The impact of fatigue: Jo’s Story

Fatigue is more than just tiredness. From her social life to her work life, Jo describes how her fatigue symptoms have impacted both.

Lifestyle adjustments to manage fatigue: Jo’s Story

From diary organization to diet management, Jo explains her individual lifestyle adjustments to manage fatigue.

Knowing liver function: Jo’s Story

PBC affects the bile ducts that run through your liver. Liver blood tests that show high levels of ALP (alkaline phosphatase) are a marker of PBC disease progression. Jo explains why understanding what impacts your liver function is an important part of taking charge of your PBC journey.

Finding a community: Jo’s Story

Jo describes the importance of community in PBC management.

Advice to her past self: Jo’s Story

Jo shares the PBC advice she would give to her past self.

Fatigue and brain fog: Wendy’s Story

Fatigue and brain fog can impact everyone differently. Wendy explains what her symptoms can feel like.

Knowing your PBC: Wendy’s Story

Everyone’s PBC is different, and knowledge of your PBC is power. Wendy shares her hopes for her own disease management.

Navigating the diagnosis journey: Gill’s Story

Gill explains the impact that her doctor’s guidance and patient community support had on her diagnosis journey.

PBC symptoms: Gill’s Story

The ‘PBC itch’ is a common and often debilitating symptom of PBC. Gill describes how fatigue and the ‘PBC itch’ have affected her life.

Lifestyle adjustments: Gill’s Story

Just like you are a unique individual, the lifestyle adjustments you make to manage PBC must be specific for you. Gill describes the steps that she takes.

Knowing what to ask your doctor: Gill’s Story

Coming to your PBC appointment prepared with a list of questions can help you get the most out of your PBC care. Gill recounts what she would advise herself when she was first diagnosed.

Her hope for PBC management: Gill’s Story

Gill shares her hope for PBC management.

“My role is to ensure that every product we develop meets the highest standards of quality, safety, efficacy, and compliance.” Robustness is not a box to check—it’s the foundation of every decision. As a Senior Director in the Pharmaceutical Development function in Ipsen’s drug product development team, Prince Korah focuses on driving the pharmaceutical development strategy by overseeing the end-to-end life cycle of drug products with his team—from early development through manufacturing and clinical delivery—ensuring each stage meets rigorous quality, regulatory, and performance standards. His goal is clear: design for stability, simplify where possible, and always center the science.

In development of drug products, the pressure is real. Timelines are short, stakes are high, and there is little room for error. Prince responds by defining and controlling the key variables that influence stability. “We design with the end in mind,” he says. “The patient, the dose, the shelf life—it all has to hold together.”

That mindset requires discipline. Every formulation choice ties back to rigorous quality and regulatory performance standards. Platform strategies help, but only if they serve the product. “If you can build a platform approach, you gain consistency,” he explains. “It allows you to apply lessons across programs.” But he cautions against rigid thinking. “Even within a platform, we ask: can we make it simpler, safer, and more robust?”

Process design is where his work becomes most visible. He collaborates cross-functionally across chemists, formulators, analytics, manufacturing experts, quality, and regulatory experts to define how the drug product is produced, filled, and finished. It is not just about technical accuracy—it is about reliability. If a process fails in production, speed means nothing. “You have to build it to endure,” he says.

Prince looks ahead to a future shaped by continuous manufacturing and AI-driven control systems. He sees the shift toward personalized medicine as a scientific challenge worth solving. “It will change everything we know about production,” he says. “We need to prepare for the scientific rigor now.”

His work remains grounded in one goal: predictability. A well-built product does not call attention to itself. It works every time. “If we do our job right,” he says, “no one should ever notice. It just works.”

We’re proud to share that Ipsen has been certified as a Great Place to Work in 16 countries in 2025, bringing our total to 30 countries worldwide recognized as employers of choice. This achievement is a strong testament to our efforts to build a workplace where everyone can be their true selves, grow and thrive.

A global culture of Collaboration, Excellence and Impact

At Ipsen, boosting a culture of collaboration, excellence and impact is a cornerstone of our strategy. Grounded in Collaboration, Excellence, and Impact, it brings our teams together around a shared purpose — to deliver real impact for patients and society. We collaborate, strive for continuous improvement, and celebrate the passion that drives our progress.

We are dedicated to fostering a safe, supportive and inclusive workplace, where our people have the means, trust, and care they need to perform at their best.

This recognition is a reflection of our culture and our people. It celebrates our progress and inspires us to go further, pursuing our sustainable growth journey, growing together and aiming for greatness. Thank you to all Ipsen employees for your engagement and for making this achievement possible.

*Accredited countries in 2025: Canada, USA, Mexico, Ireland, the Netherlands, Italy, Algeria, Tunisia, Colombia, Brazil, Sweden, Lithuania, Poland, Greece, UAE, Saudi Arabia

At Ipsen, we go beyond environmental responsibility to place societal impact at the heart of our actions. Through a range of Giving-Back Programs, the company aims to make a positive difference in the communities where we operate.

One standout initiative is Ipsen in Motion, a program that brings employees together around a shared cause through physical activity. Whether walking, running, or cycling, every effort is tracked via a dedicated app that collects points. Once the collective goal is reached, Ipsen unlocks a donation to a health-focused organization.

More than just a fitness challenge, Ipsen in Motion fosters team spirit, raises awareness, and promotes empathy and acts. Each year, local challenges take place across the globe to support local associations and spotlight issues that matter to our industry.

This momentum is made possible thanks to a strong network of Ipsen employee ambassadors, who meet monthly to exchange ideas, share local initiatives, and raise awareness on key sustainability topics. Their energy and commitment help bring Giving-Back Programs to life across sites and teams.

This commitment to giving back takes many forms throughout the year, thanks to the dedication of our employees, that makes it all possible. Their commitment brings this initiative to life and helps build a culture of care and impact.

A great example of this spirit was the Ipsen in Motion North America Challenge, which took place across the U.S., Canada, and Mexico. The initiative supported the National Alliance for Caregiving (NAC), a U.S.-based organization that advocates for family caregivers and promotes recognition of their essential role in society. Each region organized its own activities:

• In the U.S., the challenge was launched with a roundtable on caregiving, featuring the National Alliance for Caregiving, which shared key data and insights about caregivers in the country. The initiative concluded with employees coming together for the Half Marathon on November 2nd.

• In Canada, on October 14th, teams gathered for a group walk around Mississauga. A book exchange was also organized, and to ensure the initiative continues, a small library was set up in the kitchen – a great reminder that caring for your health means nurturing both body and mind!

• In Mexico, some employees took part in a Día de los Muertos run on Thursday, October 30th, further strengthening the collective spirit across borders and creating a meaningful moment to come together around a cultural event with colleagues.

Thanks to the incredible energy and commitment of employees across North America and across Ipsen, who collectively reached 600,000 points, NAC received a donation of €15,000. A powerful testament to what we can achieve together.

Through Ipsen in Motion and the broader Giving Back Programs, Ipsen reaffirms that sustainability is also about empowering individuals and strengthening communities.

This Movember, Ipsen once again champions men’s health through our “Get Men Talking” podcast series — a heartfelt initiative encouraging open conversations about prostate cancer and wellbeing.

In this year’s episode, Ipsen’s own Tim Batchelor, now 10 years clear of prostate cancer, sits down with Errol McKeller, fellow survivor and men’s health advocate, and Olympic legend Colin Jackson. Together, they share powerful personal stories, break down stigma, and inspire men everywhere to take charge of their health — because talking can save lives.

The “Get Men Talking” campaign is part of Ipsen’s continued commitment to raising awareness around early screening, prevention, and support for men affected by prostate cancer. Through authentic voices and real experiences, we’re driving an open dialogue that empowers men — and their loved ones — to act early.

👉 Listen to the podcast, share it with your network, and join us in keeping the conversation going.
Together, we can make a difference — one conversation at a time.

Targeting what cannot be seen: Lindsey Rodrigues on the complexity of epigenetics or mutated in a particular cancer. We have to ask, ‘do we have an understanding of how this gene works? Can we design a medicine to treat it or to target it?”’

Lindsey Rodrigues works in the early research space—well before molecules reach the clinic. Her focus is epigenetics, an area where cancer cells are reprogrammed at the level of gene expression, not genetic mutation. “You have these proteins that are responsible for reading, writing, and erasing epigenetic marks,” she says. “And that controls whether a gene is turned on or off. You can’t see it with traditional tools. That’s what makes it so interesting.”

At Ipsen, she leads the biology strategy for early-stage research programs and supports external innovation teams as they evaluate potential assets to add to Ipsen’s pipeline. “You might have two molecules with similar data,” she says, “but very different mechanisms. We’re asking: is this selective? Does it hit the right target? Do we understand the patient population?”

Her background in academic oncology helps guide that analysis. She trained in microfluidics, RNA biology, and CRISPR screening—which is used to run large-scale experiments that show how genes affect a certain trait, disease, or cell behavior. “I try to find something real in the data,” she says. “Especially something robust.”

Lindsey sees epigenetics as a space where good ideas often fail to translate. “It’s a moving goal,” she says. “They are context-dependent. They may work in a cell line, but not in a patient, and the patient population may be narrower than the sponsor originally believed.”

For her, this makes the science more urgent, not less. “There are so many failures,” she says. “But if something works, it’s powerful.”

Her team works cross-functionally from the beginning, engaging with chemistry, clinical, and pharmaceutical development functions to assess what a compound would need to succeed. “We’re not working in isolation,” she says. “We need input across the board.” That collaborative pressure keeps her focused. “We ask: is this a project that we would all be proud to work on? Is this a treatment we would want our families to receive?” Those questions drive her and her team to the finish line of discovery, playing a part in the larger role of medicinal development.

Daily Highlights From The Liver Meeting

Catch up on each day’s key moments and breakthroughs with daily updates from Hugo Gomes da Silva, sharing expert commentary and reflections on the science shaping this year’s meeting. New episodes uploaded daily throughout the congress:

Conversations That Matter: Video Interviews

Hear directly from key opinion leaders, patient advocates, and Ipsen experts as they discuss new data, evolving perspectives, and the future of RLD care – recorded live at The Liver Meeting 2025. Watch exclusive interviews from the meeting:

Why Liver Disease? A Vision for Change

Liver disease has emerged as a critical focus for the healthcare science community, driven by significant unmet needs—particularly in rare and underserved conditions. The liver plays a central role in human biology, acting as a hub for carbohydrate, lipid, and protein metabolism. It is also the origin of many inherited metabolic disorders, most of which stem from autosomal recessive mutations in single genes. These conditions are more common than many realize, with estimates suggesting that one in every 800 live births is affected by a form of inherited hepatic metabolic disorder.

For decades, liver transplantation has been the only curative option available to these patients. However, the landscape is shifting. Early efforts in diseases such as Primary Biliary Cholangitis (PBC), Progressive Familial Intrahepatic Cholestasis (PFIC), and Alagille syndrome have shown that targeted innovation can deliver first-in-class treatments to patients who previously had few or no options. Companies like Ipsen are leading the way, demonstrating how focused investment in hepatology can set new standards for rare disease innovation and patient care.

Smarter Trial Design: Building the Foundation

As the science evolves, so too does the way we design clinical trials. Across the industry, there is a growing emphasis on smarter, more agile approaches to development. These new models are grounded in real-world patient insights and increasingly enhanced by artificial intelligence and real-world data, which enable more accurate predictive modelling.

Rare diseases, by their nature, present unique challenges due to their complexity and heterogeneity. To address this, the industry is embracing adaptive trial designs, single-arm studies with external controls, and Bayesian methodologies. These approaches not only improve study outcomes but also accelerate the path to approval.

Patient-centricity is also becoming a cornerstone of innovation. The development of patient-reported outcomes (PROs), such as the FDA-recognized pruritus tool developed by Ipsen, reflects a broader commitment to ensuring that the patient voice is embedded in every stage of the development process.

Where We Are Today: Expanding the Liver Portfolio

The liver disease space has expanded dramatically in recent years. What was once a field dominated by symptomatic treatments is now home to a growing portfolio of disease-modifying therapies. Advances in precision medicine, the use of novel clinical endpoints, and value-based profiling are helping to tailor treatments more effectively to individual patients.

At the same time, our understanding of disease biology is deepening. Innovations in proteomics and pathway modulation are opening new avenues for intervention. These scientific advances are increasingly aligned with regulatory preparedness, enabling faster and more efficient development pathways. Ipsen and other forward-thinking companies are helping to drive this alignment, ensuring that innovation is not only possible but also accessible.

Looking Ahead: Gene Editing in Liver Disease

Liver diseases caused by single-gene mutations affect millions of people worldwide. While traditional treatments have focused on managing symptoms, they rarely offer a cure. Gene editing is now emerging as a transformative approach, with technologies like CRISPR/Cas9 enabling gene insertion, correction, and knockdown.

However, the liver presents unique challenges for gene editing due to its low cell division rate and inefficient DNA repair mechanisms. A promising solution is Repair Drive, a novel strategy developed by Rice University and Baylor College of Medicine. This approach enhances gene correction efficiency by giving edited cells a survival advantage, allowing them to outcompete diseased cells during natural liver regeneration. Importantly, Repair Drive is compatible with existing delivery platforms such as adeno-associated viruses (AAV) and lipid nanoparticles.

This innovation could mark a turning point in the treatment of pediatric and early-onset liver conditions, shifting the paradigm from symptom management to curative therapies.

The Next Frontier: Regenerating the Liver

Beyond gene editing lies an even more ambitious goal: regenerating liver tissue itself. Cellular regenerative therapies represent the next frontier in hepatology, moving us from treatment to true restoration.

Emerging technologies are exploring bio-delivery systems that can transform liver cells into temporary implantable, tissue-like therapies. One such example is the work being done by Dimension Bio, which is pioneering what it calls “tissue therapeutics.” These therapies have the potential to delay——the need for liver transplantation in cases of liver failure.

This vision of regenerative medicine challenges us to think differently. Are our regulatory and reimbursement systems ready to support such transformative innovation? The answer to that question will shape the future of care.

The System Challenge: Innovation vs. Infrastructure

While scientific progress is accelerating, healthcare infrastructure is struggling to keep pace. Regulatory frameworks are often slow to accommodate novel endpoints and adaptive trial designs. Reimbursement pathways, too, are frequently misaligned with the speed and scope of scientific breakthroughs.

Without systemic alignment, even the most promising therapies risk being delayed or rendered inaccessible. The challenge is not just to innovate, but to ensure that innovation reaches the patients who need it most.

Bridging the Gap: Proactive Engagement

To overcome these barriers, industry leaders are taking a more proactive role in shaping the future of healthcare. This includes engaging with regulators and payers to advocate for adaptive trial models, novel endpoints, and more flexible reimbursement structures.

Collaboration is key. By working together—across industry, regulatory bodies, payers, and patient advocacy groups—we can create an ecosystem that supports innovation from bench to bedside.

Shared Values: Curiosity, Courage, Commitment

At the heart of this transformation are the values that drive the sector forward. Curiosity fuels the exploration of new scientific frontiers. Courage empowers us to challenge conventional thinking. And commitment ensures that we never lose sight of the patients we serve—especially those in vulnerable and underserved communities.

Ipsen exemplifies how these values can be brought to life, combining scientific excellence with advocacy to elevate the voices of patients who are too often overlooked.

Redefining What’s Possible

The journey in liver disease is about more than developing new medicines. It’s about reshaping the future of care. By pushing the boundaries of clinical science and advocating for systemic reform, we can ensure that innovation is not only achieved but also delivered.

The future of hepatology will be defined by how well science and systems evolve together. If we get this right, we won’t just treat liver disease—we’ll redefine what’s possible for generations to come.