Early in 2020, Michael Ehlers, a former Biogen executive turned venture capitalist and biotech entrepreneur, was brainstorming ways to create a different kind of gene therapy.
Amid a genetic medicine boom, dozens of companies had built and advanced into testing experimental therapies that used the same principles, tucking functional genes into viruses capable of shuttling DNA into cells.
Ehlers and the startup he began to build that spring with his venture firm Apple Tree Partners imagined something different. Rather than rely on viruses, which are very efficient at their job but can bring safety concerns, Ehlers and his team proposed doing away with them entirely. Instead, they aim to deliver circular strands of DNA with a twist on electroporation, a decades-old technique that uses electrical fields to open doorways into cells.
"Could we completely replace viral-based [therapies] with a novel synthetic DNA form? "Ehlers said in an interview. "And, second, could we build upon 40 years of electroporation, reduce it to practice and create a platform that really succeeds?"
The startup Ehlers and Apple Tree Partners are launching Thursday, called Intergalactic Therapeutics, aims to answer those questions and has $75 million in funding to get started. It's one of a handful of new biotech companies that aim, in different ways, to create non-viral gene therapy, a group that includes Generation Bio, Code Biotherapeutics and the recently launched Anjarium Biosciences. (All are early stage and their science has not yet been proven.)
Ehlers, who has had a hand in launching other companies as Apple Tree Partners' chief scientist, likens Intergalactic's approach as a marriage between gene therapy and interventional medicine.
The company envisions using synthetic DNA, constructed as a circle rather than a linear strand, as the basis of its therapy, which will be delivered into cells via an electroporation system dubbed COMET. Once there, the genetic instructions can act as other virally delivered genes might, replacing missing proteins or re-enabling lost cellular functions.
Because Intergalactic's therapies will be non-viral, the company may be able to sidestep the preexisting immunity and immune responses that can be triggered by viral-based gene therapies. Intergalactic can also work with larger genes than would be possible to incorporate into a virus and consider administering its medicines more than once. The latter point is a distinction from most gene therapies now in testing, which are thought of as one-time treatments in part because of how the immune system might react to them.
For Ehlers, Intergalactic builds on his first role with an Apple Tree Partners company after stepping down as Biogen's head of research in the fall of 2019. Along with then becoming Apple Tree's chief scientific officer, he jumped in as CEO of a new gene therapy company called Limelight Bio that similarly aimed to "overcome key limitations of current gene therapy."
Limelight was later closed, a spokesperson confirmed to BioPharma Dive, and Intergalactic is "a completely separate and totally different company."
Ehlers estimates Intergalactic is about a year to 18 months away from the preclinical studies that would enable human testing, and said the company could begin its first clinical trial as early as within two years.
Intergalactic will first target diseases affecting the eye, for which its research is most advanced, as well as respiratory diseases and cancer. But other diseases could be considered as well, said José Lora, the company's chief scientific officer.
"If there is imaging technology that actually can get into that tissue, we can get there as well," Lora said. "It's a matter of engineering" the electrical field delivery system for each tissue type, he added.
The eye, which is segmented off from some of the body's systems, is a safe starting point, one chosen by a number of gene therapy and gene editing companies. Luxturna, the first gene replacement therapy approved in the U.S., is for a form of childhood blindness. Editas Medicine, which uses CRISPR gene editing, recently unveiled the first data from a trial of its therapy for another type of eye disease.
Intergalactic, which employs about 30 people, is also launching with a five-year manufacturing deal in place with Resilience, a newly built manufacturer for biotech companies backed by ARCH Venture Partners.
Ehlers will serve as CEO. Joining him and Lora at Intergalactic are Eileen Higham, formerly of Sana Biotechnology, as head of technical operations; ex-Akili Interactive executive Vincent Hennemand as chief operation officer; and Robert Farra, previously chief executive of a maker of wearable insulin patch, as chief engineer.