Some diseases with known and understood genetic causes can be addressed with oligonucleotide-based drugs, therapies consisting of synthetic DNA and RNA fragments. Research on oligonucleotides dates back decades, but the list of FDA-approved therapies using this nucleic acid chemotherapy is relatively short and consists entirely of drugs for rare diseases.
Creyon Bio co-founder and CEO Chris Hart said developing oligonucleotide drugs is a slow and expensive process, which limits the scope of such drugs. The startup aims to advance how oligonucleotides are discovered, using artificial intelligence-based technology to make the process faster and more efficient. Creyon, which operates between San Diego and Research Triangle Park in North Carolina, has been developing its technology for the past two years.It’s now out of stealth, backed up $40 million in terms of funding.
Hart has a deep understanding of the oligonucleotide field, having spent nearly eight years at Ionis Pharmaceuticals, an oligonucleotide drug developer with a portfolio of three FDA-approved products. The way Ionis and other biotech companies develop their oligonucleotide drugs has been trial and error, screening a molecule to assess its activity on a target and its potential to cause toxic effects beyond that target. Creyon designs RNA-based therapies. But the process doesn’t start with RNA. It starts with data.
“We’re a data-first company,” Hart said. “We can’t build any models until we create the data. We create the data and build the models, and those models are orders of magnitude more efficient at creating leads.”
Creyon co-founder and chief scientific officer and Ionis veteran Swagatam Mukhopadhyay said the startup does oligonucleotide research by reimagining how drug development works. The company has built massive datasets containing chemical information on oligonucleotides. Using artificial intelligence and machine learning, Creyon’s technology can reveal everything you need to know about a potential drug, such as how it’s delivered to its target and how toxic it is in a particular tissue.
These measurements were tested in human cells to put them in the context of how they act as treatments. With the insights gained from the analysis, Creyon produces its drugs — highly programmable therapies that target specific tissues, backed up by data showing that the molecules are safe and effective.
The recently announced financing is a combination of seed and Series A investments led by DCVC Bio and Lux Capital. Other investors backing Creyon include Casdin Capital, Alexandria Venture Investments and BioBrit. The company is using the cash to optimize its technology, expand its workforce and lay the foundation for a drug pipeline that can span a range of modalities, from antisense oligonucleotides to small interfering RNAs and DNA and RNA editing therapies.
Creyon’s specific disease targets remain undisclosed, but Hart said the technology yields insights that could take oligonucleotides beyond rare diseases. By effectively answering questions about a drug’s safety and efficacy before it is tested in humans, the technology will enable the company to enter the therapeutic arena for rare, ultra-rare and common diseases, he said.
Creyon joined more and more companies appear Artificial Intelligence and Machine Learning Technologies intend to make Drug research is faster and more efficientBut Hart’s goal is what they have so far failed to achieve or claim: getting the treatment to the clinic without first testing it in animals. The FDA requires toxicity studies in animals to support investigational new drug applications. But Hart said Creyon’s technology will answer those toxicity questions without the need for those studies. Mukhopadhyay added that this approach provided a “completely predictable and knowable safety profile,” so when the Creyon drug entered the body, the results weren’t surprising.
It’s not entirely clear whether the FDA will accept this argument. But Hart said that if Creyon proves the platform can generate safety and toxicity data without the need for animal testing, a regulatory framework could be devised to support it. He added that many drugs tested in animals have failed successfully in humans.
“Current practices are not conducive to making safe medicines for people,” Hart said. “We are confident we can do better.”
Photos by Flickr users Blake Burckhardt through Creative Commons license
