Raina Biosciences Inc., an MIT spinout company, announced the publication of breakthrough data from its generative AI platform GEMORNA in the journal Science, demonstrating significant advances in mRNA therapeutic design and optimization. The platform represents the world's first generative AI system purpose-built for mRNA design, showing state-of-the-art performance across diverse therapeutic applications.
Revolutionary AI Platform Demonstrates Superior Performance
The Science article, titled "Deep generative models design mRNA sequences with enhanced translational capacity and stability," presents data supporting GEMORNA's ability to generate novel mRNA sequences with superior drug properties compared to existing discovery methods. The platform addresses two major limitations that have constrained mRNA-based therapeutics: suboptimal expression levels and insufficient durability.
GEMORNA distinguishes itself from prior RNA language models by directly designing novel sequences from a near-infinite design space, rather than focusing solely on predictive tasks. This approach enables the platform to create both linear and circular mRNA drug molecules with optimized therapeutic profiles.
Impressive Therapeutic Performance Across Applications
The published data reveals remarkable performance improvements across multiple therapeutic areas. In vaccine applications, GEMORNA-generated mRNAs elicited significantly higher immune responses in mice compared to leading commercially available mRNA vaccine sequences, potentially supporting therapeutic vaccine development for diseases including cancer.
For gene therapy applications, the platform achieved up to a 150-fold increase in human erythropoietin (hEPO) expression compared to optimized benchmarks. This dramatic improvement could significantly enhance mRNA medicines for gene therapy applications.
Perhaps most notably, in CAR-T cell therapy applications, GEMORNA demonstrated a 5-fold increase in CD19 CAR expression and a 2-fold improvement in durability compared to patented benchmarks. These enhancements resulted in nearly 100% anti-tumor efficacy in primary human T cells, potentially supporting mRNA therapies for in vivo CAR-T applications.
Expert Leadership and Strategic Vision
"Raina's GEMORNA platform is built upon a decade of the team's foundational work in synthetic biology and artificial intelligence," said Jicong Cao, Ph.D., Chief Executive Officer and co-founder of Raina Biosciences, who also served as corresponding author of the Science paper. "We are excited to work with industry-leading pharma and biotech companies to expand the usage of mRNA-based therapeutics while we prepare to build an internal pipeline."
Timothy Lu, M.D., Ph.D., Raina's Chairman of the Board and former MIT faculty member, emphasized the platform's transformative potential: "Raina has the potential to transform mRNA therapeutics by rapidly and reliably generating novel sequences with greater performance, precision and efficacy. The GEMORNA platform could be a sea change for biopharma companies pursuing mRNA-based medicines beyond traditional infectious disease vaccines ranging from neoantigen cancer vaccines, in vivo CARs, to gene editing or gene therapy applications."
Strong Foundation and Financial Backing
The Boston-based company brings together leaders in mRNA and AI with proven track records in biotechnology. Co-founder and Chief Technology Officer He Zhang, Ph.D., who served as first author of the Science paper, previously worked as a Senior Staff Scientist at Baidu Research. Chief Corporate Development Officer Joel Edwards contributes over 25 years of leadership experience in corporate strategy and deal-making, including his tenure as Vice President of Corporate Strategy at Ionis Pharmaceuticals.
The company benefits from scientific guidance by Jeff Coller, Ph.D., Bloomberg Distinguished Professor and Inaugural Director of the RNA Innovation Center at Johns Hopkins University. Raina successfully closed a $5.7 million angel round upon company formation, providing resources to advance its platform and establish industry partnerships.
Accelerating Drug Discovery Timelines
GEMORNA's efficiency in designing optimized mRNA sequences represents a significant advancement in drug discovery acceleration. By eliminating the need for iterative sequence optimization, the platform can significantly reduce development timelines while opening new therapeutic areas for mRNA applications.
The company is actively partnering with leading pharmaceutical and biotech companies to create next-generation mRNA-based therapeutics, positioning itself to transform the broader mRNA therapeutics landscape through AI-driven innovation.
