Ahead of the Curve
ORNA’S APPROACH & STRATEGY
Orna Therapeutics is leaving linear thinking behind to create fully engineered oRNATM circular RNAs – an entirely new class of RNA medicines that can realize the full potential of RNA and which is poised to change the way we treat disease. By marrying the power of oRNATM with superior delivery techniques, we are pioneering the next breakthrough in RNA therapeutics.
LIMITLESS POTENTIAL
Orna’s technology has a breadth of applications across multiple disease areas including cancer, regenerative medicine, protein replacement, infectious diseases, and autoimmunity. Our lead program is an in situ CAR therapy that combines oRNATM molecules and proprietary lipid nanoparticles (LNPs) to deliver chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (isCARTM therapy).
Our Team
Creating a new class of therapeutics requires thinking outside the lines. Comprised of a team of skilled scientists, molecular engineers, and industry veterans in addition to a world class Board, we are well-suited for the challenge of bringing the promise of our oRNATM therapeutics to patients.
Tom is a veteran entrepreneur who has turned creative scientific visions into successful businesses for both startups and established organizations for over 20 years and has co-founded or helped launch several companies, including Intellia Therapeutics and Eleven Biotherapeutics.
While at Intellia Therapeutics, a CRISPR/Cas9 company which Tom joined as Chief Scientific Officer, he helped build the research team, raise funds (including an IPO), and provide a vision for technology extension. He played a significant role as the corporate spokesperson to lay and industry press, and to governmental and non-governmental organizations on the rapidly evolving and intensely followed CRISPR landscape. Tom has wide-ranging knowledge of biological systems through his work across diverse platforms including gene editing, genomics and gene discovery, small molecule drug repositioning, and protein engineering. In addition to Tom’s many accomplishments he has also survived a shark tank!
He received his PhD from the University of Cambridge and completed research fellowships at Harvard Medical School and McGill University. He is currently also an Executive Partner at MPM.
Nelson serves as Senior Vice President, Platform at Orna, where he leads our efforts to develop highly stable, long lasting circular RNA.
Nelson is a seasoned biotech veteran with over 15 years of experience focusing on developing RNA-based medicine. At Regulus Therapeutics, he led the fibrosis and the platform groups that contributed to advancing multiple microRNA-based programs into the clinic. One of the candidates, lademirsen, is in a phase 2 trial for the treatment of Alport Syndrome. At TranslateBio, Nelson was Vice President of Drug Discovery where he led the effort to identify and validate long non-coding RNA targets. Prior to joining Orna Therapeutics, he served as Vice President of Discovery and Translational Research at LogicBio Therapeutics. Nelson was responsible for building out the research team and leading preclinical research of multiple AAV-mediated gene editing programs. The most advanced program, LB-001, is currently being evaluated in pediatric methylmalonic acidemia patients.
Nelson received his PhD from the Johns Hopkins University and was a Damon Runyon Fellow at University of California San Diego. He is an author of over 50 peer-reviewed publications, patents and patent applications. Along with academia, Nelson enjoys being out on the water. One time while fishing, he experienced a breaching whale very close by!
Michael is Orna’s Head of Facilities & Operations, leading the charge on the company’s Real Estate, Facilities & Laboratory Operations, Security, EH&S, Capital Projects & Engineering.
Bringing more than a decade of operational and facility experience to Orna, his prior life science experience includes ARIAD Pharmaceuticals, Takeda and, most recently, Genocea Biosciences, where he held increasing roles of leadership within the Operations department and the greater organizations. Michael has a passion for developing and mentoring those who work for him.
Michael received his bachelor’s degree in Marine Safety & Environmental Protection from the Massachusetts Maritime Academy. When he’s not hard at work, he enjoys spending time with his wife and 4 children, and his family hopes to welcome another child through adoption soon.
Claudia serves as Orna’s Senior Vice President, Preclinical Safety.
She is a recognized biotechnology leader who built and managed high-performing teams supporting preclinical laboratory operations for Amgen and Biogen, as well as preclinical development for Avrobio, where she was most recently Vice President Preclinical Development. She is a relationship builder, proactive problem solver, and adaptable, result oriented team member with a demonstrated ability to identify, plan, and successfully execute cross organizational project initiatives across R&D as well as develop and manage internal and external strategic alliances.
Her experience spans from being accountable for laboratory management, assay development, preclinical strategy, non-GLP and GLP study design and analysis, and compliance to support drug discovery, early through mid-stage clinical drug development. Her industry exposure includes small molecules, replacement proteins, gene therapy, and biologics in lysosomal storage diseases, immunology, hemophilia, and neurology.
Claudia completed a Doctor of Veterinary Medicine (DVM) degree from Tufts University and a Postdoctoral Associate in Biomedical Research at the Division of Comparative Medicine at Massachusetts Institute of Technology (MIT). She is certified as Diplomate of the American College of Laboratory Medicine (DACLAM) as well as Diplomate of the American Boards of Toxicology (DABT). She discovered, characterized, and named Helicobacter cetorum, co-authored peer-reviewed papers, published a book and several book chapters. In her spare time she loves to spend time with her family, ski, fly fish, bike, hike and paint.
Daniel G. Anderson is a Professor in the Department of Chemical Engineering, the Institute for Medical Engineering and Science, the Koch Institute for Integrative Cancer Research, and the Harvard-MIT Division of Health Science and Technology at MIT. The research done in Prof. Anderson’s laboratory is focused on developing new materials for medicine. He has pioneered the development of smart biomaterials, and his work has led to advances in a range of areas, including medical devices, cell therapy, drug delivery, gene therapy and material science. Prof. Anderson received a B.A. in mathematics and biology from the University of California at Santa Cruz and a Ph.D. in molecular genetics from the University of California at Davis. His work has resulted in the publication of over 400 papers, patents and patent applications. These advances have led products that have been commercialized or are in clinical development, as well as to the foundation of companies in the pharmaceutical, biotechnology, and consumer products space. Dr. Anderson is a founder of Living Proof, Olivo Labs, Crispr Therapeutics, Sigilon Therapeutics, Verseau Therapeutics, VasoRx, and Orna.
Tom is a veteran entrepreneur who has turned creative scientific visions into successful businesses for both startups and established organizations for over 20 years and has co-founded or helped launch several companies, including Intellia Therapeutics and Eleven Biotherapeutics.
While at Intellia Therapeutics, a CRISPR/Cas9 company which Tom joined as Chief Scientific Officer, he helped build the research team, raise funds (including an IPO), and provide a vision for technology extension. He played a significant role as the corporate spokesperson to lay and industry press, and to governmental and non-governmental organizations on the rapidly evolving and intensely followed CRISPR landscape. Tom has wide-ranging knowledge of biological systems through his work across diverse platforms including gene editing, genomics and gene discovery, small molecule drug repositioning, and protein engineering.
He received his PhD from the University of Cambridge and completed research fellowships at Harvard Medical School and McGill University. He is currently also an Executive Partner at MPM.
After earning a degree in psychology in Hamburg, Thomas began his career with Reemtsma in 1987, where he served as the product manager responsible for the “West” brand, among other duties. In 1991, he moved to Pepsi-Cola Germany, where he served as a marketing manager. In 1993, he was appointed Marketing Director for the entire beverage portfolio. Starting in 1994, he was National Sales and Franchise Director for the retail and restaurant sales of Pepsi-Cola. In 1996, he was appointed General Manager of Pepsi-Cola Germany.
In 1997, Thomas moved to Novartis, where he served as General Manager of Novartis Nutrition for Germany and Austria. In 1998, he assumed worldwide leadership of the Novartis Nutrition Division. In this position, he headed the three business units Medical Nutrition, Health Nutrition and Consumer Retail Brands, where, among other duties, he was responsible for the leading U.S. baby food brand Gerber. In 1998, he was appointed to the Novartis Executive Committee. From 2000 to October 2007, he served as CEO of the global Novartis pharmaceuticals business. Under his leadership, the company’s revenues more than doubled from US $11 billion to US $22.6 billion, after which he was CEO of the Novartis Consumer Health Division from 2007 to 2009.
From 2009 to 2018, Thomas served as CEO of ProSiebenSat.1 Media SE, and has been a member of the supervisory board of Bayer AG since 2012 as well as a member of board of directors at Sivantos (Hearing Aids) since 2016 and GFK since 2017. Thomas is on the Board of Directors of Cullinan and is a member of the Advisory Board of the UBS Oncology Impact Fund by MPM Capital.
Ansbert is co-founder and Managing Director of MPM and its venture capital activities, as well as an investment committee member of MPM’s impact collaboration with UBS that is a public/private crossover vehicle managed by MPM. He and his partner, Luke Evnin, PhD, founded MPM in 1997. Ansbert and the MPM team have been the inspiration and driving force behind building leading biopharmaceutical companies such as BioMarin Pharmaceuticals, Idenix Pharmaceuticals (acquired by Merck & Co.), Mitobridge (acquired by Astellas), Pharmasset (acquired by Gilead Sciences) and Radius Health. MPM believes that these companies, in which Ansbert was the lead investor and served on the boards, are some of the biggest successes in biotech history – companies that ultimately resulted in helping thousands of patients live longer and vastly improved lives. Prior to launching MPM’s venture investing activities, Ansbert led MPM’s Advisory and investment business from 1992 to 1997, and prior to that he was at The Boston Consulting Group in their Boston office.
Both Ansbert and Luke Evnin are recipients of the 2017 Global Oncology Visionary Award. Ansbert is active in guiding MPM portfolio companies and serves as Chairman of the Board at Cullinan Oncology, NextPoint Therapeutics, Orna Therapeutics and TCR2 Therapeutics, and he serves as a Board Director at ElevateBio and iTeos Therapeutics. He is also a member of the Harvard Medical School Board of Fellows and the Research Advisory Council of Massachusetts General Hospital. Ansbert received an MD from JW Goethe University in Frankfurt and held research positions at the Whitehead Institute for Biomedical Research at the Massachusetts Institute of Technology and the Biochemistry Department at Harvard University. While at the German Cancer Research Center, he focused on HPV16 and 18 in Professor Harald zur Hausen’s group (Nobel Laureate in Physiology or Medicine, 2008).
NEWS & EVENTS
Orna Therapeutics Unveils Progress of Circular RNA Platform and Improvements in Lead Program ORN-101 at PEGS and ASGCT
ASGCT: in Situ CAR Therapy Using oRNA Lipid Nanoparticles Regress Tumors In Vivo
PEGS: In situ CAR Therapy Using oRNA
ORN-101, an optimized development-stage in situ CAR program, shows tumor eradication in a mouse model at doses far lower than previously reported results
Orna’s FoRCE screening platform has enabled the identification of oRNAs that drive high protein expression, creating broad value across all of Orna’s programs
CAMBRIDGE, Mass., May 18, 2023 — Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA (oRNA) therapies, presented data on the progress of lead program ORN-101, a development-stage in situ CAR program, at the Protein Engineering Summit (PEGS) and at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Los Angeles.
The presentations include a session and talk at PEGS, as well as a poster presentation at ASGCT, which may be viewed on the Orna website here.
“Using the Orna FoRCE platform, we’ve been able to develop ORN-101, a potent anti-cancer circular RNA therapy delivered via LNP,” said Robert Mabry, PhD, Chief Scientific Officer at Orna. “We have achieved 20-fold lower dosing in animal models compared to earlier versions of the product, allowing us the flexibility to administer multiple doses of our off-the-shelf immunotherapy – something that could allow us to treat more patients quickly and easily.”
Synthetic Circular RNA as a New Therapeutic Modality
Elevated and durable protein expression – two features where mRNA falls short – are maximized with circular RNA in part due to Orna’s discovery of novel internal ribosome entry site (IRES) elements necessary for translation. Compared to previously known IRES elements, Orna’s library of IRES elements can be used to drive higher protein expression in desired cell type targets. oRNA, because of its shape, is more resistant to quick degradation in the body, is easier to manufacture and formulate in the lab, and is immunoquiescent. These desirable features further allow oRNA therapeutics to be developed for applications beyond infectious disease, including oncology and genetic disorders.
In situ CAR Therapy Using oRNA
Over the past four years, Orna has worked to develop a therapeutic class capable of delivering a chimeric antigen receptor (CAR) to immune cells within a patient, eliminating the need for lymphodepletion typically required for engineered cell therapies, while providing off-the-shelf redosability in an autologous setting. ORN-101 combines oRNA and a proprietary LNP, and these latest data show ORN-101’s tumor eradication ability after 2-3 doses in a mouse model, effective at 10-20-fold lower doses than Orna has previously reported. ORN-101 features high expression of the CAR driven by an optimized IRES element, selected by Orna’s FoRCE platform to yield durable protein expression.
About ORN-101:
ORN-101, Orna’s lead program, is a development-stage in situ CAR therapy designed to modify a patient’s immune cells inside their body. Comprising an oRNA molecule packaged inside a proprietary lipid nanoparticle (LNP) formulation, this easily redosable format could avoid patient lymphodepletion and allow for reliable dose control, overcoming barriers of existing autologous ex vivo CAR-T therapies without sacrificing efficacy. Preclinical data demonstrates tumor suppression and eradication in animal models, suggesting the possibility that oRNA-LNP based cancer therapies could disrupt traditional CAR-T cellular therapies.
About Orna Therapeutics:
Orna Therapeutics is reshaping RNA therapeutics to open a new world of treatment possibilities. Orna’s circular RNA (oRNA) is created in one step by self-circularization of an engineered linear RNA. In this form, oRNA enjoys unmatched advantages over traditional linear mRNA therapies including simplified production, increased protein expression, easier encapsulation into lipid nanoparticles (LNPs), and immunoquiescence. Orna’s proprietary LNPs deliver the oRNA to specific areas of the body, such as immune cells. Founded in 2019 based on groundbreaking research from MIT, Orna was built by MPM Capital and its affiliate BioImpact Capital. To learn more visit: www.ornatx.com and follow us on Twitter and LinkedIn.
Media Contact
Peg Rusconi, Verge Scientific Communications
prusconi@vergescientific.com