AI is helping biotech companies speed up drug discovery and gene therapy development for rare diseases by automating tasks that once required large teams of scientists.
Boston, MA: Modern biotech companies have the tools to edit genes and design drugs, but thousands of rare diseases remain untreated. \n\nAccording to company leaders, AI is becoming the force multiplier that lets scientists take on problems the industry has long left untouched. \n\nThe companies say this technology can increase productivity and tackle the shortage of workers and talent in the field.
Insilico Medicine recently launched its “MMAI Gym” that trains generalist AI models like ChatGPT to perform as well as specialist models. \n\nThe goal is to build an AI system that can solve many different drug discovery tasks at the same time with super-human accuracy. \n\nThe company says its AI platform can look through huge collections of information to find promising drug candidates and even reuse existing medicines, all while saving time and money.
GenEditBio is working on a new way to deliver gene-editing tools directly into the body using tiny delivery vehicles. \n\nTheir AI system analyzes data to figure out which chemical designs work best for reaching specific body parts like the eye or liver. \n\nThe company recently received FDA approval to start trials of CRISPR therapy for a rare eye disease.
Both companies face a big challenge: they need more high-quality data to train their AI systems. \n\nMuch of the available data comes from Western countries, which means the AI models may not work as well for people from other parts of the world. \n\nThe companies are working to generate more diverse data and build virtual models of human biology to run computer-based clinical trials. \n\nThey hope this will lead to more treatment options for rare diseases in the next 10 to 20 years.