Orbital Therapeutics Launches to Advance New Frontiers of Science with the Next Generation of Innovative RNA Medicines

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Orbital Therapeutics launched today with a vision of enhancing global health by unleashing the full potential of RNA medicines to treat human disease in ways that were not previously possible.

“The breakthroughs in RNA therapeutics over the last decade have been remarkable achievements by the biopharmaceutical industry, with several approved products for a range of diseases and many more in development. This frontier of science represents the future of medicine, and we are just beginning to realize the full breadth of its applications in treating a wide range of serious diseases,” said John Maraganore, Ph.D., Orbital co-founder and chairman of the board of directors. “Orbital Therapeutics has a unique opportunity to integrate and apply a spectrum of innovative RNA technologies to advance a portfolio that could dramatically expand the potential of today’s RNA therapeutic approaches.”

Game-Changing RNA Vision
RNA-based medicines represent a fast-growing and disruptive class of therapeutics for a breadth of disease areas. First-generation RNA treatments successfully overcame multiple hurdles, such as the rapid degradation of exogenous RNA, delivery of RNA and complications associated with immunogenicity.

To further broaden the application of this important class of treatments, Orbital is building a first-in-kind RNA platform that integrates both established and emerging technologies and delivery mechanisms, excluding RNAi. This platform is designed to extend the durability and half-life of Orbital’s novel RNA therapeutics, while also expanding their delivery to a larger number of cell types and tissues. As the company grows, Orbital intends to continue investing in the growth of its platform capabilities and technologies.

With a platform that provides access to state-of-the-art RNA and novel delivery technologies, Orbital plans to build an expansive portfolio across a range of human diseases, including in the areas of vaccines, immunomodulation, protein replacement and regenerative medicine.

Collaboration with Beam Therapeutics
As part of its launch, Orbital and Beam Therapeutics have entered into a license and research collaboration under which Orbital and Beam have each granted the other access to respective RNA technology and non-viral delivery technology. Orbital’s exclusive field of use consists of vaccines and certain therapeutic proteins, while Beam’s exclusive field of use consists of gene editing and conditioning for use in cell transplantation.

“The field of RNA medicines is advancing rapidly, and we are excited to participate in the launch of Orbital. It’s also an exciting opportunity for Beam, as we continue our strategy of pursuing creative partnerships that can generate value from the full breadth of our platform and accelerate the development of novel and diverse medicines for patients,” said John Evans, chief executive officer of Beam Therapeutics. “The collaboration with Orbital also enables us to leverage cutting-edge advancements in RNA science for the development of our pipeline of potential transformative medicines focused on gene editing.”

World-Class Founders
Orbital is founded by a collective group of scientific pioneers and proven biopharma company builders, including:

• Howard Chang, M.D., Ph.D., professor of cancer research and professor of genetics, Stanford University
• Ravi Majeti, M.D., Ph.D., professor of medicine, chief of division of hematology, Stanford University
• Drew Weissman, M.D., Ph.D., professor in vaccine research, Perelman School of Medicine, University of Pennsylvania
• Gene Yeo, Ph.D., MBA, professor of cellular and molecular medicine, UC San Diego
• Giuseppe Ciaramella, Ph.D., president and chief scientific officer, Beam Therapeutics
• John Maraganore, Ph.D., former founding chief executive officer of Alnylam Pharmaceuticals
• Kristina Burow, managing director, ARCH Venture Partners
• Carol Suh, partner, ARCH Venture Partners

In addition, Orbital is initially funded by ARCH Venture Partners, a16z Bio + Health and Newpath Partners.

“The formation of Orbital by a group of visionary leaders and scientific pioneers establishes a single organization that integrates a wide expanse of RNA technologies with a goal of delivering new medicines with a far-reaching impact on human health worldwide,” said Ms. Burow. “The ability to combine the highest caliber science with potentially revolutionary technologies and a dynamic organizational structure led by proven experts in RNA, provides a special opportunity to create a therapeutically transformational company, and we at ARCH are thrilled to be a part of such a bold and impactful mission with Orbital.”

Expert Leadership and Board
Orbital will be led by Giuseppe Ciaramella, Ph.D., who will serve as interim chief executive officer and a member of the board of directors, in addition to his ongoing role as president and chief scientific officer at Beam Therapeutics. Dr. Ciaramella has more than 25 years of drug discovery expertise and is a leader in the field of RNA research and drug development. Prior to Beam, Dr. Ciaramella served as chief scientific officer of the infectious diseases division at Moderna Therapeutics, where he led the establishment of its initial mRNA vaccine pipeline and the execution of its first investigational new drug application submission.

“In the history of medicine, certain therapeutic classes have revolutionized the treatment of both prevalent and rare diseases, and RNA-based therapeutics is undoubtedly one of them,” said Dr. Ciaramella. “The creation of Orbital brings together a critical mass of the latest innovations in RNA technology under one roof to enable near-term clinical readiness, while advancing the durability, tissue-specific programmability and breadth of clinical applications. We stand at the forefront of science with an opportunity to treat human diseases in ways that have not yet been done, and I am excited to be leading the company in this endeavor.”

Orbital has also appointed Gilles Besin, Ph.D., as chief scientific officer, bringing more than 15 years expertise in immunology and vaccines for infectious diseases, oncology and metabolic disorders. Dr. Besin joins Orbital from Affinivax Inc., where he served as vice president, head of discovery, leading all research efforts. Previously, Dr. Besin had increasingly senior roles at Moderna Therapeutics, where he led the platform immunology group and the efforts to modulate T cell responses in cancer and autoimmune diseases using mRNA lipid nanoparticles. Earlier in his career, Dr. Besin led research and discovery groups at In-Cell-Art, a biopharmaceutical company specializing in the development of DNA/RNA based vaccines and therapeutics. Dr. Besin earned an Engineering degree in biotechnology (Master’s-equivalent) from École Supérieure de Biotechnologie de Strasbourg (Strasbourg Graduate School of Biotechnology), as well as a Ph.D. in immunology from the Max Planck Institute of Immunobiology and Epigenetics. Dr. Besin is a member of the Scientific Advisory Board of Ovensa Inc.

In addition to Dr. Ciaramella, Orbital’s highly experienced board of directors includes:

• Vineeta Agarwala, M.D., Ph.D., general partner, a16z Bio + Health
• Kristina Burow, managing director, ARCH Venture Partners
• John Evans, chief executive officer, Beam Therapeutics
• John Maraganore, Ph.D., former founding chief executive officer of Alnylam Pharmaceuticals, and chairman of the Orbital board of directors
• Carol Suh, partner, ARCH Venture Partners

To support the near-term growth of the company, Orbital will leverage the resources and talent of the Beam team in addition to Dr. Ciaramella, for leadership capabilities, operational support, and research and development.

About Orbital Therapeutics
Orbital Therapeutics aims to enhance global health by unleashing the full potential of RNA-based medicines (excluding RNAi therapeutics) to treat human disease in ways that were not previously possible. The company is building a first-in-kind platform designed to sit at the intersection of RNA technology delivery methods, data science and automation to develop an expansive portfolio of medicines, initially focused in the areas of vaccines, immunomodulation, protein replacement and regenerative medicine. Founded by experts in the fields of genetic medicine and RNA development and delivery, Orbital has a dynamic operational structure designed to harness the ingenuity of a deep and diverse team of scientists, drug developers and business leaders. For more information, please visit www.OrbitalTx.com.

Beam Therapeutics Inc.’s Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to Beam’s license and collaboration agreement with Orbital and any potential benefits that may be achieved thereunder. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, the risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” in Beam’s Annual Report on Form 10-K for the year ended December 31, 2021, Beam’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2022, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause Beam’s actual results to differ may emerge from time to time. Beam undertakes no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

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