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.
Dr. Frank Neumann brings a record of proven leadership in cell therapy and oncology clinical development to his role as Chief Medical Officer.
Prior to joining Orna, Frank most recently served as Senior Vice President and Global Head of Clinical Development at Kite, a Gilead Company. He previously held positions as Chief Medical Officer at Verastem, Inc. (also known as Verastem Oncology) and as Vice President, Head of Oncology Clinical Research at bluebird bio. In addition, Frank was clinical development head of all global cell therapy approaches at Takeda Pharmaceuticals, where he held various leadership roles of increasing prominence, including global clinical lead and medical team lead for two distinct cancer therapies. Earlier in his career, he was a member of the oncology medical teams at AstraZeneca and Sanofi-Aventis.
Board certified in Hematology/Oncology, Internal Medicine and Palliative Care Medicine, Frank was a research scholar at the University of Texas MD Anderson Cancer Center and is currently an assistant professor at the Heinrich Heine University in Düsseldorf, Germany, where he received his M.D. He earned his Ph.D. from the Rheinische-Friedrich-Wilhelm University in Bonn, Germany. In addition to his role with Orna, Frank is an Entrepreneur Partner with MPM BioImpact.
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 Preclinical Data Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases at the American Society of Gene and Cell Therapy Annual Meeting
Orna Therapeutics Announces Presentation at the American Society of Gene and Cell Therapy Annual Meeting Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases
Orna Therapeutics Appoints Joseph Bolen, Ph.D., as Chief Executive Officer
WATERTOWN, Mass., May 15, 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 the presentation of new preclinical data supporting its in vivo CAR therapy approach in autoimmune diseases during an oral session at the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting being held May 13-17, 2025, in New Orleans, Louisiana.
“The preclinical data presented today at ASGCT highlight our potential to deliver on the promise of in vivo CAR T therapy,” said Joseph Bolen, Ph.D., Chief Executive Officer of Orna Therapeutics. “Our CD19 panCAR program has demonstrated not only successful delivery of our lead panCAR LNP to disease-relevant immune cell types, but also robust and sustained B cell depletion at low doses in both peripheral blood and lymphoid tissues in non-human primates (NHPs). These compelling results continue to reinforce our commitment to translating our promising science into meaningful therapies for patients and we look forward to advancing our CD19 panCAR program towards the clinic in 2026.”
Presentation Details:
Title: In Vivo panCAR™ Therapy Using Circular RNA for the Treatment of Autoimmune Disease
Speaker: Megan Hoban, Ph.D., panCAR Program Lead, Orna Therapeutics
Date/Time: Thursday, May 15, 2025, 8:00 AM – 9:45 AM CDT
Session Name: Cellular and Gene Therapies for Autoimmune Disease
Location: Room 388-390
In today’s presentation, Orna will showcase preclinical data demonstrating the potential of its in vivo panCAR therapy, enabled by its proprietary circular (oRNA®) technology and best-in-class lipid nanoparticle (LNP) delivery system to achieve robust and sustained B cell depletion in both humanized mouse models and non-human primates across multiple doses.
Key findings from the study include:
- Validated extra-hepatic delivery to disease-relevant immune cell types, including T cells, in mice and NHPs without requiring targeting ligands.
- Lead panCAR LNP achieved over 60% delivery to peripheral blood and splenic T cells in NHPs.
- CD19 panCAR doses as low as 0.03mpk led robust B cell depletion, with multi-dosing achieving increased B cell depletion in humanized mice.
- In a humanized lupus mouse model, CD19 panCAR showed strong B cell depletion and a meaningful and differentiated reduction in dsDNA titers compared to rituximab.
- CD19 panCAR induced full depletion of B cells across peripheral blood, spleen, lymph nodes, and bone marrow in NHPs, with peripheral B cells beginning to reconstitute after three weeks.
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, comprehensive editing programs and validating large pharma partnerships 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., May 5, 2025 – Orna Therapeutics, a biotechnology company dedicated to engineering immune cells in vivo to treat autoimmune and oncology diseases, today announced an upcoming presentation at the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting being held May 13-17, 2025, in New Orleans, Louisiana. The presentation will highlight data supporting Orna’s in vivo CAR therapy approach in autoimmune diseases.
“We are excited to present new data at the upcoming ASGCT annual meeting as we advance our panCAR™ programs towards the clinic,” said Joseph Bolen, Ph.D., Chief Executive Officer of Orna Therapeutics. “Our platform, which pairs our circular (oRNA®) technology and best-in-class lipid nanoparticle (LNP) delivery has the potential to deliver a pipeline in a product and treat multiple diseases and therapeutic areas. The data to be presented will highlight the ability of our platform to enable sustained pharmacodynamic effects at low doses, durable protein expression, and repeat dosing. We look forward to advancing our CD19 autoimmune panCAR program into the clinic in 2026, followed by our BCMA panCAR program in oncology.”
Presentation details are as follows:
Title: In Vivo panCAR™ Therapy Using Circular RNA for the Treatment of Autoimmune Disease
Speaker: Megan Hoban, Ph.D., panCAR Program Lead, Orna Therapeutics
Date/Time: Thursday, May 15, 2025, 8:00 AM – 9:45 AM CDT
Session Name: Cellular and Gene Therapies for Autoimmune Disease
Location: Room 388-390
By leveraging its leading oRNA technology and potentially best-in-class LNP delivery, Orna’s in vivo CD19 panCAR platform holds the potential to benefit patients across multiple B cell driven autoimmune diseases. New data to be presented at ASGCT will highlight the ability of Orna’s CD19 panCAR platform to generate deep and sustained B cell depletion in non-human primates across multiple doses.
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., March 31, 2025 – Orna Therapeutics, a biotechnology company dedicated to engineering immune cells in vivo to treat oncology and autoimmune diseases, today announced the appointment of Joseph Bolen, Ph.D., as Chief Executive Officer. Dr. Bolen most recently served as Orna’s Chief Scientific Officer (CSO) and succeeds Amit Munshi who is stepping aside for personal reasons.
Orna expects to initiate clinical studies in 2026 for its two wholly owned, potentially best-in-class in vivo panCAR assets, including its CD19 program for B cell-driven autoimmune diseases and a BCMA program for multiple myeloma. In addition to its wholly owned pipeline, Orna has secured two major, potentially multi-billion-dollar pharma partnerships with Merck for the development of vaccines and therapeutics in infectious disease and oncology and with Vertex for next generation approaches to sickle cell disease and transfusion-dependent beta thalassemia.
“At its core, Orna is a science driven organization that has greatly benefited from Joe’s breadth and depth of drug development expertise,” said Ansbert Gadicke, M.D., Chairman of Orna and Managing Partner of MPM BioImpact. “This is the perfect time for Joe to take the helm as Orna advances both its proprietary programs in autoimmune and oncology to the clinic. Joe’s scientific expertise and organizational leadership capabilities, in conjunction with the Company’s strong cash position, will enable us to achieve key milestones across our wholly owned and partnered programs.”
Dr. Gadicke continued, “Amit leaves Orna in a strong position and we would like to express our deep appreciation for his commitment and contributions.”
Dr. Bolen joined Orna through its acquisition of ReNAgade Therapeutics and has served as CSO since November 2024, concurrent to his role as an Entrepreneur Partner at MPM BioImpact. Prior to Orna, Dr. Bolen served in leadership roles at ReNAgade Therapeutics. Previously, he oversaw all aspects of R&D for Moderna Therapeutics as CSO and President of Research and Development. Before Moderna, Dr. Bolen was CSO at Millennium Pharmaceuticals and Global Head of Oncology Research at Takeda Pharmaceutical Company. Earlier in his career, he held senior R&D positions at Hoechst Marion Roussel, Schering-Plough (DNAX) and Bristol Myers Squibb. Dr. Bolen began his career at the National Institutes of Health (NIH). He 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.
“I am excited to lead our world-class team, as we advance some of the most transformative science I’ve seen in my 35-year career in this industry. Our leading circular RNA paired with our potentially best-in-class delivery technologies hold the potential to overcome the limitations of existing ex-vivo technologies and deliver large-scale impact to patients across a range of diseases,” said Dr. Bolen. “With robust data in hand demonstrating our ability to deliver and engineer immune cells in vivo, our focus is now on advancing our first two panCAR programs into the clinic in 2026 and continuing to progress our pharma partnerships. I look forward to leading our team through this next exciting chapter of the Company’s growth as we unlock the potential of RNA medicines.”
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 circular RNA 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