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Start-Up Licenses Gene-Edited Drug Discovery Tech.

African clawed frog

African clawed frog (Brian Gratwicke, Flickr.

27 Sept. 2022. A new company is acquiring a technology using genome-edited tadpoles that finds current drugs addressing precise genetic conditions underlying rare diseases. Unravel Biosciences, a one year-old enterprise in Boston, is licensing the technology from the Wyss Institute for Biologically Inspired Engineering, a research center at Harvard University.

The technology called CogniXense is developed by researchers at Wyss Institute and Tufts University, and seeks to discover drugs meeting a rare-disease patient’s unique medical conditions, defined by symptoms and RNA transcribed from that person’s genetic code. The technology first creates computational models of the disease focusing on anomalies in the RNA transcribed from a patient’s genes. Next, CogniXense extends those algorithms to screen currently approved drugs with potential to address the identified transcription anomalies for restoring health to the patient.

The technology then uses the genome editing technique Crispr to genetically engineer tadpoles from the Xenopus laevis or African clawed frog species, to reflect the patient’s symptoms and genetic conditions. African clawed frogs are a common model organism and widely studied in biology labs because of their rapid development cycle. In this case, the tadpoles’ response to the identified drugs are observed and assessed in neurological tests, such as ability to learn and navigate simple mazes. CogniXense also tests for possible toxicities.

The Wyss Institute researchers, led by Richard Novak, then a staff engineer, applied CogniXense to Rett Syndrome as a test case. Rett syndrome is a genetic neurological disorder affecting girls caused by a mutation in the MECP2 gene. The disease occurs in 1 in 9,000 to 10,000 female births, with symptoms somewhat similar to autism, including language and communication problems, learning difficulties, and lack of coordination. The MECP2 gene gives instructions for making a protein needed to maintain synapses or connections between neurons in the brain.

“Computationally predict which existing drugs could restore health.”

The Rett syndrome case shows the CogniXense algorithms can replicate behavioral differences between healthy individuals and those with Rett syndrome, as well as identify transcription characteristics of drug candidates for reversing symptoms of the disease. When tested with genetically edited tadpoles to display Rett syndrome characteristics, CogniXense identified several drugs that reverse symptoms of the disease.

Harvard University is granting Unravel Biosciences an exclusive license to apply and commercialize the technology for prevention, diagnostics, and treatment of specified but undisclosed neuro-developmental disorders, with new and current drugs discovered by the technology. Financial details of the license agreement were also not disclosed.

Novak founded Unravel Bio in Sept. 2021 with Wyss Institute director Donald Ingber, staff scientist Frederic Vigenault, and Tufts biology professor Michael Levin, also an associate faculty member at Wyss Institute. Novak and Vigenault are the company’s CEO and chief scientist respectively, while Ingber and Levin are advisors to the company.

“We computationally predict which existing drugs could restore health in that patient,” says Novak in a Wyss Institute statement, “validate those drugs in our engineered animal models and focused clinical trials, and from there identify the underlying molecular targets that could be drugged to treat the disease across patient populations.”

Unravel Bio now calls its platform BioNav, with algorithms trained by more than 12,000 gene interactions and some 40,000 drug compounds. The company says it can produce Crispr-edited tadpole models of disease and screen for relevant solutions in about three weeks.

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