Positive data from Phase 1 trial of RBI-4000
vaccine achieve WHO-established surrogate of protection across all
dose cohorts
Vector improvements demonstrate broad
potential of Replicate's srRNA for expanded indications in complex
infectious disease and in situ expression of therapeutic
proteins
SAN
DIEGO, May 11, 2024 /PRNewswire/ -- Replicate
Bioscience, a clinical-stage company pioneering novel
self-replicating RNA (srRNA) technology for use across a range of
infectious disease, oncology, autoimmune disease indications and
beyond, presented new preclinical data and today will share interim
clinical trial results from an ongoing Phase 1 study at the
American Society of Gene & Cell Therapy (ASGCT) Annual Meeting,
May 7-11 in Baltimore, Maryland.
"At ASGCT, Replicate is presenting two important breakthroughs
in srRNA technology," said Nathaniel
Wang, Ph.D., CEO of Replicate. "For vaccines, our clinical
data demonstrate the induction of protective levels of immunity at
doses significantly lower than any other reported mRNA or srRNA
vaccine. This potency, combined with a best-in-class safety
profile, demonstrates the capability of our technology to greatly
expand the utility of RNA technology for vaccines. Beyond vaccines,
further improvements to our library of vectors now enable us to
control and prolong in vivo production of therapeutic
proteins compared to circular RNA, linear mRNA, and current
state-of-the art srRNA, opening up therapeutic areas such as
immunology and metabolic disease."
RBI-4000's Phase 1 results will be presented on Saturday, May 11 at 10:15
a.m. ET by Replicate's Chief Medical Officer, Zelanna
Goldberg, M.D., in an oral presentation titled "Single and Low Dose
Self-Replicating RNA Vaccine Provides Effective Immune Protection
Against Rabies in Healthy Volunteers." The results highlight the
strong immunogenicity and favorable safety profile of RBI-4000 in
the Phase 1 trial, which is the first clinical validation of
Replicate's next-generation srRNA technology. Additional takeaways
are as follows:
- Day 85 datasets for all cohorts met the WHO-established
surrogate of protection at doses significantly lower than any other
reported RNA-based vaccines;
- Substantive majority of participants achieved metric in all
dose cohorts.
- In the previously unreported 10 mcg dose cohorts:
-
- 100% of participants achieved surrogate of protection after two
vaccine doses.
- 92% of participants achieved surrogate of protection after a
single vaccine dose.
- Safety data from the interim dataset demonstrate RBI-4000 was
well tolerated with no severe adverse events across all cohorts;
reactogenicity was transient and self-limiting.
- No dose limiting toxicity (DLT) was observed; maximum tolerated
dose (MTD) has not been reached, enabling further dose
escalation.
On May 9, Parinaz (Paris) Aliahmad, Ph.D., Head of
Research and Development at Replicate, delivered an oral
presentation at ASGCT titled "Novel Self-Replicating RNA Vectors
Broaden Therapeutic Window and Expand Use Outside of Vaccines." The
results underscore the broad potential of srRNA as a new treatment
modality across a wide range of disease areas. Additional takeaways
are as follows:
- Vaccines using Replicate's optimized vectors achieve protective
immunity at ultra-low doses (1 picogram) with minimal
reactogenicity.
- Beyond vaccines, Replicate's novel srRNA vectors demonstrate
>100-fold increased expression of encoded proteins and improved
durability compared to circular RNA, linear mRNA vectors, and
current state-of-the-art srRNA technologies.
- Replicate's novel srRNA vectors can expand utility of RNA
technology for templated expression of biotherapeutic proteins for
applications outside of vaccines, in areas such as immune
disorders, metabolic disease, and cancers.
About Replicate Bioscience
Replicate Bioscience, an
Apple Tree Partners portfolio company, is a clinical-stage company
amplifying the power of RNA therapeutics by pioneering its novel
self-replicating RNA (srRNA) technology to overcome the
shortcomings of existing mRNA approaches, with potential
improvements in bioactivity at lower doses, induction of more
robust and durable immune responses, and improved tolerability.
Replicate's off-the-shelf srRNAs contain two components: virally
derived genetic code to drive controlled and self-limiting
amplification, and the RNA encoding therapeutic proteins. The
company's library of viral vectors, selected for driving robust and
sustained protein expression and orders-of-magnitude improved
performance over linear mRNA, allow for the development of
treatments in applications including oncology, infectious disease,
and autoimmunity. Differentiated by a team of srRNA experts, a
customizable library of synthetic srRNA vectors, and end-to-end
development capabilities, Replicate is uniquely positioned to
finally expand the reach of RNA treatments toward widespread use in
infectious disease, immuno-oncology, autoimmune disease, and more.
Visit us at www.replicatebioscience.com.
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SOURCE Replicate Bioscience