Envisagenics Awarded $1.5M to Refine SpliceCore, Platform Targeting Protein Production and Ways of Preventing Errors

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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Envisagenics announced that it has been given a two-year, $1.5 million grant from the National Institutes of Health (NIH) to further advance SpliceCore, a new biomarker and drug discovery platform.

The award, a Small Business Innovation Research (SBIR) Phase 2 grant, will help Envisagenics in further developing SpliceCore, a cloud-based drug discovery platform that uses machine learning and artificial intelligence to analyze ribonucleic acid (RNA) sequencing data. Such data is important to the discovery of new RNA-targeted treatments.

RNA is the in-between molecule in the process that reads the information in DNA to generate a functional protein. Errors in RNA splicing that affect protein development are a cause of many genetic diseases, including spinal muscular atrophy (SMA).

Envisagenics was awarded a $225,000 Phase 1 SBIR grant in 2015 to develop an infrastructure capable of analyzing significant amounts of RNA sequencing data in a scalable manner. That first grant was also used to help build the largest database to date of RNA splicing events, and to develop machine learning algorithms to prioritize splicing errors relevant to diseases like SMA.

The Phase 2 grant will allow the company to expand the knowledge base and predictive functions of its SpliceCore platform, which focuses on RNA splicing.

“The discovery of disease-causing proteins was at the center of pharma innovation for decades, but the new century brought us not only better knowledge of genetic information but also the computer power to interpret it,” Martin Akerman, co-founder and chief technology officer of Envisagenics, said in a press release. “The RNA splicing treatments that we develop target the flow of genetic information, so disease-causing proteins cannot be formed in the first place.”

Work supported by this SBIR Phase 2 grant will take place at Cold Spring Harbor Laboratory, under the leadership of Dr. Adrian Krainer, a specialist in RNA splicing, and at Rockefeller University under Dr. Thomas Tuschl, professor and head of the laboratory of RNA molecular biology.

Envisagenics was founded in 2014 as a spin-off company from the Cold Spring laboratory,  with a goal of developing treatments through RNA splicing analytics and  artificial intelligence (AI).

“We’re excited for the next stage of growth for the company to leverage the power of AI and RNA sequencing data internally and in collaboration with biopharma partners to unlock new treatments,” said Maria Luisa Pineda, company co-founder and chief executive officer.