BridgeBio

Many people living with limb-girdle muscular dystrophy (#LGMD) wait years for an accurate diagnosis.    Even after being told that they have LGMD, the journey doesn’t end there because knowing the exact subtype is essential to managing care.    One form, LGMD2I/R9, is caused by mutations in the FKRP gene, and it can’t be confirmed without genetic testing.    There are no approved therapies for LGMD2I/R9, but early, accurate diagnosis can open doors to options and hope.    Learn more about how to access no-cost testing: https://lnkd.in/eRVbcmzW     

We’re pleased to announce that we will participate in fireside chats at the following upcoming healthcare investor conferences: 📅 Piper Sandler Healthcare Conference, New York, NY Tuesday, December 2 at 10:30 am ET 📅 Evercore ISI HealthCONx Conference, Miami, FL Wednesday, December 3 at 3:00 pm ET 🔗 https://bit.ly/4oZw3iX

We’re excited to introduce new updates to MyAchonJourney! This one-stop educational resource, developed in collaboration with advocacy leaders and healthcare experts, now includes guidance for two new age groups: • Middle childhood (ages 6-12) • Teenage years (ages 13-17) These additions build on existing resources for early childhood, ensuring families have access to reliable information at every stage of their journey. Explore the latest updates here: myachonjourney.com

At our BridgeBio October Town Hall, we celebrated recent clinical milestones in LGMD2I/R9 and ADH1 programs. We also recognized continued commercial progress of our therapy for ATTR-cardiomyopathy. Guided by our values of putting patients first, thinking independently, and making every minute count, leaders across R&D, Commercial, and our international team shared what this moment means and how we are preparing for the road ahead. We are grateful to every teammate, partner, clinician, and advocate who keeps patients at the center of everything we do. Together, we remain focused on translating scientific progress into meaningful impact for the communities we serve.

🎧 “We want to help as many patients as possible with new and meaningful medicines.” Our founder & CEO, Neil Kumar joined Luke Timmerman on The Long Run Podcast for an inside look at how we build medicines differently from strategy to scale. In this episode, Neil discusses: 🔬 Objective function & strategy: A disciplined focus on genetic diseases, NPV-positive programs, and first- or best-in-class medicines 💡 Momentum across programs: Positive readouts in LGMD2I/R9 and ADH1 both in the same week 🧬 Modality-agnostic toolbox: From small molecules to gene therapy, using the right tool for each disease Listen to the full conversation to hear how BridgeBio is redefining what it means to translate genetic discoveries into medicines that matter: https://bit.ly/4o5r66M

We were proud to join the cardiovascular community at the American Heart Association (AHA) Scientific Sessions 2025 (#AHA2025), sharing our 10 digital moderated posters, connecting with peers, and learning from the latest advancements in heart science. Thank you to the AHA community for the meaningful discussions, collaboration, and shared dedication to improving outcomes in cardiovascular disease. ❤️ We look forward to continuing the momentum together.

This #NationalFamilyCaregiversMonth and every month, we honor those whose care makes progress possible. Behind every patient story is a network of caregivers from parents to partners to siblings to friends, who navigate the daily realities of rare disease with strength and compassion. At BridgeBio, we are deeply grateful for the caregivers who advocate, listen, and persevere alongside their loved ones. Your resilience and insight remind us why we pursue science with urgency, empathy, and purpose. 💜

A clinical exam alone can’t confirm LGMD2I/R9. Diagnosing LGMD2I/R9 requires genetic confirmation, but many #LGMD patients still go years without the right testing.    Now, no-cost genetic testing is available for patients and providers.    ➡️ Learn more:  https://bit.ly/3VnyNJM 

Millions of people live with a rare disease, yet <10% have an approved treatment. Our new analysis shows how economics, not science alone, limit development. By shifting key levers such as genomics, genetic validation, policy, and financing, we can expand the frontier of feasibility and unlock 500+ underserved diseases. Read the paper:https://bit.ly/48aJINZ

We’re excited to share that Aaron C. Daugherty, Ph.D., Associate Director of Computational Biology at BridgeBio, will moderate a panel at the AI Drug Discovery & Development Summit. The session will explore how knowledge graphs powered by AI can integrate genetic, proteomic, and clinical data into unified models that accelerate target discovery and disease understanding and will discuss strategies for data normalization and curation that ensure biological datasets are AI-ready, clean, and accurate, paving the way for faster and more meaningful therapeutic development. At BridgeBio, we move when the data is clear and the purpose is urgent because that’s how patients get what they need, faster.