Why We Exist – The RareMoon Mission

Not long after experiencing a rare disease in my immediate family for the first time, I found myself wondering—what more could I do?

At the time (2009), I didn't know the term "rare disease" or that millions of people around the world were facing the same heartbreak and uncertainty that I felt when my father was diagnosed with Glioblastoma (GBM). I was 27 and in the early years of my career working in drug discovery and development. I had yet to realize that an entire industry sector was dedicated to helping patients like my dad. My grandfather passed away from GBM in 2019. My best friend in 2023 by the same disease.

In 2013, I took my first role in orphan drug regulations—and everything changed. In 2016, I co-founded RareMoon Consulting. As chance would have it, my co-founder had also lost two friends to GBM and shared the same focus: to make a difference. We have since dedicated our careers and professional focus to using our experience and understanding of orphan drug regulations to navigate the unique challenges of rare disease drug development.

Every day, we get to work alongside a team dedicated their entire careers to fighting for patients like my dad—scientists, doctors, and regulatory specialists who have been with us since day one. They have spent their entire careers in the rare disease space, guiding programs from concept to clinic and ultimately into the hands of patients. I am proud to collaborate with these individuals who write and operate with excellence, who navigate complex endpoint and manufacturing challenges, help justify a path forward when non-clinical models are unavailable, and ultimately work to overcome these common challenges many of our clients face.

I am humbled to be part of such a passionate group of people, a team of partners in the fight against rare diseases.

The Challenges of Rare Disease Drug Development

The stakes are high, the timelines are critical, and the hurdles are unique.

• Small Patient Populations – Traditional clinical trial models don't apply. Data is limited, and recruitment can be challenging.
• Limited Regulatory Precedent – With so few approved therapies for rare diseases, companies must navigate a path with little historical data or information.
• Complex Science – Many rare disease treatments involve gene therapies, cell therapies, or precision medicine approaches requiring subject-matter expertise.
• Uncertain Regulatory Expectations – The FDA, EMA, and other health agencies recognize the urgency of rare disease therapies, but the evolving nature of regulations requires strategic, early engagement.

For biotech teams developing orphan drugs, these challenges can feel overwhelming. As a team dedicated to supporting orphan drugs and advanced therapies, we regularly engage with regulators. Our extensive experience and deep expertise enable us to anticipate challenges, navigate complexities, and operate within—and occasionally around—the regulatory framework, bringing life-changing therapies closer to patients.

Our Commitment

Our mission is clear: To guide orphan drug and advanced therapy programs through the regulatory framework, driving them toward pivotal milestones that support biotech innovation and ultimately benefit patients.

Our commitment is unwavering: To push forward—alongside regulators, biotech innovators, and patient advocates—because we believe in the mission of bringing novel therapies to those who need them most. RareMoon's team isn't just part of the rare disease ecosystem; we are deeply embedded in the journey, working relentlessly to overcome obstacles and deliver solutions that make a difference.

Excellence in Everything We Do

Our team comprises individuals who have dedicated their entire careers to the rare disease space. They bring unparalleled expertise in:

Our Impact in the Rare Disease Community

Since our founding in 2016, RareMoon has guided dozens of rare disease programs through critical regulatory milestones. We've helped bring multiple therapies to market, advancing treatments for conditions that previously had no approved options.

Our approach is built on three foundational principles:

• Deep Scientific Understanding: We immerse ourselves in the science behind each therapy
• Regulatory Expertise: Our team includes former FDA reviewers who understand agency perspectives
• Patient-Centered Focus: Every decision is made with patient benefit at the forefront

"On Rare Disease Day and every day, we remain committed to making a lasting impact in this space—because when we work together, we don't just advance science, we change lives."

Looking Forward: Our Continued Mission

The rare disease landscape continues to evolve, with new scientific breakthroughs and regulatory pathways emerging regularly. Gene therapies, cell therapies, and precision medicine approaches are transforming what's possible for rare disease patients.

As we look to the future, RareMoon remains committed to:

• Advancing innovative regulatory strategies for emerging therapeutic modalities
• Building bridges between biotech innovation and regulatory approval
• Advocating for patient access to life-changing treatments
• Sharing our expertise to elevate the entire rare disease community

Join Us in the Fight

The work we do at RareMoon isn't just about regulatory consulting—it's about hope. Hope for families facing devastating diagnoses. Hope for researchers working tirelessly to find answers. Hope for a future where rare disease patients have access to the treatments they desperately need.

Every program we guide, every regulatory hurdle we help overcome, and every therapy we help bring to market represents progress in this fight. We're not just moving science forward; we're moving toward a world where rare diseases no longer mean rare hope.

This is why we exist. This is our mission. And we won't stop until every rare disease patient has the chance for a better tomorrow.