GondolaBio

Last week, GondolaBio's Mackenzie McGrath and BridgeBio's Kirsten Kiefer headed to Colorado for the National Organization for Rare Disorders Living Rare conference! It was a truly moving and eye-opening experience to connect with patients and families affected by rare disorders. We’re grateful to be part of the conversation around putting patients first and ensuring their experiences help shape the future of care.

“What drives me is a love of life. I want to experience everything, even though I’ve got porphyria.”   Today is #GlobalPorphyriaDay. Pete was diagnosed with #ErythropoieticProtoporphyria (EPP) at just two years old. Living with EPP means navigating a life where even brief exposure to sunlight can trigger excruciating pain and swelling. For Pete, this means avoiding sunlight and taking significant precautions to protect his skin, like using blinds, traveling at night and wearing protective clothing.   Wearing full protective clothing outdoors can also come with an emotional toll. Pete knows he is often seen as different, and the attention, stares, and negative interactions that come with needing to fully cover up can make everyday situations feel uncomfortable and isolating.   Despite these challenges, Pete’s story is one of resilience, adaptation, and determination to keep moving forward without letting EPP define his life.   We’re grateful to Pete and the British Porphyria Association for bringing his EPP story to light and helping raise awareness for the community.

Today marks World #NF1AwarenessDay, a life-altering genetic condition that can present in both visible and invisible ways. Listen below as Aaron shares his experience living with NF1, reflecting on the challenges and resilience that have shaped his journey. From navigating social pressures at a young age to making a massive decision that ultimately improved his quality of life, his journey is a reminder that no two experiences are the same. At GondolaBio, we’re committed to working on behalf of the NF1 community and re-engineering the future of NF1 disease management.

Preeclampsia, a serious pregnancy condition marked by high blood pressure, affects 1 in 20 women worldwide. It remains a leading cause of maternal and infant illness and mortality. At GondolaBio, we’re committed to advancing therapies where the need is greatest. This #WomensHealthMonth, we’re proud to announce our focus on developing a treatment for #preeclampsia. https://lnkd.in/eYDUR_2j

May is #TSCAwarenessMonth. To kick off this important month, we’re honored to continue sharing Riona’s story. Tuberous Sclerosis Complex (TSC) is a rare, genetic condition that can affect multiple body systems such as the skin, eyes, brain, kidneys, heart, and lungs. Despite many daily challenges, Riona's family enjoys an active life together and remains hopeful for the future. In this short film, we see more of what living with TSC is like. View more of Riona's story here. https://lnkd.in/eGkhN_BV Thank you again to the TSC Alliance for helping us share Riona’s story.

This weekend, the GondolaBio team attended the FD/MAS Alliance Research Priorities Workshop in Philadelphia! This meeting brought together clinicians, researchers, and patients and caregivers impacted by fibrous dysplasia and McCune-Albright Syndrome to shape the FD/MAS research agenda and identify the most pressing needs. Thank you to all who made this a successful gathering.

Putting patients first isn’t just a value at GondolaBio, it’s how we approach every decision, every program, and every step forward. We’re driven by the urgency of unmet need and the responsibility to translate science into therapies that truly matter, especially for those living with rare and underserved genetic conditions. Our work is grounded in the belief that innovation succeeds only if it improves real lives. This starts with listening to lived experience and ensuring those insights actively shape the therapies we develop. Because we believe in building alongside the communities we serve, not just for them.

Tomorrow is the night! We’re proud to sponsor Mission: Cure’s Moments of Hope gala, bringing the #pancreatitis community together for a night to honor the strength of patients, celebrate progress, and support what’s to come. Learn more about this free, virtual event here. https://lnkd.in/ef7XyUR7

We are proud to be a platinum sponsor of this week’s Mission: Cure Moments of Hope gala. #Pancreatitis is a devastating disease with limited treatment options, and we share the urgency to change that. We invite the community to join us at this free virtual event. Find more details from Mission: Cure below.

This weekend, we joined the TSC Alliance at the 25th annual Comedy for a Cure at the Irvine Improv! Each year, this event raises awareness and funds to advance research and support for the #TSC community, with a little good-humored fun along the way. We are honored to support this event and TSC Alliance's mission.