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Envisagenics and Biogen partner for RNA splicing research

photo of Maria Luisa Pineda working in a lab
Maria Luisa Pineda working at Cold Spring Harbor Laboratory in 2010. She is now the chief executive officer of Envisagenics.

The Cold Spring Harbor Laboratory (CSHL) spin-out company Envisagenics and Biogen Inc. announced a new collaboration on May 13, 2021. This collaboration aims to advance RNA research in central nervous system diseases by leveraging the Envisagenics artificial intelligence–based platform called SpliceCore®.

Genes, encoded in DNA, are transcribed into the related language of RNA. RNA is then translated into proteins. In many cases, proteins are encoded in RNA segments that need to be pieced—or spliced—together in the correct order. Incorrect RNA splicing can lead to disease, such as spinal muscular atrophy. Envisagenics uses its proprietary algorithms to identify potential RNA splicing errors and design potential therapeutics to fix them.

Biogen is a global biotechnology company that pioneers treatments for neurological diseases such as multiple sclerosis, spinal muscular atrophy, and Alzheimer’s. The collaboration allows Biogen to identify, test, and validate many splicing errors at a time.

The SpliceCore® platform was developed by Martin Akerman when he was a postdoc in CSHL Professor Adrian Krainer’s laboratory. Biogen and Ionis Pharmaceuticals collaborated with Krainer’s team to release Spinraza®, the first FDA-approved treatment for spinal muscular atrophy, in 2016. Akerman, now co-founder and chief technology officer of Envisagenics, is using SpliceCore® to discover splicing errors and design treatments for other RNA splicing-related diseases. So far, the Splicecore® platform database has recorded approximately 7 million potential RNA splicing errors.

“The technology behind the SpliceCore® software platform has been in continuous development since it was created at CSHL, in part to help advance our research on Spinraza® almost ten years ago,” Krainer says. “SpliceCore® will now propel Envisagenics’ and Biogen’s joint efforts on central nervous system diseases. As a member of Envisagenics’ scientific advisory board, I am very excited about this collaboration.”

Maria Luisa Pineda, chief executive officer of Envisagenics and CSHL School of Biological Sciences alum says, “Envisagenics is thrilled to work with Biogen because we share a commitment to identifying potential treatments for central nervous system diseases through innovative artificial intelligence technology like the SpliceCore® platform.”

Written by: Luis Sandoval, Content Developer/Communicator | sandova@cshl.edu | 516-367-6826

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