Looking to take advantage of 'silenced' enzymes, SalioGen emerges from stealth with eyes set on gene therapy 3.0
Gene therapy has made big strides over the years, from the first generation AAV-based therapies to the CRISPR/Cas9 technology that has now taken the scientific world — and Nobel Prize committee — by storm. But where exactly does the future, or gene therapy 3.0, lie?
That’s the question a new biotech is aiming to answer, as SalioGen Therapeutics emerges from stealth Monday morning with a $20 million Series A. And they believe they’ve found a new delivery system that can more precisely deliver genes in vivo than the relatively large adeno-associated virus or CRISPR system: mammalian-derived enzymes.
Monday’s round was led by PBM Capital and included other undisclosed investors.
Based in Burlington, MA, SalioGen came about when CEO Ray Tabibiazar and co-founder Joseph Higgins, who worked on the Human Genome Project in the 1990s, set out roughly a year and a half ago looking to find what the natural evolution of gene therapy might be. They believed that while CRISPR and other technologies like Precision Bio’s ARCUS were promising, researchers could really only go after disorders that involve bigger genes with specific mutations, as those platforms involve cutting, knocking out or replacing specific DNA sequences.
The pair identified three issues that needed solving in the current gene therapy field in order to get the company off the ground. First was the gene editing itself, or how SalioGen could take a different approach than what’s already out there. Second is the delivery method: Tabibiazar and Higgins felt they couldn’t rely on the AAVs. And last was how to make manufacturing cheaper in order to make the therapies, which often run up several hundred thousands of dollars in costs, more accessible.
Through these steps came SalioGen’s proprietary Exact DNA Integration Technology platform, or EDIT. The goal, Tabibiazar told Endpoints News, is to essentially pluck these mammalian enzymes that have been “silenced” over the course of evolution and repurpose them into delivering the gene therapy. And the whole procedure of taking genes and putting them into new cells takes less than a month, sharply cutting down on production costs.
“It puts the genetic code into the genome, and then it’s done, it’s gone,” Tabibiazar told Endpoints.
Not only does that address the three issues they’d hoped to fix, it allows for a genome to be rewritten inside the body without the worry AAVs or Cas9s will continue making changes after the targeted gene has been fixed. Tabibiazar is calling this process “gene coding,” in that it puts new genetic code back into one’s genome through the enzyme delivery.
He likened it to a software and hardware upgrade analogy — the enzymes, or the software, contains the update and fixes the issues in the body’s hardware.
“If you have an Apple [product] and you get Apple software, Apple hardware, you’re not going to use the app on Android,” Tabibiazar said. “It’s the same thing here, if you have a mammalian enzyme, which rewrites the software into your mammalian hard drive, you want it to be a fit between the software and the hardware.”
Though Tabibiazar says the application of the platform is quite wide, SalioGen will be focusing on familial hypercholesterolemia and inherited macular degeneration first. In FH, the company is taking what it sees as a new approach, aiming to replace the entire receptor gene in the liver responsible for the dangerously high cholesterol and LDL levels associated with the disease. It’s a field with a few players already, with Verve and Precision Bio both going after HoFH.
Both programs are still in the preclinical stage, but Tabibiazar hopes they can be in the clinic within the next two to two-and-a-half years. The key now is ensuring the therapies can be safe and building out the platform with the financing. Tabibiazar said having PBM Capital on their side, who also made early-stage investments in the biotechs that created Luxturna and Zolgensma, provides a crucial validation for SalioGen.
“All our focus is to do it very specific,” Tabibiazar said. “Not only do we know how to target it to a specific cell, we know how to target it to a specific location within the genome.”