BridgeBio

Sean, who lives with ATTR-CM, recently joined our #OnRarePodcast and spoke about the many symptoms he experienced before receiving a diagnosis. He noted that finding out about his condition was a tremendous sense of relief, but a big change for him that he chose to look at positively. Learn more about Sean’s story: https://bit.ly/3VQUPEo?

Ivan, a longtime physician, mistook the initial signs of ATTR-CM as part of aging. But after feeling out of breath from routine activities such as climbing stairs, Ivan suspected something more serious was happening. After receiving his ATTR-CM diagnosis, Ivan has learned to embrace a slower pace of life, but says he has no plan of giving in to the disease. Learn more about his story: https://bit.ly/3yFDYwU ??

We are excited to announce that the U.S. FDA approved our orally-administered near-complete (≥90%) stabilizer of TTR for the treatment of adult patients with transthyretin cardiac amyloidosis (ATTR-CM).? Next, we're looking to pursue approvals globally in Europe, Japan, and Brazil, and will continue exploring the full potential of this treatment. We are proud of the opportunity to serve patients within this community. Learn more: https://bit.ly/3V5wVp4

The team Aspa Therapeutics celebrated the important work of National Tay-Sachs & Allied Diseases Association (NTSAD), at the 2024 Imagine & Believe event in Cambridge, MA. We were proud to be present alongside families, advocates, researchers, clinicians, and industry partners, all united by the shared vision of a future with effective treatments for Tay-Sachs, Canavan, GM1, and Sandhoff diseases. This inspiring event raised critical funds to support NTSAD’s essential family services, programs, and research. Learn more about NTSAD’s important work at www.ntsad.org.?

We're thrilled to have presented positive initial outcomes from the ATTRibute-CM open-label extension (OLE) study of acoramidis in ATTR-CM at #AHA24. ATTRibute-CM was designed to evaluate the efficacy and safety of acoramidis, an investigational, near-complete, orally-administered, small molecule stabilizer of TTR. Results from the ATTRibute-CM OLE continue to showcase the potential of acoramidis. Read more here: https://bit.ly/4fNnL8P

We're pleased to announce that positive 18-month results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with #achondroplasia, were published as an original research article in the New England Journal of Medicine (NEJM) today. Infigratinib is an oral small molecule designed to inhibit FGFR3 signaling and target achondroplasia at its source. Read more about the announcement here: https://bit.ly/3YWTSMz

Caregivers are often those who take on the day-to-day work of supporting loved ones living with conditions like limb-girdle muscular dystrophy type 2I (LGMD2I/R9). They juggle responsibilities, make countless sacrifices, and act as a source of strength through every high and low.? During #NationalCaregiversMonth, we share our deepest gratitude to these caregivers who do so much for others.? A special thank you to the CureLGMD2i Foundation for sharing this video with us and for their commitment to supporting people living with LGMD2I/R9 and the people who care for them.??

Recently, BridgeBio Gene Therapy’s Srivatsan Ramesh was interviewed by the Cell & Gene Therapy Insights (CGTI) on Process Optimization for AAV-Based Gene Therapy. We were excited to hear Srivatsan share insights on key challenges in downstream AAV purification and how BridgeBio is harnessing novel technologies to accelerate the translation of therapies to clinical manufacturing. A few highlights from the interview include: Tackling complex challenges in downstream purification Leveraging advanced tools for faster clinical readiness Staying true to our mission to bring transformative therapies to patients Thank you, CGTI, for this opportunity to spotlight our ongoing efforts and priorities in gene therapy manufacturing! Read the interview here: https://bit.ly/3UM6kgY #GeneTherapy #AAV #Biomanufacturing #ProcessOptimization #BridgeBio #CGTI

We’re excited to share details of our upcoming participation at #AHA24! The BridgeBio team will be engaging in discussions on the latest innovations in cardiovascular science and medicine with the American Heart Association. We’ll also be presenting a featured science presentation on our outcomes data through 42 months from the open-label extension of ATTRibute-CM, our Phase 3 Study of acoramidis for ATTR-CM and three moderated digital poster presentations. Explore the details below, and learn more: https://bit.ly/4eYVDPW

This past weekend, we had the privilege of attending the first The Little Legs Big Heart Foundation Heart Skeletal Dysplasia Conference—an unforgettable event filled with learning, laughter, and lasting connections.?A very special thank you to Kristen DeAndrade, founder of the Little Legs Big Heart Foundation, for uniting key voices in the skeletal dysplasia community, including children, siblings, parents, advocates, and healthcare professionals. We’re grateful to have been part of such a meaningful and enriching experience.