Senior Scientist

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

AskBio doses first patient in Phase 1/2 gene therapy trial for late-onset Pompe disease. AskBio has dosed the first participant in a Phase 1/2 clinical trial evaluating AB-1009, an investigational AAV-based gene therapy for late-onset Pompe disease. AskBio has dosed the first participant in PROGRESS-GT LOPD, a Phase 1/Phase 2 clinical trial evaluating investigational gene therapy AB-1009 for the treatment of late-onset Pompe disease (LOPD).[1] The milestone advances the company's efforts to develop a potentially durable gene therapy approach for the rare neuromuscular disorder, which continues to present significant unmet medical needs despite the availability of enzyme replacement therapies (ERTs). AB-1009 is an adeno-associated virus (AAV)-based investigational gene therapy designed to enable sustained production of acid alpha-glucosidase (GAA), the enzyme deficient in patients with Pompe disease.[1] The therapy is being developed by AskBio, a wholly owned and independently operated subsidiary of Bayer AG.[1] The open-label PROGRESS-GT LOPD trial is expected to enroll approximately 12 adult participants across sites in the United States.[2] Investigators will evaluate the therapy's safety, tolerability, and preliminary efficacy in patients with LOPD.[2] Addressing long-term challenges in Pompe disease. Pompe disease is a rare inherited lysosomal storage disorder caused by mutations affecting production of GAA, leading to glycogen accumulation in muscle cells.[3] In LOPD, patients typically experience progressive skeletal muscle weakness and respiratory decline that worsens over time.[3] "Current treatment approaches may not fully meet the long-term needs of patients, and ongoing research into potential new options remains critical." - Tahseen Mozaffar, MD Although multiple recombinant human GAA enzyme replacement therapies have been approved, clinical limitations remain.[1] Some patients experience declining therapeutic response over time, requiring ongoing infusions and increasing treatment burden.[4] Researchers have increasingly explored gene therapy approaches as a potential strategy for sustained enzyme production and reduced dependence on chronic ERT administration. "Current treatment approaches may not fully meet the long-term needs of patients, and ongoing research into potential new options remains critical," said Tahseen Mozaffar, MD, director of the UCI Health ALS & Neuromuscular Center and principal investigator for the AB-1009 clinical program, in the company announcement.[1] According to AskBio, AB-1009 is designed to address the underlying genetic cause of Pompe disease by enabling continuous endogenous production of GAA following a single administration.[1] Regulatory momentum supports program development. The investigational therapy has already received both FDA Fast Track and Orphan Drug designations, reflecting regulatory recognition of the significant unmet need in Pompe disease.[1] The FDA also accepted the investigational new drug application for AB-1009 earlier this year.[1] Fast Track designation is intended to facilitate development and expedite review of therapies targeting serious conditions with limited treatment options.[5] Orphan Drug designation may provide incentives including market exclusivity, tax credits, and regulatory support for therapies addressing rare diseases.[6] "Reaching this milestone reflects the momentum behind our gene therapy platform and our commitment to working toward improving quality of life for people with rare diseases," said Canwen Jiang, MD, PhD, chief development officer and chief medical officer at AskBio, in the company statement.[1] The clinical advancement also highlights continued investment in neuromuscular gene therapies, an area that has seen substantial growth across both biotech and pharmaceutical sectors over the past several years. Gene therapy continues expanding in rare disease. AAV-based gene therapies have emerged as a major area of focus in rare genetic disease research, particularly for neuromuscular disorders caused by single-gene mutations.[7] Developers are increasingly targeting diseases such as Duchenne muscular dystrophy, limb-girdle muscular dystrophy, spinal muscular atrophy, and Pompe disease using viral vector platforms intended to provide long-term protein expression after a single treatment.[7] Despite growing momentum, challenges remain, including durability of expression, immune responses, manufacturing complexity, and long-term safety monitoring.[7] Still, industry investment continues to accelerate as companies pursue therapies capable of transforming chronic rare diseases into potentially manageable conditions with one-time administration approaches. For patients with LOPD, the initiation of the PROGRESS-GT LOPD study represents another step toward evaluating whether gene therapy may offer a more durable alternative to lifelong enzyme replacement treatment.