Circio (CRNA) R&D Update summary

Market and Technology Landscape

• Circular RNA is a novel therapeutic field with major pharma interest, validated by high-value deals such as Vertex licensing ORNA's LNP-circRNA platform for up to $700M and Novartis acquiring Kate Therapeutics for $1.1B, with deal values up to $3.5B.

• Circio’s technology is unique, focusing on DNA and viral vectors to express circular RNA, differentiating it from competitors.

• The company has increased visibility through scientific presentations, publications, and media coverage, including features in Nature Reviews Genetics.

Technology and R&D Update

• circVec 3.0 platform validated in multiple vector formats, showing a 27x improvement in vitro over the first generation and robust performance in therapeutic applications.

• LNP delivery collaboration achieved efficient, durable spleen targeting, surpassing mRNA vectors and enabling new cell therapy opportunities.

• AAV-based circVec vectors show up to 50% higher protein expression than mRNA vectors after 90–106 days in vivo, with some reports of 3-5x enhanced potency and durability.

• Multiple in vivo experiments and collaborations are ongoing, with interim data expected in Q2 and further updates planned for 2025.

• R&D priorities include optimizing circVec 3.0, expanding patent filings, and seeking partnerships with gene therapy companies.

Collaborations and Business Development

• Active collaborations with Certest, 4basebio, and four undisclosed partners to optimize delivery and expand applications.

• Several delivery technologies are being tested, including LNPs and novel chemistries for spleen targeting.

• Ongoing discussions with pharma and gene therapy companies, with potential for deeper partnerships if data is positive.

• TG cancer vaccine program TG01 is in clinical trials for RAS-mutated pancreatic, lung cancer, and multiple myeloma, supported by research grants and collaborative networks.

• Dialogue with IOVaxis in China is ongoing, contingent on their fundraising to activate a license agreement.