iNFixion seeks NF1 treatment by enhancing what the body is doing right

The BIO 2026 Start-up Stadium winner targets a rare disease.

As Herb Sarnoff sits in the hospital, waiting for a pain specialist to see his daughter, he describes how a mutation on just one side of the NF1 gene causes neurofibromatosis type 1 (NF1).

“You have one good NF1 gene producing the neurofibromin protein your body needs, and one missing or mutant allele that isn’t, so every cell is not getting enough normal NF1 protein,” he explains. The result is a wide range of difficult symptoms, including painful tumors, bone conditions like scoliosis and osteoporosis, high rates of cancer, and neurocognitive manifestations such as learning disabilities, autism and sleep dysregulation.

Since discovering his daughter’sNF1, a rare disease impacting 1 in 3000 people, nearly 25 years ago, Sarnoff has slowly transformed himself from a tech entrepreneur with limited knowledge of biology to leading iNFixion Bioscience, a biotech developing treatments for NF1. Infixion’s novel NF1 approach aims to supercharge the function of the one good allele rather than trying to fix the mutant one. And the judges were impressed enough to award Infixion a Start-Up Stadium top prize at the 2026 BIO International Convention.

The award recognizes just two start-ups—from among 50 presenting—that investor judges deem to be most promising. iNFixion Bioscience won the Emerging Company Category (between $1 million-$10 million raised). The Seed-Stage winner, with less than $1 million raised, is BrainStorm Therapeutics.

iNFixion’s innovation

Gene therapies often seek to correct mutations, but as Sarnoff explains, “fixing” mutant NF1 genes is difficult. “The gene itself is extremely large: 320,000 base pairs of DNA,” he says, adding that sequencing has identified over 5,000 unique NF1 gene mutations, with no hotspots. “So gene therapy for NF1 is really, really challenging.”

Instead, iNFixion is targeting the healthy allele, increasing the amount of NF1 protein it can produce by blocking microRNAs that modulate protein expression.

“MicroRNAs are basically a feedback loop regulating protein in the body by putting a brake on protein production when necessary,” Sarnoff explains. “If you can block the right microRNAs, you’re essentially taking the brake off. But microRNAs are not specific to one gene. So if you take out a microRNA, you’ll likely have off-target effects.”

Instead of completely blocking the targeted microRNA, iNFixion seeks to block its function exclusively in the NF1 gene.

“The approach being pursued utilizes antisense oligonucleotides (ASOs) that block microRNA binding only on NF1 mRNA. This prevents that microRNA from putting a brake on NF1 protein production,” Sarnoff explains. “But that microRNA is still free to work on other genes, thus limiting potential off-target effects.”

iNFixion has shown in early cellular in vitro experiments that its ASO can boost NF1 protein expression by up to 70-80%. They have also adapted their ASO from the human version to a mouse version and shown they can boost NF1 protein expression in the brains of mice. The next step is to optimize for safety and efficacy in both human NF1 organoids and NF1 animal models.

The drive for funding

As with all start-ups, iNFixion is constantly seeking funding to continue its work. Thus far, most of the work has been funded by the National Institutes of Health (NIH), Department of Defense (DoD), and foundation grants, Sarnoff says.

“We’ve also been fortunate to get lab space at J&J’s San Diego incubator called JLABS,” he says. “It’s a prestigious and collaborative environment for drug discovery start-ups.”

As he seeks future funding, Sarnoff is hopeful of attracting investor interest. One advantage: Even though NF1 is a rare disease it is not that rare, affecting 125,000 Americans and 2.5 million worldwide.

“So it’s a bit of a sweet spot,” he explains. “We get all the benefits of orphan disease, including special treatment at the FDA, access to priority review vouchers and R&D tax incentives, but it’s a big enough market that there’s definitely interest from pharma.”

Sarnoff says iNFixion is in discussions with pharmaceutical companies interested in iNFixion. As for venture capital, Sarnoff notes that few VC’s provide early-stage funding for orphan disease, although a handful of professional investors are showing interest.

Personal motivation

Having explained the disease and Infixion’s approach, Sarnoff begins to grow impatient waiting for his daughter’s pain specialist. Managing pain is a big part of NF1, according to Sarnoff, who says his daughter has had chronic pain for over 10 years related to tumors and bone issues.

His daughter has had 10 major surgeries for scoliosis and tumor removal, and lives with neurocognitive “executive functioning issues,” Sarnoff says. “She’s certainly had her challenges along the way.”

Sarnoff isn’t the only one in his company with personal motivation. Of the seven members in iNFixion’s core team, Sarnoff and three others are NF1 parents, including a pathologist and PhD scientist.

“I made a commitment back in 2017. I said, ‘I’m going to do this.’, and I’ve been working full time on it since,” he says.

An inventor with several patents and previous CEO experience launching a (retail/tech) start-up, Sarnoff is comfortable in the start-up world, but biotech was new.

“I’ve had to start a new career, learning biology and drug discovery, which were completely foreign,” he explains. “I just started gathering resources like any start-up. You begin with an idea and limited resources, and you start building pieces.”

iNFixion is now winning prizes and impressing investors, and Sarnoff clearly seems dedicated to building the other pieces needed for Infixion’s success—and his daughter’s health.

The post iNFixion seeks NF1 treatment by enhancing what the body is doing right appeared first on Bio.News.