Julia Vitarello, whose daughter Mila received a custom-designed genetic therapy in 2016, announced this week she is forming a new biotechnology company to mass-produce individualized treatments for rare mutations. The venture comes just months after her previous startup, EveryONE Medicines, shut down due to insufficient regulatory clarity from the U.S. Food and Drug Administration.
“We proved that one child can be saved with a bespoke molecule. Now we must prove it can be done for hundreds,” Vitarello said in an interview. “The science is there; the business model and FDA pathway are not—yet.”
Background
Mila Makovec was diagnosed with Batten disease, a fatal neurodegenerative disorder, caused by a unique mutation in the CLN7 gene. In 2016, Vitarello partnered with Boston Children’s Hospital and a biotech firm to create milasen, an antisense oligonucleotide tailored exactly to Mila’s genetic error. The therapy stabilized her condition for years, a landmark in personalized medicine.
In 2019, Vitarello founded EveryONE Medicines to replicate this approach for other patients. The company sought to develop “n-of-1” therapies under a dedicated FDA framework. But in early 2024, EveryONE folded. Vitarello cited a key reason: the FDA’s 2023 draft guidance on individualized therapies did not provide a clear enough reimbursement or approval pathway to attract sustained investor interest.
“Investors need to see a path to return. The guidance was a step forward, but it left too many risks unaddressed,” she explained.
New Venture, Same Mission
Vitarello’s new company—name not yet disclosed—aims to overcome those barriers. The business model will focus on modular manufacturing and shared regulatory infrastructure to lower costs per therapy. She is currently seeking a new set of funders, including venture philanthropists and strategic partners.
“We’re not asking for a special break; we’re asking for a consistent framework that allows us to plan,” Vitarello said. “EveryONE failed, but its mission didn’t. We’ve learned what investors need to see.”
What This Means
The collapse of EveryONE and the birth of a successor highlight a critical gap in the emerging field of precision genetic medicine. While the FDA has acknowledged the potential of bespoke therapies, the lack of a formal approval mechanism—akin to the Orphan Drug designation but for n-of-1—stifles commercial viability.
If Vitarello succeeds, it could unlock treatments for thousands of patients with ultra-rare mutations who currently have no options. However, experts warn that scaling production while maintaining quality and affordability remains a massive engineering challenge.
“This is a test case for whether personalized medicine can be industrialized,” said Dr. Anne Chung, a genetic medicine researcher at Stanford University who is not involved in the venture. “The science is ready; the ecosystem is not. Julia is trying to build that ecosystem.”
Key hurdles ahead
- Establishing a regulatory framework that allows rapid, repeated approvals of individualized drugs.
- Developing scalable manufacturing processes for short-run RNA and DNA therapies.
- Securing reimbursement from insurers and health systems that typically demand large clinical trials.
Vitarello remains undeterred. “Mila is 14 now. She defied the odds. We owe it to every family like ours to keep pushing.”