Replicate Bioscience, Inc.

Ascidian Therapeutics CEO Michael Ehlers is leaving the venture investor that serves as his home base and will join MPM BioImpact as an entrepreneur-partner on Monday, the investment firm told Endpoints News . Ehlers will help MPM create new startups, find investment opportunities and assess clinical development, regulatory and commercial strategies at its portfolio biotechs. For the past five years, Ehlers has been chief scientific officer at venture investor and biotech incubator Apple Tree Partners, better known as ATP. He also helped found multiple ATP portfolio companies, including the RNA biotech Ascidian. He’ll remain in the CEO role at Ascidian , which in January became the first company to get FDA clearance for human trials of RNA editing. MPM is not an investor in Ascidian, Ehlers confirmed. “Something I’ve always been impressed by at MPM from the outside is just the depth and rigor of the science, the really critical eye toward the future: in other words, the kinds of technologies and programs that are going to matter, not just today, but five to 10 years down the road,” Ehlers said in an exclusive interview. He said he’s excited by computational-based methods that are spreading across the industry, RNA biology and RNA modality advancements, improvements in gene editing and the convergence of technologies. New approaches to “classic areas” like small molecule drug discovery are also intriguing, Ehlers said. “It’s just a smorgasbord of things to be able to do, and MPM is just a perfect venue or a perfect platform to be able to do it,” Ehlers said. “There is so much opportunity,” Ehlers said. “It’s a little bit like walking to the Old Country Buffet and trying to figure out which flavor of pudding and Jello you’re going to get when there’s too much.” Ehlers was a neurobiology professor at Duke for about a dozen years and then an investigator at Howard Hughes Medical Institute before switching to industry in 2010, when he joined Pfizer. He eventually became the pharma giant’s chief scientific officer for neuroscience and then was executive vice president of R&D for Biogen until 2019. At ATP, he helped form Ascidian, Aulos Bioscience , Replicate Bioscience and Intergalactic Therapeutics , a non-viral gene therapy biotech that shut down last year. MPM said Ehlers will continue serving on the boards of Aulos and Replicate. Ehlers joins MPM after a string of recent financings, including Carolyn Bertozzi-associated ADC biotech Firefly Bio and Alzheimer’s startup AstronauTx . Neuroscience continues to intrigue him. “It is an area with lots of opportunity and it remains one of the big submerged icebergs,” Ehlers said. “There’s massive unmet medical need in that area, and that’s not going away anytime soon.”

SAN DIEGO, Aug. 21, 2024 /PRNewswire/ -- Replicate Bioscience, a clinical-stage company pioneering novel self-replicating RNA (srRNA) technology for applications across infectious disease, immunology, immuno-oncology and beyond, will be participating in the following upcoming investor conferences: Morgan Stanley 22nd Annual Global Healthcare Conference in New York, N.Y. Dates: September 4-6, 2024 Fireside chat to be held Friday, September 6 at 10:45 a.m. EDT Replicate participants: Nathaniel Wang, Ph.D., CEO; Rachael Lester, CBO Cantor Global Healthcare Conference in New York, N.Y. Dates: September 17-19, 2024 Replicate participants: Nathaniel Wang, Ph.D., CEO; Rachael Lester, CBO Replicate leadership will also present at the following upcoming scientific meetings: RNA Leaders USA Congress in San Diego, Calif. Keynote panel title: Scaling RNA Therapies to Deliver on the Promise of Precision Medicine Presenter: Nathaniel Wang, Ph.D., CEO Date and time: Wednesday, September 4, 2024 at 9:55 a.m. PDT Presentation title: Next-Generation Self-Replicating RNA Drives Major Improvements to Clinical Bioactivity while Retaining a Strong Safety Profile Presenter: Nathaniel Wang, Ph.D., CEO Date and time: Wednesday, September 4, 2024 at 4:25 p.m. PDT 3rd Non-Viral RNA Delivery Systems Summit in Boston, Mass. Presentation title: LNP & Polymer Delivery of Self-Replicating RNA: Unlocking the Potential of Low Dose RNA Vaccines & Gene Therapy Presenter: Andy Geall, Ph.D., CDO Date and time: Thursday, October 3, 2024 at 9:30 a.m. EDT About Replicate Bioscience Replicate Bioscience 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, induction of more potent immune responses, and improved tolerability. RBI-4000, the company's clinical-stage srRNA rabies vaccine, has demonstrated protective levels of immunity at doses lower than any other reported mRNA or srRNA vaccine in Phase 1 trials. Funded by Apple Tree Partners, Replicate is advancing a robust pipeline of srRNA vaccines and therapeutics, fueled by its proprietary library of customizable viral vectors. With deep expertise in srRNA and end-to-end development capabilities, Replicate is uniquely positioned to expand the reach of RNA treatments toward widespread use in infectious disease, immunology, immuno-oncology and more. Visit us at . SOURCE Replicate Bioscience

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 . SOURCE Replicate Bioscience