Orna’s Approach & Strategy 
We’re reshaping RNA therapeutics to open an entirely new world of possibilities in the treatment of disease. Our field-defining engineered circular RNA (oRNA®) has unmatched advantages compared to linear RNA, and our groundbreaking LNP delivery to sites other than the liver has led the world in unlocking the treatment of a far greater span of diseases than previously addressable.
Limitless Potential 
Our vision is to deliver first-in-class innovative RNA drugs wherever they’re needed in the body. Initially focusing on oncology and autoimmune diseases (with our panCAR™ in vivo CAR technology) and infectious disease (with our Merck collaboration), our delivery solutions also open up the possibility of treating certain genetic diseases, such as sickle-cell disease and beta-thalassemia, directly in the bone marrow.
Our technology will allow an immediate, off-the-shelf treatment option that will not require harsh preconditioning regimens, offering a significant advancement over traditional cell engineering therapies.
About Us 
Creating a new class of therapeutics requires thinking outside the lines. Comprising a team of skilled scientists, molecular engineers, and industry veterans in addition to a world-class Board of Directors and Scientific Advisors, we are well-suited for to bring the promise of our unique oRNA® and LNP delivery method to patients.
Our Team 
Joe has more than 45 years of industry and research experience. He is an Executive Partner at MPM BioImpact and was formerly Chief Scientific Innovation Officer at ReNAgade Therapeutics and Chief Scientific Officer at PureTech Health. Before joining PureTech, he oversaw all aspects of research and development for Moderna Therapeutics as President of Research & Development and Chief Scientific Officer. Previously, Joe was Chief Scientific Officer at Millennium Pharmaceuticals and Global Head of Oncology Research at Takeda Pharmaceutical Company. He held senior R&D positions at Hoechst Marion Roussel, Schering-Plough (DNAX) and Bristol Myers Squibb. Joe began his career at the National Institutes of Health (NIH), where he contributed to the discovery of a class of proteins known as tyrosine kinase oncogenes as key regulators of the immune system. Joe earned a Ph.D. in Immunology and conducted his postdoctoral training in molecular virology at Kansas State University Cancer Center. He graduated from the University of Nebraska with a B.S. in Microbiology and Chemistry.
Hope joins Orna with more than 25 years of legal experience, with the majority of her career spent in the healthcare industry, and deep knowledge of pharmaceutical, gene therapy and life sciences companies. Most recently, she was General Counsel at Applied Genetic Technologies Corporation (AGTC), where she oversaw legal, IP and compliance. Before her tenure at AGTC, Hope served as General Counsel and Vice President of Administration at Spirovant Sciences, where she was a member of the company’s executive leadership team helping to develop strategy for, and lead operations of, the company. Hope also served as counsel at Reed Smith, LLC, representing biotechnology and pharmaceutical companies in a variety of strategic collaborations, and as Executive-In-Residence at Militia Hill Ventures, supporting the building and growth of successful life sciences companies. Previously, she was Vice President and Associate General Counsel at GlaxoSmithKline, where she supported worldwide business development on numerous strategic collaborations, including licenses, divestments, joint ventures and mergers and acquisitions. Hope earned her J.D. at the University of Pennsylvania Law School and her B.S. in Economics from the University of Delaware.
Ciaran has deep experience in building and leading teams across biotech and consulting sectors. He was most recently Chief Operating Officer of ReNAgade Therapeutics. Prior to ReNAgade, he held a principal leadership role at The Boston Consulting Group where he led large projects / programs across corporate strategy, R&D operations, M&A, and commercial. Ciaran was previously an early scientific lead at Moderna where he helped build out delivery capabilities.
Teresa Lipcsey brings deep and broad experience throughout the organizational life cycle, building high performing finance teams and implementing strategic financial planning, processes and controls. She has a proven track record supporting IPOs and M&A, driving organizational growth, expansion and value. Teresa most recently served as a Vice President of Accounting & Finance at Wave Life Sciences. Before that, she was a Vice President, Corporate Controller at axogen. Previously, Teresa held key financial leadership positions including Vice President of Finance at SomaLogic, Invisible Sentinel and BioBDx (Formerly BioBehavioral Diagnostics Company). She began her professional career at Deloitte.
Teresa holds a Bachelor of Science in Accounting and Finance from the University of Maryland and is a certified public accountant.
Managing Partner, MPM BioImpact
Managing Partner, MPM BioImpact
Ansbert is Chairman of the Board and Co-Founder of Orna, and Managing Director of MPM BioImpact. He founded MPM Capital in 1992 as a biotech venture capital firm and later its affiliate BioImpact Capital for private/public funds. Ansbert and the MPM BioImpact 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, more recently, Cullinan Oncology (NASDAQ: CGEM), ElevateBio, Orna Therapeutics and ReNAgade Therapeutics. MPM BioImpact believes that these companies, in which Ansbert was the lead investor and served on the Boards of Directors, are some of the biggest successes in biotech – companies that ultimately result in helping thousands of patients live longer and vastly improved lives. Prior to founding MPM, Ansbert was at The Boston Consulting Group in its Boston office.
Ansbert’s commitment to drug discovery and curing disease inspires his work both within and outside of MPM BioImpact. In recognition of MPM’s novel work to finance and build companies which may have a significant impact on cancer care and cures globally, Ansbert and Luke Evnin are recipients of the 2017 Global Oncology Visionary Award. He is a member of the Harvard Medical School Board of Fellows and the Research Advisory Council of Massachusetts General Hospital.
Ansbert received an M.D. from J.W. 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). Ansbert has published in leading scientific journals such as Nature and Cell.
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.
David currently serves as Vice President of Cell Biology at Kite Pharma. Prior to joining Kite, he held several translational research positions at Tmunity Therapeutics and spent over sixteen years at the Children’s Hospital of Philadelphia. David holds a PhD in Human/Medical Genetics and an MD from the Virginia Commonwealth University.
Dr. Mike Ehlers is an Entrepreneur Partner at MPM BioImpact. He is currently the CEO of Ascidian Therapeutics, a clinical-stage RNA editing company developing exon editing technology for gene correction in hereditary diseases.
Prior to joining MPM BioImpact, Dr. Ehlers was General Partner and CSO at Apple Tree Partners (ATP) where he was co-founder of Ascidian Therapeutics; Aulos Bioscience, a clinical-stage immuno-oncology company; and Replicate Bioscience, a clinical-stage RNA therapeutics company. Prior to ATP, Dr. Ehlers was Executive Vice President for Research & Development at Biogen with a focus on neurological, immunological, and rare diseases. During his time at Biogen, he advanced 20 novel clinical candidate compounds including overseeing the approval of SPINRAZATM (nusinersen), the first drug approved for spinal muscular atrophy, and VUMERITYTM (diroximel fumarate), the second oral fumarate approved for multiple sclerosis, as well as advancing development programs for currently approved LEQEMBITM (lecanemab), the first fully approved drug for slowing cognitive decline in Alzheimer’s disease, and QALSODYTM (tofersen) the first genetic medicine for ALS.
Previously, Dr. Ehlers was Senior Vice President for BioTherapeutics and Chief Scientific Officer for Neuroscience at Pfizer where he created and advanced the Neuroscience and Rare Disease portfolios, bringing 22 compounds into the clinic as well as directing global development activities, and steering a network of academic collaborations focused on immunology and oncology. He advanced development programs for currently approved BEQVEZTM (fidanacogene elaparovovec) for hemophilia B, one of the first ten viral-based gene therapies approved in any disease. A subset of Dr. Ehlers’ Neuroscience programs formed the basis of the Pfizer spin-out Cerevel Therapeutics.
Dr. Ehlers is the recipient of numerous awards including the 2003 Eppendorf & Science Prize in Neurobiology, the 2007 John J. Abel Award in Pharmacology, the 2007 Society for Neuroscience Young Investigator Award, a NIMH MERIT Award, and the 2009 National Alliance for Schizophrenia & Depression Distinguished Investigator Award. He received the 2008 Breakthrough Research Award of the North Carolina Biotechnology Center, the 2016 Biomedical Research Award of the Massachusetts Medical Society, and was recognized as a 2024 PharmaVoice 100 Biotech Pathfinder. In 2013 he became the 11th recipient of the Thudichum Medal of the Biochemical Society of the United Kingdom. Dr. Ehlers has authored over 100 scientific papers, served on the Editorial Boards of Annual Reviews in Medicine, Annual Reviews in Pharmacology and Toxicology, the Journal of Neuroscience, the Journal of Biological Chemistry, and Molecular and Cellular Neuroscience, and sat on advisory committees of the NIH. He is a member of the Board of Directors of Ascidian Therapeutics, Aulos Bioscience, Replicate Bioscience, and the McKnight Endowment Fund for Neuroscience.
Dr. Elhers earned his M.D. and Ph.D. degrees from the Johns Hopkins University School of Medicine, and his B.S. in chemistry from Caltech.
Dr. Carolyn Bertozzi is a pioneer in emerging technologies who has cofounded 14 biopharmaceutical companies and guided more than a dozen academic and professional organizations and life sciences companies through her leadership and board positions. Her research interests span the disciplines of chemistry and biology with an emphasis on studies of cell surface sugars important to human health and disease.
Dr. Bertozzi is the Anne T. and Robert M. Bass Professor of Chemistry and Professor of Chemical & Systems Biology and Radiology, the Baker Family Director of the Sarafan ChEM-H Institute at Stanford University, and a Howard Hughes Medical Institute Investigator. She was awarded the 2022 Nobel Prize in Chemistry for the development of click chemistry and bioorthogonal chemistry.
An elected member of the National Academy of Medicine, National Academy of Sciences, National Academy of Engineering, and American Academy of Arts and Sciences, Dr. Bertozzi has been awarded the Lemelson-MIT Prize, Heinrich Wieland Prize, ACS Award in Pure Chemistry, Chemistry of the Future Solvay Prize, Wolf Prize in Chemistry, and a MacArthur Foundation Fellowship, among other awards and honors during her distinguished career.
News & Events
Orna Therapeutics Presents New Data Supporting its in vivo CAR Programs for Autoimmune Diseases and Oncology at the 67th American Society of Hematology Annual Meeting
Orna Therapeutics Announces Multiple Presentations Supporting its in vivo CAR Programs for Autoimmune Diseases at the 67th American Society of Hematology Annual Meeting
Orna Therapeutics Announces Encore Presentation at the American College of Rheumatology Annual Meeting Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases
Anti-CD19 panCARTM program (ORN-252) demonstrated robust B cell depletion with significant reduction in anti-dsDNA titers in humanized lupus model
Anti-BCMA panCARTM program showed robust and durable plasma cell depletion in preclinical models
Orna expects to submit its first Clinical Trial Application for ORN-252 by the end of 2025 and anticipates initiating a first-in-human study in early 2026
WATERTOWN, Mass., Dec. 7, 2025 – Orna Therapeutics (Orna), a biotechnology company dedicated to engineering immune cells in vivo to treat autoimmune and oncology diseases, today announced new preclinical data supporting the Company’s in vivo CAR programs to target and treat a broad range of B cell-mediated autoimmune diseases and plasma cell or BCMA-related diseases at the 67th American Society of Hematology (ASH) Annual Meeting taking place in Orlando, Florida from December 6-9, 2025.
“The preclinical data presented at this year’s ASH Annual Meeting further highlights our potent, non-viral, transient, tunable, and scalable approach to in vivo CAR therapies in both autoimmune diseases and oncology,” said Joseph Bolen, Ph.D., Chief Executive Officer of Orna. “Our panCAR platform has demonstrated robust activity in B cell autoimmunity and BCMA-related diseases without the need for preconditioning lymphodepletion. Additionally, the non-human primate (NHP) data presented across both programs demonstrated specific and targeted depletion of B cells or plasma cells. As we look ahead, we anticipate submitting our first Clinical Trial Application for ORN-252, our anti-CD19 panCAR program this year and entering the clinic in early 2026.”
Oral Presentation details:
In Vivo panCAR™ Therapy Utilizing Circular RNA for Treatment of Autoimmune Diseases
In this presentation, Orna highlighted preclinical data demonstrating the potential of its proprietary circular (oRNA®) technology paired with its best-in-class lipid nanoparticle (LNP) delivery platform to enable the next generation of in vivo therapies and treat a variety of B cell-mediated autoimmune diseases.
Key findings in the study include:
- ORN-252, Orna’s anti-CD19 panCAR mediated robust B cell depletion in humanized mice in vivo at doses as low as 0.03 mg/kg.
- In a humanized mouse lupus model, ORN-252 showed robust B cell depletion with concurrent reduction in dsDNA titers in contrast to rituximab.
- Treatment with ORN-252 led to complete peripheral and splenic B cell depletion in NHP at doses as low as 0.1 mg/kg.
- CAR+ T cells upregulated cytotoxicity markers after the first dose when the majority of cell killing occurs.
- ORN-252 demonstrated durable B cell depletion with a reduction in switched memory and an increase in naïve B cell phenotype upon repopulation.
- ORN-252 showed superior binding affinity, expression, and killing of CD19 cells in human vs. NHP cells, potentially providing increased potency upon translation to humans.
Orna anticipates filing a Clinical Trial Application for this program by the end of 2025 and expects to initiate a first-in-human study in early 2026.
Poster Presentation details:
In Vivo panCARTM Therapy Utilizing Circular RNA for Treatment of Multiple Myeloma
In today’s poster presentation, Orna will showcase preclinical data demonstrating the potential of its in vivo panCARTM therapy to treat a range of plasma cell or BCMA-related diseases including multiple myeloma.
Key findings in the study include:
- High surface expression of anti-BCMA panCAR was observed in a dose-dependent manner on human and cynomolgus immune cells and was maintained at least 72 hours in vitro.
- In a humanized mouse model engrafted with BCMA-expressing tumor cells, anti-BCMA panCAR demonstrated superior tumor control, eliminating tumors for at least 30 days, functionally outperforming a clinically validated anti-BCMA binder.
- In NHPs, treatment with BCMA panCAR resulted in specific and targeted plasma cell depletion.
- Anti-BCMA panCAR is highly selective, demonstrating no significant impact on the overall B cell repertoire in NHPs.
About Orna Therapeutics
Orna Therapeutics is dedicated to designing and delivering a new class of fully engineered circular RNA (oRNA®) therapeutics to unlock the potential of RNA medicine to treat diseases anywhere in the body. Orna’s oRNA transcripts have advantages over traditional mRNA approaches, including simplified production, improved formulation into lipid nanoparticles, and superior protein expression. Its industry-leading LNP-based delivery systems and comprehensive editing programs position Orna to advance novel RNA medicines with vast potential to transform patient care. To learn more, visit www.ornatx.com and follow Orna on X and LinkedIn.
Orna Therapeutics Investor Contact:
Alex Lobo
Precision AQ
Alex.lobo@precisionaq.com
WATERTOWN, Mass., Nov. 3, 2025 – Orna Therapeutics, a biotechnology company dedicated to engineering immune cells in vivo to treat autoimmune and oncology diseases, today announced upcoming presentations at the 67th American Society of Hematology (ASH) Annual Meeting being held December 6-9, 2025, in Orlando, Florida. The oral and poster presentations will highlight data supporting Orna’s leading in vivo CAR programs to target and treat a broad range of B-cell driven autoimmune diseases and its anti-BCMA platform to selectively deplete plasma cells.
“We are excited to share new data at ASH from our NHP studies for our CD19 and BCMA panCAR programs,” said Joseph Bolen, Ph.D., Chief Executive Officer of Orna Therapeutics. “Data to be presented continues to demonstrate the best-in-class nature of our in vivo CAR platform in NHP for both our anti-CD19 and our anti-BCMA programs. As we look ahead, we have completed our pre-clinical package for anti-CD19 and will be submitting our Clinical Trial Application this Quarter.”
Oral Presentation details:
Title: In Vivo pan CAR Therapy Utilizing Circular RNA for Treatment of Autoimmune Diseases
Speaker: Isin Dalkilic-Liddle, Ph.D., VP Discovery Sciences,, Orna Therapeutics
Date/Time: Saturday, December 6, 2025, 9:45 AM – 10:00 AM ET
Session Name: CAR-T Cell Therapies: Basic and Translational: In vivo CAR-T cell platforms and resistance mechanisms
Location: OCCC – Sunburst Room (W340)
Poster Presentation details:
Title: In Vivo pan CAR Therapy Utilizing Circular RNA for Treatment of Multiple Myeloma
Speaker: Rebecca Silver, Ph.D., Pr. Scientist, Orna Therapeutics
Date/Time: Sunday, December 7, 2025, 6:00 PM – 8:00 PM ET
Session Name: CAR-T Cell Therapies: Basic and Translational: Poster II
Location: OCCC – West Halls B3-B4
By leveraging its leading oRNA technology and best-in-class LNP delivery, Orna’s in vivo oRNA panCAR therapies hold the potential to benefit patients across multiple B cell driven autoimmune diseases. New data to be presented at ASH will highlight the ability of Orna’s oRNA panCAR platform to generate deep and sustained B cell depletion in non-human primates across multiple doses in multiple therapeutic areas.
About Orna Therapeutics
Orna Therapeutics is dedicated to designing and delivering a new class of fully engineered circular RNA (oRNA®) therapeutics to unlock the potential of RNA medicine to treat diseases anywhere in the body. Orna’s oRNA transcripts have advantages over traditional mRNA approaches, including simplified production, improved formulation into lipid nanoparticles, and superior protein expression. Its industry-leading LNP-based delivery systems and comprehensive editing programs position Orna to advance novel RNA medicines with vast potential to transform patient care. To learn more, visit www.ornatx.com and follow Orna Therapeutics on X and LinkedIn.
Orna Therapeutics Investor Contact:
Alex Lobo
Precision AQ
Alex.lobo@precisionaq.com
WATERTOWN, Mass., October 24, 2025 – Orna Therapeutics, a leading biotechnology company developing a proprietary pipeline of in vivo therapies across a broad range of autoimmune and oncology indications, today announced an upcoming presentation at the American College of Rheumatology (ACR) Annual Meeting being held October 24-29, 2025, in Chicago, Illinois. This dataset is a broader update on Orna’s lead program, ORN-252, as it continues toward clinical entry in the first half of 2026.
Presentation Details (encore):
Title: In Vivo panCAR™ Therapy Utilizing Circular RNA for the Treatment of Autoimmune Diseases
Speaker: Isin Dalkilic-Liddle, Ph.D., Vice President, Head of Discovery Sciences, Orna Therapeutics
Date/Time: Monday, October 27, 2025, 10:30 AM – 12:30 PM CDT
Session Name: (0934-0954) Systemic Lupus Erythematosus – Animal Models Poster
Location: Hall F1
About Orna Therapeutics
Orna Therapeutics is dedicated to designing and delivering a new class of fully engineered circular RNA (oRNA®) therapeutics to unlock the potential of RNA medicine to treat diseases anywhere in the body. Orna’s oRNA transcripts have advantages over traditional mRNA approaches, including simplified production, improved formulation into lipid nanoparticles, and superior protein expression. Its industry-leading LNP-based delivery systems and comprehensive editing programs position Orna to advance novel RNA medicines with vast potential to transform patient care. To learn more, visit www.ornatx.com and follow Orna Therapeutics on X and LinkedIn.
Orna Therapeutics Investor Contact:
Alex Lobo
Precision AQ
Alex.lobo@precisionaq.com