Ahead of the Curve

ORNA’S APPROACH & STRATEGY

Orna Therapeutics is leaving linear thinking behind to create fully engineered oRNATM circular RNAs – an entirely new class of RNA medicines that can realize the full potential of RNA and which is poised to change the way we treat disease. By marrying the power of oRNATM with superior delivery techniques, we are pioneering the next breakthrough in RNA therapeutics.

LIMITLESS POTENTIAL

Orna’s technology has a breadth of applications across multiple disease areas including cancer, regenerative medicine, protein replacement, infectious diseases, and autoimmunity. Our lead program is an in situ CAR therapy that combines oRNATM molecules and proprietary lipid nanoparticles (LNPs) to deliver chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (isCARTM therapy).

Our Team

Creating a new class of therapeutics requires thinking outside the lines. Comprised of a team of skilled scientists, molecular engineers, and industry veterans in addition to a world class Board, we are well-suited for the challenge of bringing the promise of our oRNATM therapeutics to patients.

Thomas Barnes, PhD
Chief Executive Officer
Amy Becker, PhD
Director, Immunology
Nelson Chau, PhD
Senior Vice President, Platform
Claudia Harper, DVM
Senior Vice President, Preclinical Safety
Daniel Anderson, PhD
Professor, Massachusetts Institute of Technology
Thomas Barnes, PhD
CEO, Orna Therapeutics
Thomas Ebeling
Advisor, MPM Capital
Ansbert Gadicke, MD
Chairman of the Board, Co-founder & Managing Director, MPM Capital

NEWS & EVENTS

Merck and Orna Therapeutics Collaborate to Advance Orna’s Next Generation of RNA Technology

Rahway, N.J. and Cambridge, Mass., August 16, 2022 – Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular… Read More >

Orna Therapeutics Raises $221 Million Series B Financing to Advance Circular RNA Platform and Accelerate Programs to the Clinic

Cambridge, Mass., August 16, 2022 – Orna Therapeutics, a biotechnology company pioneering a new class of fully engineered circular RNA therapies (oRNA), today announced the initial closing of its $221 million Series B financing. At… Read More >
Merck and Orna Therapeutics Collaborate to Advance Orna’s Next Generation of RNA Technology

Collaboration supports multiple discovery efforts, including vaccines

Rahway, N.J. and Cambridge, Mass., August 16, 2022 – Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA (oRNA) therapies, today announced a collaboration agreement to discover, develop, and commercialize multiple programs, including vaccines and therapeutics in the areas of infectious diseases and oncology.

             Under the terms of the agreement, Merck will make an upfront payment to Orna of $150 million, which will be expensed by Merck in the third quarter of 2022 and included in non-GAAP results. In addition, Orna will be eligible to receive up to $3.5 billion in development, regulatory, and sales milestones associated with the progress of the multiple vaccine and therapeutic programs, as well as royalties on any approved products derived from the collaboration. Orna will retain rights to its oRNA-LNP technology platform and will continue to advance other wholly owned programs in areas such as oncology and genetic disease. Merck will also invest $100 million of equity in Orna’s recently completed Series B financing round.

             Orna’s proprietary oRNA technology creates circular RNAs (oRNAs) from linear RNAs by self-circularization. oRNA molecules have been shown to have greater stability in vivo than linear mRNA and have the potential to produce larger quantities of therapeutic proteins inside the body. Newly synthesized oRNA molecules are more compactly packaged into custom lipid nanoparticles (LNPs), which Orna has engineered to target key tissues in the body. Preclinical data, including presentations at the 2022 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, have demonstrated the potential of oRNA expression and delivery as an approach for further development in multiple areas, including vaccines and oncology therapeutics.

             “This broad strategic collaboration brings together Merck’s significant expertise in nucleic acid biology, clinical development, and manufacturing with Orna’s compelling circular RNA technology to explore the opportunity to develop a new generation of potential vaccines and therapeutics,” said Fiona Marshall, senior vice president and head of discovery, preclinical and translational medicine at Merck Research Laboratories. “We look forward to working with the talented scientific and technical teams at Orna.”

             “We are thrilled to collaborate with Merck, a company committed to breakthrough science, which has recognized the potential our platform can bring to patients. Our oRNA technology plus novel delivery solutions are designed to unlock the full potential of RNA in therapeutics and vaccines,” said Tom Barnes, Chief Executive Officer of Orna. “The combined expertise of Orna and Merck aims to accelerate the development of RNA therapeutics for patients in need of better treatment options.”

About Merck

             At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on TwitterFacebookInstagramYouTube and LinkedIn.

About Orna Therapeutics

             Orna Therapeutics was founded on groundbreaking research by Alex Wesselhoeft, Ph.D., and Daniel G. Anderson, Ph.D. from MIT and built by MPM Capital and BioImpact Capital, an investment management affiliate of MPM. Orna’s proprietary circular RNA (oRNA) is engineered as linear RNA that self-circularizes. By taking a line and turning it into a circle, oRNA exhibits numerous advantages over traditional linear mRNA therapies such as simplified production, increased protein expression, and a superior immunogenicity profile. With proprietary lipid nanoparticles (LNPs), including those from Orna’s joint venture with ReNAgade Therapeutics, an RNA delivery company, our technologies expand the possibilities of what RNA therapeutics can achieve. To learn more visit: www.ornatx.com and follow Orna Therapeutics on Twitter and LinkedIn.

Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA

            This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

            Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of the global outbreak of novel coronavirus disease (COVID-19); the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

            The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2021 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov).

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Merck Media Contacts

Melissa Moody: (215) 407-3536
Justine Moore: (908) 740-6449

Merck Investor Contacts
Peter Dannenbaum: (908) 740-1037

Orna Therapeutics Media Contact
Peg Rusconi, Verge Scientific: prusconi@vergescientific.com

Orna Therapeutics Raises $221 Million Series B Financing to Advance Circular RNA Platform and Accelerate Programs to the Clinic

Funding will advance company’s isCAR program toward clinical trials as well as support development of other internal and partnered preclinical programs

Cambridge, Mass., August 16, 2022Orna Therapeutics, a biotechnology company pioneering a new class of fully engineered circular RNA therapies (oRNA), today announced the initial closing of its $221 million Series B financing. At signing, the company received approximately $121 million and expects to receive the remaining $100 million subject to customary closing conditions (including regulatory approval under the Hart-Scott-Rodino (HSR) Act). Merck participated as a new investor in the financing alongside commitments from founding investors MPM Capital and BioImpact Capital, an affiliate of MPM, among others. Orna was created in 2019 by MPM Capital and BioImpact Capital, with funding from the UBS Oncology Impact Fund.

The financing will enable Orna to continue development of its circular RNA + LNP (lipid nanoparticle) delivery platform, as augmented by its joint venture with ReNAgade Therapeutics, an RNA delivery company created by MPM Capital and BioImpact Capital, to advance its lead in situ CAR (isCAR) program toward the clinic and build manufacturing capabilities to support development of an expanding preclinical and clinical pipeline.

“In under three years, Orna has taken proprietary circular RNA from academic literature to the first proof of concept data in preclinical models,” said Tom Barnes, Ph.D., Chief Executive Officer of Orna and Entrepreneur at BioImpact Capital. “Investors continue to recognize our premier technology and the hard work of our team, and we are pleased to see such unwavering belief in the potential of circular RNA to revolutionize how we treat disease.”

Orna is expanding the reach of current RNA therapeutics with recently presented preclinical data demonstrating the potential of oRNA to treat cancer, genetic disorders, and infectious diseases. Orna’s lead isCAR program demonstrated tumor suppression and eradication in animal models, indicating significant potential that oRNA-based cancer therapies could be superior to traditional cell therapies. With the proceeds of the Series B financing, Orna anticipates advancing this program into clinical trials in 2024.

“In just a few years since we created and built the company, Orna has made remarkable progress in demonstrating the extensive power of its circular RNA + LNP delivery technologies,” said Ansbert Gadicke, M.D., Chairman of Orna, Managing Partner of BioImpact Capital and MPM Managing Director. “oRNA, with its numerous advantages over linear mRNA, is the next generation of RNA therapies, and we are excited to see the continued development and applications of this technology.”

“It has been validating to see the promise of RNA realized through linear mRNA-based vaccines,” said Daniel G. Anderson, Ph.D., Professor of Chemical Engineering and core member of the Koch Institute for Integrative Cancer Research and the Institute for Medical Engineering and Science at MIT, and Co-founder of Orna. “Orna’s next generation of oRNA therapies not only demonstrate promise as vaccines, but also as treatments for a range of diseases where other modalities have fallen short.”

SVB Securities acted as financial advisor to Orna on this transaction.

About Orna Therapeutics
Orna Therapeutics was founded on groundbreaking research by Alex Wesselhoeft, Ph.D., and Daniel G. Anderson, Ph.D., from MIT and built by MPM Capital and BioImpact Capital, an affiliate of MPM. Orna’s proprietary circular RNA (oRNA) is engineered as linear RNA that self-circularizes. By taking a line and turning it into a circle, oRNA exhibits numerous advantages over traditional linear mRNA therapies such as simplified production, increased protein expression, and a superior immunogenicity profile. With proprietary lipid nanoparticles (LNPs), including those from Orna’s joint venture with ReNAgade Therapeutics, an RNA delivery company, our technologies expand the possibilities of what RNA therapeutics can achieve. To learn more visit: www.ornatx.com and follow Orna Therapeutics on Twitter and LinkedIn.

Media Contact

Peg Rusconi, Verge Scientific Communications
prusconi@vergescientific.com

ASGCT: Systemic Delivery of Circular RNA Encoding Partial Dystrophins and Expression in Skeletal Muscle

May 17, 2022 Tatiana Fontelonga et al.