In 10, 9, 8 … let the countdown begin for Intergalactic Therapeutics as the biotech launches with $75 million of rocket fuel to fly toward the clinic with a non-viral gene therapy that might have once seemed an astronomical aspiration.
As reaching outer space becomes less of a dream and more of a commercial reality, Intergalactic hopes to replicate those increasing chances at success in the biotech world with a gene therapy that doesn't integrate into the genome and cause the adverse immune reactions that have lured the watchful eye of the FDA.
The Boston startup emerged Thursday out of incubator Apple Tree Partners (ATP) with a view to improve patients' vision and cancer outlooks as well as to achieve potentially enhanced outcomes in other diseases. It's the second time CEO Michael Ehlers, M.D., Ph.D., is attempting to get an ATP portfolio company to treat retinal and other diseases with gene therapies, except this time it's a non-viral approach.
Ehlers left his post as head of Biogen's research and development in autumn 2019 to become chief scientific officer of ATP and CEO of its $75 million-funded gene therapy portfolio biotech Limelight Bio. That startup has since shuttered its doors and returned certain rights on rare retinal disease gene therapies to University of Pennsylvania, the life sciences venture firm said in a statement.
Now, Ehlers is taking another shot at the gene therapy stars.
This time around, Intergalactic's platform includes a synthetic DNA that doesn't integrate into the genome, which is a problem with some viral gene therapies that led the FDA to hold an advisory committee meeting to discuss future studies of such treatments last month. This format allows the gene therapy to express large genes as well as multiples of genes with the ability to re-dose. Intergalactic also built a system that aims to deliver the treatments to where they are specifically needed rather than to undesired areas.
The biotech already has a strategic partner to aid in its cell-free manufacturing vision: a five-year deal with cell and gene therapy manufacturer National Resilience, which will produce DNA products for clinical trials, Intergalactic said in conjunction with the financing. You might recognize that name from Resilience's high-profile partnership with Moderna last month to help manufacture the mRNA biotech's COVID-19 vaccine in Canada. Resilience was also behind stem cell biotech Garuda's $72 million series A last month.
The telescope, er, microscope, is first directed at ophthalmological conditions, but Intergalactic also has its eyes on potentially treating tumors, respiratory diseases, kidney conditions, cardiology, hepatology, central nervous system conditions and musculoskeletal disorders. Intergalactic has data on various in vivo systems, and it "wouldn't be surprising" if the biotech took its clinical trial ambitions to the FDA within two years, said José Lora, Ph.D., chief scientific officer, in an interview with Fierce Biotech.
The biotech's approach is fully synthetic, which means Intergalactic can build the molecules without having to carry bacterial or viral sequences, Lora said. The executive's resume lends well to this work; he was previously CSO of non-viral gene therapy biotech enGene and before that was vice president of research at synthetic biology company Synlogic.
ATP asked the question: “What if we could make a truly novel synthetic DNA form that precisely mimicked endogenous chromatin and gave us persistent expression?" Ehlers said.
It's not the first time ATP has launched a space-themed company. One could say Galaxy Medical is ATP's Neil Armstrong and Intergalactic is the incubator's Buzz Aldrin.