat disease—it’s proved difficult to make the treatments specific enough to be fully safe, and delivering the therapy inside the body has also been complicated. As CEO of Scribe Therapeutics, Benjamin Oakes, 33, is working to optimize novel CRISPR enzymes and ways to package the gene-editing systems to solve those problems. “Our engineered gene editors are more active and create more productive edits, are enhanced to more specifically target any part of the genome, and are more compact,” Oakes says. All that makes it possible to target the underpinnings of many more diseases.
Amblyopia, the leading cause of vision loss in children, is usually treated by having patients wear a patch over the stronger eye to stimulate the weaker one. Despite how prevalent the condition is, affecting 3% of children, it can be hard to get kids to comply with wearing the eye patch, partly because of social stigma. Scott Xiao, 24, has won FDA approval for an alternative: a digital therapy aimed at retraining the brain to process images from both eyes properly. It involves presenting popular TV shows slightly differently to each eye via a virtual-reality headset—making it the first FDA-approved VR therapeutic for any condition.