EUCOPE Member Spotlight: Q&A with Stephanie Manton, CEO & Co-founder of WinQure Medicine

EUCOPE regularly spotlights a member company and the great work they’re doing to advance the life sciences industry and drive innovation to serve patients better. In July, we spoke with Stephanie Manton, CEO & Co-founder of WinQure Medicine.

Stephanie Manton is the CEO & Co-founder of WinQure Medicine, a biotechnology company developing precision antisense oligonucleotide (ASO) therapies for patients with ultra-rare genetic diseases. She holds a DPhil from the University of Oxford in ultra-rare genetic diseases and has spent her career at the intersection of RNA biology, venture creation, and biotechnology strategy. At WinQure, she is leading the development of a scalable platform designed to make precision ASO therapies commercially viable for patient populations that have traditionally been considered too small for conventional drug development.

 

Tell us about your organisation and its mission and how you drive innovation internally?

WinQure Medicine develops precision ASO therapies for patients living with ultra-rare genetic diseases. Our goal is to make it commercially viable to develop treatments for patients who have historically been considered to have too few patients for traditional drug development.

Innovation at WinQure is not just about the science. We are building a development platform that reduces cost and timelines, allowing programmes that would not be commercially viable on their own to become viable as part of a broader portfolio. That means rethinking everything from programme selection and manufacturing to regulatory strategy and patient access.

For ultra-rare ASO therapies, the real innovation is not the molecule. It is the development process that makes these therapies possible.

How do your organisation’s activities help patients now and into the future?

Our aim is to give patients the opportunity to receive a treatment, regardless of how rare their disease is.

In the short term, we in-license and advance programmes that have been initiated through philanthropic funding, grants, academia, patient foundations, or rare disease organisations. Many become stranded before toxicology and manufacturing, which are often the most expensive stages of development. Our platform is designed to take these programmes through to the clinic and ultimately to the broader patient community. Resources in the ultra-rare disease field are limited, so we believe every euro invested should translate into the greatest possible patient impact.

In the longer term, we will also develop programmes from the earliest stages, beginning with the identification of eligible patient variants and progressing them through our scalable development platform.

What do you see as the biggest challenge facing the life sciences industry today?

The science is advancing remarkably quickly. In many cases, our ability to identify therapeutic targets now exceeds our ability to develop and deliver treatments efficiently.

For ultra-rare diseases, the biggest challenge is no longer discovering therapies. It is creating regulatory, reimbursement, and commercial frameworks that make them viable to develop. Unless those frameworks evolve, many promising therapies will never reach patients.

What are the major health policy issues and themes that you are most focused on in 2026?

Our priority is ensuring that regulation and patient access evolve alongside scientific innovation.

Precision medicines require development pathways that are proportionate to very small patient populations. They also require more efficient reimbursement systems. Today, companies must negotiate patient access separately in each European country. For therapies that may only serve one or two patients in a given Member State, this creates a level of cost and complexity that is difficult to sustain.

If Europe wants to remain a leader in precision medicine, regulatory, HTA, and reimbursement processes need to become more proportionate and more streamlined. Otherwise, companies may ultimately be forced to prioritise larger markets over countries with only a handful of patients, which would be a failure for everyone involved.

What attracted you to join EUCOPE and how can we help you achieve your business goals?

EUCOPE brings together companies developing innovative medicines for patients with high unmet medical need, making it a natural fit for WinQure.

We joined because the barriers we face are structural rather than scientific, and those barriers cannot be solved company by company. Regulatory proportionality, sustainable reimbursement, and workable patient access all depend on frameworks that no single developer can shape alone. EUCOPE gives us a forum to work alongside other companies facing the same challenges and to help build the policy environment that ultra-rare therapies need, not just for WinQure, but for the field as a whole.