Orbital Therapeutics Launches to Advance Next-Generation of RNA Medicines

Orbital Therapeutics launched today with a strategic partnership with Beam Therapeutics and a vision of unleashing the full potential of RNA medicines to treat human disease in ways that were not previously possible.

Photo: John Maraganore, Orbital co-founder and chairman of the board of directors

“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, Orbital co-founder and chairman of the board of directors and former founding CEO of Alnylam Pharmaceuticals. “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.”

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.

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, CEO 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.”

Besides Maranganore, Orbital’s founders include Howard Chang, professor of cancer research and professor of genetics, Stanford University; Ravi Majeti, professor of medicine, chief of division of hematology, Stanford University;

Drew Weissman, professor in vaccine research, Perelman School of Medicine, University of Pennsylvania; Gene Yeo, professor of cellular and molecular medicine, UC San Diego; Giuseppe Ciaramella, president and chief scientific officer, Beam Therapeutics; Kristina Burow, managing director, ARCH Venture Partners; and Carol Suh, partner, ARCH Venture Partners.

ARCH Venture Partners, a16z Bio + Health, and Newpath Partners have provided initial funding to Orbital. Beam has an equity stake in Orbital, which will support the near-term growth of the company.

Orbital will be led by Giuseppe Ciaramella, 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. Prior to Beam, 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 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.”

Author: Rare Daily Staff