Orna’s Approach & Strategy Paint Underline

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.

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Circular photo of an Orna scientist in a lab as she pipettes into a tube, encircled by an ombre red-yellow paint stroke

Limitless Potential Paint Underline

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.

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About Us Paint Underline

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 Paint Underline

LEADERSHIP
  • Leadership
  • Board of Directors
  • Scientific Advisory Board
  • Investors
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Amit Munshi
Chief Executive Officer
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Joe Bolen, Ph.D.
Chief Science Innovation Officer
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Hope D’Oyley-Gay, JD
Chief Legal Officer
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Ciaran Lawlor, Ph.D.
Chief Operating Officer
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Ansbert Gadicke, M.D.
Chairman of the Board, Co-Founder,
Managing Partner, MPM BioImpact
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Daniel Anderson, Ph.D.
Professor, Massachusetts Institute of Technology
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Thomas Barnes, Ph.D.
Former Chief Executive Officer,
Orna Therapeutics
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David Barrett
Vice President of Cell Biology, Kite Pharma, a Gilead Company
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Tom Barnes, Ph.D.
Co-Chair
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Carolyn Bertozzi, Ph.D.
Co-Chair
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MPM Biolmpact logo
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News & Events Paint Underline

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

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…

Orna Therapeutics Appoints Nobel Laureate Carolyn Bertozzi, Ph.D., to Scientific Advisory Board

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…

ReNAgade is acquired by RNA rival a year after megaround-backed launch

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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

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

Investor-relations@ornatx.com

Orna Therapeutics Appoints Nobel Laureate Carolyn Bertozzi, Ph.D., to Scientific Advisory Board

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

Investor-relations@ornatx.com

ReNAgade is acquired by RNA rival a year after megaround-backed launch

May 23, 2024 – Two buzzy, well-funded biotech startups are combining in a bid to create a powerhouse at the bleeding edge of genetic medicines.