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 
Amit is a 30+ year industry veteran who is passionate about developing transformative medicines. Amit was most recently Chief Executive Officer of ReNAgade Therapeutics. Previously as President and CEO of Arena Pharmaceuticals Inc. (Nasdaq:ARNA), he built the company from a $300M market cap into a late clinical stage company with 37 programs, 4 drugs, and 11 indications before their $6.7B acquisition by Pfizer.
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.
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.
Orna Therapeutics
Orna Therapeutics
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.
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.
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, Eleven Biotherapeutics, and Orna Therapeutics. 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.
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 Unveils Novel In Vivo Gene Editing Data Highlighting Engineering and Delivery Approach at the 31st Annual European Society of Gene & Cell Therapy Congress
Orna Therapeutics Appoints Nobel Laureate Carolyn Bertozzi, Ph.D., to Scientific Advisory Board
ReNAgade is acquired by RNA rival a year after megaround-backed launch
Lead type V editor program in SCD demonstrates industry leading delivery and repeat dosing with a passive LNP
Results reveal unprecedented improvement of editing rates from single digits to nearly 80% in primary HSPCs
WATERTOWN, Mass., October 22, 2024 /PRNewswire/ – Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of circular RNA medicines and unprecedented lipid nanoparticle (LNP) delivery solutions for oncology and autoimmune diseases, today presented a poster highlighting preclinical data from its SiTu Editing in the Marrow (STEM) in vivo CRISPR editing platform at the European Society of Gene & Cell Therapy Annual Congress taking place October 22-25 in Rome.
The data show dramatically improved editing rates from single digits to roughly 80% in primary hematopoietic stem progenitor cells (HSPCs) from healthy donors. Orna’s STEM technology is geared to address beta-hemoglobinopathies including sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
While significant progress has been made with two recent U.S. Food and Drug Administration approvals for SCD and TDT using ex vivo approaches, patient journeys involve lengthy wait times for treatment at specialized centers and harsh therapeutic regimens that come with serious safety risks that can limit eligibility. By contrast, in vivo delivery of gene editing therapies offers a simple, off-the-shelf treatment that can be administered in an outpatient setting.
“Our results are highly promising and demonstrate the powerful potential of Orna’s type V editors when combined with HSPC-targeted RNA delivery to enable in vivo delivery without the need for a targeting ligand or antibody fragment,” said Robert Mabry, Chief Scientific Officer for Orna. “Orna Therapeutics is pioneering an entirely new approach to genome engineering through the development of our gene editing and in vivo delivery platforms, which could offer more patients access to less toxic and complicated cell-based therapies.”
Utilizing a high-throughput barcoding screening approach in non-human primates (NHPs), the company identified a series of LNPs that demonstrated tropism to a rare population of CD34+ HSPCs that reside in the bone marrow. The technology allows for repeat dosing, which is not currently feasible for viral-based delivery approaches.
The lead HSPC-tropic LNP candidate was found to have greater than 70% reporter-positive bulk CD34+ cells and greater than 95% reporter positive long-term HSCs in humanized mice. Furthermore, when tested individually in NHPs, the LNP candidate showed robust delivery to the bone marrow – revealing an average of 24% reporter positive HSPCs, while multiple doses achieved up to 30% editing.
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 Twitter and LinkedIn.
Media Contact
Peg Rusconi
Deerfield Group
Peg.rusconi@vergescientific.com
Investor Contact
WATERTOWN, Mass., July 30, 2024 – Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of circular RNA medicines, including panCAR™ in vivo CAR therapies for oncology and autoimmune diseases, today announced the appointment of Carolyn Bertozzi, Ph.D., a 2022 Nobel Laureate in Chemistry, as Co-Chair of its Scientific Advisory Board (SAB), joining Orna former Chief Executive Officer Tom Barnes, Ph.D., as Co-Chair. As a pioneer in emerging technologies, Dr. Bertozzi 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.
“Dr. Bertozzi is a visionary scientist whose discoveries have led to multiple biotech innovations, and we are very excited for her to co-lead our new Scientific Advisory Board with Dr. Barnes,” said Amit D. Munshi, Chief Executive Officer of Orna. “Her appointment will provide deep expertise across Orna’s established circular RNA expression technologies and support the expansion of our delivery technology, further solidifying our leadership position in gene editing. Dr. Bertozzi will play a crucial role in building out our SAB and advising our team to realize the full potential of RNA therapeutics for cancer and autoimmune diseases.”
“Orna is advancing a truly innovative RNA engine with the potential to overcome the current limitations of cell therapies,” said Dr. Bertozzi. “I am very pleased to join Tom on Orna’s SAB and look forward to working closely with Amit and the company’s leadership team to lend my experience in support of their quest to develop paradigm-shifting RNA medicines.”
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.
In addition to her academic work, Dr. Bertozzi has founded multiple biotechnology start-ups based on her research. An elected member of the National Academy of Medicine, National Academy of Sciences, National Academy of Engineering, and American Academy of Arts and Sciences, she 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.
About Orna Therapeutics
Founded on research by Alex Wesselhoeft, Ph.D., and Daniel G. Anderson, Ph.D., of MIT, 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 formulatability, 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.
Media Contact
Peg Rusconi
Deerfield Group
Peg.rusconi@deerfieldgroup.com
Investor Contact