BridgeBio Pharma has surged 209% over the past two years, yet Barclays analyst Eliana Merle sees potential for another 95% gain. The optimism centres on Attruby, the company's heart drug approved in late 2024 for transthyretin amyloid cardiomyopathy. In the first quarter of 2026, Attruby generated $180.6 million in US revenue. More than 7,800 patients received prescriptions from over 1,850 prescribers. Barclays projects $912 million in US sales for 2026, roughly 10% above consensus estimates. BridgeBio has two additional products awaiting FDA decisions in 2026 and 2027. Wall Street expects revenue to nearly double this year to approximately $960 million, with profitability anticipated in 2027.

The Speak Foundation announces 2026 LGMD Scientific Summit. Jul 27, 2026, 10:00 ET Global researchers, clinicians, regulators, biotechnology leaders, and patient advocates will convene to advance LGMD research and therapeutic development WASHINGTON, July 27, 2026 /PRNewswire/ - The Speak Foundation today announced the 2026 LGMD Scientific Summit, an international scientific meeting that will convene leading researchers, clinicians, biotechnology innovators, regulators, and patient advocates to discuss recent advances in Limb-Girdle Muscular Dystrophy (LGMD) research, clinical development, and therapeutic innovation. The virtual event will be held July 31, 2026. The Summit comes at a pivotal moment for the LGMD field as multiple therapeutic programs advance through clinical development, new biomarkers emerge, and innovative clinical trial approaches continue to reshape rare disease research. Scientific sessions will highlight gene therapy, regenerative medicine, biomarker qualification, natural history studies, patient-focused drug development, and collaborative strategies designed to accelerate new treatments for individuals living with LGMD. FEATURED SCIENTIFIC LEADERS Featured researchers include Douglas Sproule, MD, MSc (BridgeBio Neuromuscular); Louise Rodino-Klapac, PhD (Sarepta Therapeutics); Barry Byrne, MD, PhD (University of Florida); John Vissing, MD, DMSci (University of Copenhagen); Nicholas Johnson, MD, MSCI, FAAN (Virginia Commonwealth University); Peter Kang, MD (University of Minnesota); Tahseen Mozaffar, MD, FAAN (University of California, Irvine); Simone Spüler, MD (Charité - Universitätsmedizin Berlin); Brad Williams, PhD (Jain Foundation), and Elizabeth McNally, MD, PhD (Northwestern University). Additional scientific and clinical presentations will be delivered by representatives from biotechnology companies and nonprofit research organizations advancing therapies for LGMD, including BridgeBio Neuromuscular, Sarepta Therapeutics, AskBio, Genethon, MyoPax, Edgewise Therapeutics, Advertent Biotherapeutics, Kinea Bio, Myogenica, and Cure Rare Disease. PATIENT-CENTERED DRUG DEVELOPMENT A featured panel will bring together representatives from the U.S. Food and Drug Administration and the patient community to examine patient-centered clinical trial design, stakeholder engagement, and collaborative approaches to accelerating therapeutic development for rare neuromuscular diseases. SCIENTIFIC PERSPECTIVES Nicholas Johnson, MD, MSCI, FAAN Professor and Executive Vice Chair, Department of Neurology Virginia Commonwealth University "We have entered a transformative period for LGMD research. Scientific advances are creating opportunities that were unimaginable only a decade ago, but realizing their full potential will require continued collaboration, rigorous science, and a shared commitment to ensuring that no LGMD subtype is left behind." Douglas Sproule, MD, MSc Chief Medical Officer, BridgeBio Neuromuscular "We are deeply grateful to The Speak Foundation for convening the LGMD community and fostering collaboration among patients, caregivers, clinicians, researchers, and industry, while ensuring the patient perspective remains central to scientific progress. We also thank the individuals and families who have participated in our clinical studies and entrusted us with their time and hope. It is a privilege to contribute to advancing therapies for LGMD, and that trust reinforces our commitment to rigorous science and the urgent development of meaningful new treatments." The Speak Foundation gratefully acknowledges BridgeBio Neuromuscular, Platinum Sponsor of the 2026 LGMD Scientific Summit, and AskBio, Bronze Sponsor, for their support and commitment to advancing LGMD research and therapeutic development. Registration is open to researchers, clinicians, biotechnology professionals, regulators, patients, caregivers, and members of the global LGMD community. To view the program and register, visit TheSpeakFoundation.com/LGMD-Summit. ABOUT THE SPEAK FOUNDATION Founded in 2008, The Speak Foundation is a patient-led nonprofit organization dedicated to improving the lives of individuals and families affected by Limb-Girdle Muscular Dystrophy through advocacy, research, education, and public policy. The Foundation convenes the global LGMD community by bringing together patients, researchers, clinicians, industry, regulators, and policymakers to accelerate research, improve access to specialized care, and advance therapeutic development. Signature initiatives include the International LGMD Conference, LGMD Scientific Summit, LGMD Scientific Workshop, LGMD Day on the Hill, LGMD News Magazine, and the LGMD Centers of Excellence. SOURCE The Speak Foundation

Rinascera Therapeutics launches with clinical-stage pipeline to transform the treatment of genetic skin diseases. - Initial financing led by Double Point Ventures with participation from Olive Tree Capital, Civilization Ventures and Toba Capital - - Supports advancement of RIN-001 for Gorlin Syndrome and RIN-002 for Dystrophic Epidermolysis Bullosa (DEB) - BOSTON, July 16, 2026 (GLOBE NEWSWIRE) - Rinascera Therapeutics, a clinical-stage biotechnology company developing transformative therapies for rare genetic skin diseases, today announced its launch and an initial financing led by Double Point Ventures, with participation from Olive Tree Capital, Civilization Ventures and Toba Capital. The Company's pipeline reflects a disciplined strategy of developing scientifically differentiated therapies for rare genetic skin diseases with well-defined biology, significant unmet medical need, and the potential to meaningfully improve patients' lives. RIN-001 is a novel topical kinase inhibitor being developed as a cancer interception therapy for Gorlin Syndrome and patients with high-frequency sporadic basal cell carcinomas. RIN-002 is an intravenous recombinant human collagen VII replacement therapy being developed as a systemic treatment for Dystrophic Epidermolysis Bullosa (DEB). Rinascera acquired RIN-002 from BridgeBio in connection with the Company's formation and financing. Both programs have generated clinical proof-of-concept evidence, with later-stage clinical development planned. Rinascera was incubated by Olive Tree Capital under the leadership of co-founder and Chief Executive Officer Nichola Eliovits with a vision to build a biotechnology company dedicated to patients living with rare genetic skin diseases. The Company's name is inspired by the Italian verb rinascere ("to be reborn") and the Latin root renasci ("to be born again"). "People living with diseases like Gorlin Syndrome and DEB are not asking for marginal improvements. They are asking for lives with less pain, fewer surgeries, fewer wounds, fewer limitations and more possibility," said Nichola Eliovits, co-founder and Chief Executive Officer of Rinascera Therapeutics. "We created Rinascera to develop therapies that address the biological foundations of rare genetic diseases and give patients and their families a genuine second chance at life. With RIN-001, we seek to intercept BCC before surgery becomes necessary. With RIN-002, we seek to move DEB treatment beyond the individual wound by systemically replacing collagen VII across affected tissues." RIN-001: Intercepting BCC before surgery becomes necessary Gorlin Syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome (NBCCS), is a rare inherited disorder caused primarily by mutations affecting the sonic hedgehog signaling pathway, most commonly the PTCH1 gene. These mutations drive constitutive pathway activation and predispose patients to develop basal cell carcinomas (BCCs), the most common form of skin cancer. Unlike most people, who may experience an isolated BCC, patients with Gorlin Syndrome can develop dozens to hundreds of tumors over their lifetimes, resulting in lifelong surveillance, repeated biopsies and surgeries, scarring, pain and persistent anxiety about the next tumor. RIN-001 is a first-in-class topical kinase inhibitor being developed as a cancer interception therapy designed to treat early-stage BCCs before they progress to surgical eligibility. Rinascera believes this approach has the potential to shift the treatment paradigm from repeated surgical intervention toward earlier, localized and non-invasive disease management. Cancer interception is an emerging therapeutic approach that seeks to identify and actively treat cancer at its earliest stages, before progression to more advanced disease requiring greater intervention. RIN-001 is designed to apply this concept directly to BCC by treating early lesions before surgery becomes necessary. The RIN-001 program has generated preclinical and clinical evidence of prompt and visible responses across treated BCCs. Beyond Gorlin Syndrome, Rinascera sees potential opportunities in patients with high-frequency sporadic BCCs who develop numerous tumors over their lifetimes. RIN-002: Systemic collagen VII replacement for DEB Dystrophic Epidermolysis Bullosa (DEB) is a group of inherited disorders caused by mutations in COL7A1, the gene encoding collagen VII, a structural protein essential for anchoring the epidermis to the underlying dermis and supporting the integrity of other epithelial tissues. Patients with DEB can experience painful blistering, chronic wounds and scarring; in more severe forms, patients may also experience nutritional complications, esophageal strictures, corneal erosions, osteoporosis, and an elevated risk of aggressive squamous cell carcinoma. Patients with DEB may live with dozens or even hundreds of chronic wounds at any given time, many of which heal slowly or incompletely and can lead to progressive scarring, contractures and, in severe cases, loss of digits. DEB includes both Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Dominant Dystrophic Epidermolysis Bullosa (DDEB), which share an underlying defect in collagen VII but can vary considerably in severity. RIN-002 is being developed as an intravenous recombinant collagen VII replacement therapy designed to address the underlying loss of functional collagen VII systemically. Unlike localized approaches intended to treat individual wounds, Rinascera's strategy for RIN-002 is to advance an intravenous protein replacement therapy capable of targeting the cutaneous and systemic manifestations of DEB. The RIN-002 program has generated preclinical and clinical evidence supporting systemic collagen VII deposition after intravenous infusion of recombinant collagen VII and encouraging signals of wound healing and broader disease benefit. Rinascera intends to apply disciplined development, manufacturing and regulatory strategies for RIN-002, building on the substantial work already completed by BridgeBio. A rare disease company built with commercial discipline Rinascera is building a commercially disciplined rare disease company with the potential to establish multiple specialty dermatology franchises. RIN-001 addresses a population characterized by repeated surgical intervention, significant healthcare utilization and substantial quality-of-life burden, with potential expansion into additional high-frequency skin cancer populations. RIN-002 addresses DEB as a systemic genetic disease affecting the skin and other epithelial tissues, with a systemically delivered collagen VII replacement therapy designed to address disease across affected tissues rather than individual wounds alone. Together, the programs reflect Rinascera's strategy of building differentiated therapeutic franchises around diseases with clear biological foundations, substantial patient need and focused commercial opportunities. "At Double Point Ventures, we invest in companies that we believe can fundamentally change the standard of care for patients," said Dan Yadegar, Managing Partner at Double Point Ventures and a member of Rinascera's Board of Directors. "Rinascera combines exceptional leadership, differentiated clinical-stage programs and a disciplined strategy focused on diseases with substantial unmet need and clear underlying biology. We believe RIN-001 and RIN-002 provide the foundation to build a durable rare disease company capable of delivering meaningful benefits for patients while creating significant long-term strategic value." Leadership Rinascera's founding leadership team includes co-founder and Chief Executive Officer Nichola Eliovits, co-founder and Chief Operating Officer Max Dawson, and co-founder and Executive Medical Advisor Karl Beutner, M.D., Ph.D. "Gorlin Syndrome and DEB are very different diseases, but they share an important feature: both are rooted in well-understood biology, and both impose enormous lifelong burdens on patients and their families," said Karl Beutner, M.D., Ph.D., co-founder and Executive Medical Advisor of Rinascera Therapeutics. "Rinascera approaches these diseases with a translational strategy grounded in the patient experience and the clearly defined genetic drivers of disease. This is the right way to build medicines in rare dermatology." About Rinascera Therapeutics Rinascera Therapeutics is a biotechnology company developing transformative therapies for patients living with rare genetic skin diseases. The Company's clinical-stage pipeline includes RIN-001, a topical cancer interception therapy being developed for Gorlin Syndrome and high-frequency sporadic BCC populations, and RIN-002, a systemic recombinant collagen VII replacement therapy being developed for Dystrophic Epidermolysis Bullosa (DEB). Rinascera's mission is to give patients and their families a genuine second chance at life. For more information, please visit www.rinascera.com. Investor Contact