A Mother’s Mission: From a Single Bespoke Medicine to a Scalable Platform
Eight years ago, Julia Vitarello’s daughter Mila received a one-of-a-kind medication—a bespoke therapy designed specifically for Mila’s rare disease-causing genetic mutation. Now, Vitarello is embarking on a new venture: building a company that can produce these individualized therapies at scale, turning what was once a desperate hope for one child into a viable pathway for many.
The Story of Mila and the Birth of N-of-1 Medicine
Mila suffered from a rare neurological disorder caused by a unique mutation in the MEF2C gene. With no existing treatment, Vitarello teamed with researchers to create a custom antisense oligonucleotide (ASO) therapy—a drug tailored to correct Mila’s specific genetic error. The success of this approach, often called “Mila’s Miracle,” sparked widespread interest in n-of-1 medicine, where drugs are designed for a single patient.
This personalized strategy holds incredible promise for the estimated 7,000 rare diseases, many of which involve ultra-rare mutations that would never attract commercial drug development. However, as Vitarello discovered, turning a one-off success into a reproducible process is fraught with challenges.
The Rise and Fall of EveryONE Medicines
After Mila’s treatment, Vitarello co-founded EveryONE Medicines, a startup aimed at creating a platform to develop custom ASOs for multiple patients simultaneously. The company attracted investors and began working with academic labs to identify candidates. Yet earlier this year, EveryONE shut down. According to Vitarello, the key reason was that new regulatory guidance from the U.S. Food and Drug Administration (FDA)—while encouraging the development of customized therapies—did not go far enough to provide a clear approval pathway that satisfied investors’ risk and return expectations.
“Investors need to see a replicable, scalable business model,” Vitarello explained. “The FDA’s guidance was a positive step, but without a defined regulatory framework for these personalized therapies, venture capital remains hesitant.”
The Regulatory Hurdle: Why Bespoke Medicines Are Hard to Scale
The FDA’s 2021 draft guidance on individualized ASO therapies outlined a path for sponsors to submit data for a single patient under an investigational new drug (IND) application. However, it did not create a dedicated approval category like “customized drug product” with streamlined requirements. For companies like EveryONE, which aimed to manufacture dozens of different ASOs each year, the per-patient regulatory burden remained prohibitively high. Each new ASO essentially requires its own IND, toxicology studies, and manufacturing validation—costs that multiply linearly with each patient.
“The science is ready,” Vitarello noted, “but the business model isn’t—not without regulatory innovation that matches the manufacturing flexibility.”
Starting Anew: The Vision for a Scalable Biotech
Undeterred, Vitarello is now in the early stages of forming a new company, whose name has not yet been disclosed. The goal is to leverage lessons from EveryONE and recent technological advances to create a more efficient platform for developing n-of-1 therapies at a fraction of the cost and time.
Central to the new venture is a strategy that integrates several innovations:
- Modular manufacturing: Using standardized production processes that can be quickly adapted for different ASO sequences.
- AI-driven design: Machine learning algorithms that predict optimal ASO candidates for a given mutation, reducing trial-and-error in the lab.
- Shared toxicology data: Building a library of safety information across many ASOs to extrapolate risk, rather than requiring full studies for each one.
- Partnerships with academic centers: Leveraging existing research networks to identify patients and mutations more rapidly.
“We’re not trying to reinvent the wheel for each patient,” Vitarello said. “We’re building a chassis that can accommodate many different engines.”
Investor Interest: Cautious but Growing
While the new company is still seeking funding, Vitarello reports that several venture firms and philanthropic organizations have expressed interest. The landscape has shifted since EveryONE’s inception: more proof-of-concept studies have been published showing that n-of-1 ASOs can be safe and effective, and the FDA has initiated pilot programs like the Support for Clinical Trials Advancing Rare Disease Treatments (START) pilot. These changes may make investors more willing to take a chance on a scalable personalized medicine platform.
“EveryONE was ahead of its time,” said Dr. Karen Hastings, a biotech venture partner not involved in the new company. “Now the regulatory and scientific conditions are maturing. Julia’s new effort could be the one that cracks the code.”
What Would Help? Calls for Regulatory Evolution
Vitarello and other advocates are pushing for further FDA action. Suggestions include:
- Creating a dedicated “customized therapeutic” approval pathway with flexible evidence standards.
- Allowing master INDs that cover a family of ASOs with shared chemical backbones.
- Establishing a public-private consortium to pool resources for safety testing.
Such changes would reduce the per-patient regulatory cost and make venture financing more predictable. Vitarello hopes that her new company can demonstrate the model’s viability even under the current framework, thereby encouraging further regulatory reform.
A Personal Drive That Resonates
For Vitarello, this is not just a business endeavor. Mila, now a teenager, is doing well on her custom medicine—but she is one of many. Thousands of families around the world are waiting for their own “Mila’s miracle.” The new company represents a second chance to deliver hope on a broader scale.
“The first time was personal—it was for my daughter,” Vitarello said. “Now it’s personal for every family who has a child with a mutation that has no name, no advocacy group, and no drug. We owe it to them to make this work at scale.”
Looking Ahead: Key Milestones
Over the next 12 months, Vitarello aims to secure initial seed funding, assemble a scientific advisory board, and identify the first two or three patient mutations to target. If successful, the company could begin clinical manufacturing within two years. “We’re moving as fast as we can, but we need the ecosystem—regulators, investors, academia—to move with us.”
The story of Mila began with a desperate mother and a determined scientist. That collaboration produced a breakthrough for one child. Now, that same mother is working to ensure that no child is left behind by the one-size-fits-all model of drug development.
This article is adapted from original reporting available to STAT+ subscribers.