Beam-linked startup launches with an eye on the next generation of RNA drugs

Dive Brief:

• Orbital Therapeutics, a Cambridge, Mass.-based biotechnology startup focused on RNA-based medicines, launched Wednesday with a research collaboration with gene editing company Beam Therapeutics.
• The new company will initially be run on an interim basis by Beam’s chief scientific officer Giuseppe Ciaramella and has access to some of the biotech’s drug delivery technology. It aims to use a variety of different technologies to broaden the reach of RNA drugs, which are currently limited to certain diseases, but didn’t provide specifics.
• Orbital is backed by venture firms Arch Venture Partners, a16z Bio + Health and Newpath Partners. The company is chaired by John Maraganore, the longtime CEO of Alnylam Pharmaceuticals, and counts University of Pennsylvania professor, Drew Weissman, a pioneering RNA researcher, among its founders.

Dive Insight:

Over the last decade, several technologies focused on RNA, the messenger molecules that help cells make proteins, have come of age. Two approaches — RNA interference and antisense oligonucleotides — have led to drugs for rare diseases and high cholesterol. Another method based on messenger RNA produced the COVID-19 vaccines that have saved millions of lives during the pandemic.

Those successes have shown the potential of RNA drugs to make new medicines, but also their limitations. RNA interference and antisense-based medicines remain largely constrained to disease targets in the liver, for instance, leaving many conditions beyond companies’ reach.

A wave of recently launched biotech startups aim to expand drugmakers’ toolkit. Over the last few years, investors have poured hundreds of millions of dollars into a handful of companies exploring next-generation RNA technologies. Large drugmakers have also begun to show interest.

Orbital is the latest to emerge. Though it’s unclear what its specific focus will be, the company says its platform includes both “established and emerging” RNA drug technologies and delivery tools. Those capabilities could help “extend the durability and half-life” of Orbital’s drug candidates as well as expand the types of cells and tissues they can reach, the company said in a statement.

“We see a huge opportunity in the setting of RNA-based therapies to overcome some of the shortcomings that have been there with the first generation of RNA companies,” said Maraganore, the longtime Alnylam CEO and an Orbital co-founder, in an interview.

Maraganore said Orbital is not developing RNA interference drugs, Alnylam’s specialty, or antisense oligonucleotides, which were advanced by Ionis Pharmaceuticals and others.

Instead, the company is exploring how RNA-based drugs might be used more broadly to make vaccines, treatments for autoimmune disease and protein replacement therapies. The company declined to say which specific conditions it’s targeting, or how far away it is from clinical testing.

Orbital’s research collaboration with Beam allows the companies to use each other’s technologies. Orbital will get access to Beam’s delivery tools, and be able to exclusively use them to develop vaccines and target certain unspecified therapeutic proteins. Beam has sole access to Orbital’s research for gene editing and the “conditioning” regimens used to prepare patients for cell therapies, the companies said.

The collaboration helps Beam “leverage cutting-edge advancements in RNA science” to develop gene editing drugs, Beam CEO John Evans said in a statement.

Beam will also own 75 million shares in Orbital, according to a Wednesday note from RBC Capital Markets analyst Luca Issi. The exact stake in Orbital that those shares give Beam will depend on future fundraising, Issi wrote.

The alliance with Orbital is the latest in a string of deals with up-and-coming biotech companies that Beam has signed.

Verve Therapeutics licensed technology from Beam to create a gene editing treatment for heart disease that recently began human testing. Prime Medicine, a richly funded biotech startup, announced a collaboration with Beam in 2019 to develop more precise DNA changing therapies. And in 2021, Beam bought Guide Therapeutics and its drug delivery tools for $120 million in stock.

This post has been syndicated from a third-party source. View the original article here.