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.
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.
Partner, MPM BioImpact
Partner, MPM BioImpact
Dr. Brian Goodman is responsible for investment identification, due diligence, business development and new company creation activities at MPM BioImpact Capital. Brian served as the founding Head of Business development at Orna Therapeutics prior to joining the Board of Directors. Additionally, Brian is a cofounder and head of corporate development and operations at Aktis Therapeutics and serves as a Board Observer of Triplet Therapeutics.
Prior to joining MPM BioImpact, Brian co-founded Evelo Biosciences (EVLO) and served as Head of Technology and Innovation. At Evelo, Brian led a team that established new programs to discover, formulate, and evaluate the pharmacology of microbial drugs across multiple therapeutic areas. He contributed to the in-licensing of foundational IP and forged collaborative research with multiple academic groups to complement Evelo’s internal research and development efforts. He is an inventor on multiple patent applications. Before taking the operational role at Evelo, Brian was a Senior Associate at Flagship Pioneering where he worked with the entrepreneurial division to build and launch new biotech ventures.
Brian holds a Ph.D. in Biological Chemistry and Molecular Pharmacology from Harvard Medical School, and B.S. and B.A. degrees in biochemistry and economics, respectively, from Brandeis University.
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
Simnova and Orna Expand Strategic Partnership to Include BCMA-Targeted RNA Therapeutics
Orna Therapeutics Appoints Vikas Sinha to its Board of Directors
Orna Therapeutics Establishes Strategic Collaboration with Vertex Pharmaceuticals to Develop Next Generation Approaches for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)
SHANGHAI and CAMBRIDGE, Mass. , Jan. 15, 2025 /PRNewswire/ — Orna Therapeutics, Inc. (“Orna”) and Shanghai Simnova Biotech Co., Ltd. (“Simnova”) are pleased to announce the expansion of their strategic collaboration to include BCMA (B-cell maturation antigen) as a designated biological target for RNA-based therapeutics development. This partnership leverages Orna’s groundbreaking circular RNA (oRNA®) technology and Simnova’s expertise in cell therapy to deliver transformative treatments for patients worldwide.
Under the agreement, Simnova will pursue the research and development and commercialization of in vivo panCar cell therapies targeting BCMA in greater China and Orna will retain all rights for development and commercialization in the rest of the world. Each party will have the right to receive an upfront payment within their respective licensed territories and is eligible for clinical development, regulatory, and commercialization milestones as well as royalties on any approved products derived from the collaboration.
In January 2023, Simnova and Orna announced a collaboration agreement granting Simnova exclusive rights to develop and commercialize Orna’s in vivo cell therapy products in the Greater China region. This includes Orna’s lead isCAR project targeting CD19, “ORN-101.”
“We are excited to deepen our partnership with Simnova to bring an in vivo BCMA panCAR therapy to patients with multiple Myeloma,” said Ansbert Gadicke, M.D., Chairman of Orna and Managing Partner of MPM BioImpact. “Orna’s panCAR approach holds the potential to introduce a novel class of in vivo CAR therapies that overcomes the limitations of current ex vivo cell therapies. The exciting pre-clinical and non-human primate data that Orna has generated continues to reinforce our commitment in this area. We look forward to advancing our programs towards the clinic.”
Dr. Zhuoxiao CAO, CEO of Simnova, added, “Our shared commitment to advancing immunotherapies and oncology treatments opens new opportunities to address unmet medical needs through the development of BCMA-targeted therapeutics.”
About Orna
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.
About Simnova
Shanghai Simnova Biotech Co., Ltd. (www.simnovabio.com) is dedicated to bringing innovative cell therapy products to oncology and autoimmune patients. Simnova Biotech has a rich R&D pipeline, with technical advantages in its proprietary universal off-the-shelf CAR-NK and BiTE CAR-T programs, both of which are currently in clinical development stage.
Orna Therapeutics Investor Contact:
Alex Lobo
Precision AQ
alex.lobo@precisionaq.com
WATERTOWN, Mass., January 9, 2025 – 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 announced the appointment of Vikas Sinha to its Board of Directors.
“We are thrilled to welcome Vikas to our Board of Directors. He brings a wealth of leadership experience spanning finance, corporate strategy and capital raising in the cell and gene therapy space,” commented Amit Munshi, Chief Executive Officer of Orna. “We look forward to leveraging his expertise as we enter our next stage of growth and advance our panCAR™ programs for patients with cancer and autoimmune diseases.”
Mr. Sinha brings more than 25 years of experience working in executive finance roles in the life sciences industry. He currently serves as Chief Financial Officer and co-founder of ElevateBio, a holding company focused on building cell and gene therapy companies. Mr. Sinha also serves as President, Chief Financial Officer, and Director of AlloVir, an ElevateBio portfolio company. Concurrently, he serves as a member of the board of directors of Verona Pharma. Prior to joining ElevateBio and AlloVir, he was Chief Financial Officer of Alexion Pharmaceuticals. Before that, Mr. Sinha held various positions with Bayer across the world including Chief Financial Officer, Bayer North America and Chief Financial Officer, Bayer Yakuhin, Japan.
Mr. Sinha holds an MBA from the Asian Institute of Management. He is a qualified Chartered Accountant from the Institute of Chartered Accountants of India and a Certified Public Accountant in the U.S.
“I am excited to join Orna’s Board of Directors and have been impressed by the breadth and depth of the Company’s platforms and pipeline, which hold the potential to overcome existing limitations of cell therapies to deliver outsized impact to patients living with cancer and autoimmune diseases,” said Mr. Sinha. “I look forward to working with the Orna Board and leadership to advance its lead panCAR programs towards the clinic and position the organization for future growth.”
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.
Investor Contact:
Precision AQ
Alex Lobo
alex.lobo@precisionaq.com
— Collaboration to leverage Orna’s differentiated lipid nanoparticle (LNP) delivery solutions for patients with SCD and TDT —
— Orna to receive an upfront payment and is eligible to receive potential pre-clinical, clinical, and commercialization milestone payments and royalties —
WATERTOWN, Mass., Jan. 7, 2025 /PRNewswire/ — Orna Therapeutics (through its wholly owned subsidiary ReNAgade Therapeutics Inc.) announced a three-year strategic research collaboration with Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) to utilize Orna’s novel and proprietary LNP delivery solutions to enhance Vertex’s efforts in developing next generation gene editing therapies for patients with SCD and TDT.
“Vertex is a leader in delivering next-generation approaches to treating hemoglobinopathies, and we are excited to collaborate with them to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to HSCs,” commented Amit Munshi, Chief Executive Officer of Orna. “Today’s collaboration further validates our industry leading extra-hepatic LNP delivery chemistries and highlights the importance of delivery to enable the next wave of RNA medicines.”
Ansbert Gadicke, M.D., Chairman of Orna and Managing Partner of MPM BioImpact added, “Today’s collaboration leveraging Orna’s industry-leading non-viral HSC delivery represents our second major partnership and is testament to the breadth and potential of the Company’s platform. We look forward to partnering with Vertex to develop transformative therapies for SCD and TDT, while simultaneously advancing our lead panCAR™ pipeline programs in autoimmune and oncology. This deal further solidifies Orna’s leadership in next generation RNA medicines and has the potential to deliver large-scale impact to patients.”
About the Collaboration
Under the terms of the agreement, Orna will receive upfront payments of $65 million, including an investment in the form of a convertible note, and is eligible to receive up to $635 million based upon the achievement of specified pre-clinical, research, development, regulatory and commercial milestones related to SCD/TDT products. Additionally, Orna is further eligible to receive up to $365 million in additional option fees and milestones per product for up to ten additional products if Vertex options rights in additional indications. Orna will be eligible to receive tiered royalties on future net sales of any products that may result from this collaboration. Vertex is funding the three-year research collaboration and holds an option to extend the research collaboration term.
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