Replicate Bioscience Shares Promising Phase 1 and Preclinical Data on Self-Replicating RNA at 2024 ASGCT Meeting

Replicate Bioscience, a clinical-stage company based in San Diego, is at the forefront of developing innovative self-replicating RNA (srRNA) technology aimed at treating a variety of infectious diseases, cancers, and autoimmune disorders. Recently, at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Baltimore, the company unveiled promising preclinical and interim clinical trial data for its ongoing Phase 1 study of the RBI-4000 Vaccine.

Nathaniel Wang, Ph.D., CEO of Replicate, highlighted the advancements presented in the srRNA technology. "Our clinical data show that protective immunity can be achieved at much lower doses compared to existing mRNA or srRNA vaccines. This potency and our strong safety profile indicate that our technology could significantly broaden the applications of RNA-based vaccines," said Wang. He also noted that improvements in their vector library now allow for prolonged in vivo production of therapeutic proteins, surpassing the capabilities of circular RNA, linear mRNA, and current srRNA technologies, thereby opening new therapeutic possibilities in immunology and metabolic diseases.

Replicate's Chief Medical Officer, Zelanna Goldberg, M.D., presented the results of the Phase 1 trial of RBI-4000 on May 11. The trial, titled "Single and Low Dose Self-Replicating RNA Vaccine Provides Effective Immune Protection Against Rabies in Healthy Volunteers," revealed several key findings:
- By Day 85, all dose cohorts met the WHO-established surrogate of protection at doses significantly lower than other RNA-based vaccines.
- A substantial majority of participants across all dose cohorts achieved this protective metric.
- In the 10 mcg dose cohort, 100% of participants met the surrogate of protection after two doses, and 92% did so after just one dose.

The interim safety data were equally promising, showing that RBI-4000 was well tolerated with no severe adverse events across all cohorts. Reactogenicity was transient and self-limiting, and no dose-limiting toxicity was noted. The maximum tolerated dose has not yet been reached, allowing for further dose escalation.

On May 9, Parinaz (Paris) Aliahmad, Ph.D., Head of Research and Development at Replicate, delivered another presentation at ASGCT titled "Novel Self-Replicating RNA Vectors Broaden Therapeutic Window and Expand Use Outside of Vaccines." This presentation emphasized the broad potential of srRNA technology across various disease areas. Key points included:
- Vaccines using Replicate's optimized vectors achieve protective immunity at ultra-low doses (as low as 1 picogram) with minimal reactogenicity.
- Beyond vaccines, these novel srRNA vectors demonstrated over 100-fold increased protein expression and improved durability compared to other RNA technologies.
- The srRNA vectors have the potential to expand RNA technology applications for expressing biotherapeutic proteins, which could be pivotal in treating immune disorders, metabolic diseases, and cancers.

Replicate Bioscience is an Apple Tree Partners portfolio company, committed to advancing RNA therapeutics by overcoming the limitations of existing mRNA approaches. Their srRNA technology promises improvements in bioactivity at lower doses, more robust and durable immune responses, and better tolerability. The company's viral vector library is designed to drive sustained protein expression, significantly outperforming linear mRNA. This positions Replicate uniquely to broaden the reach of RNA treatments in infectious diseases, immuno-oncology, autoimmune diseases, and beyond.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!