FSHD行业协作组织新增四家生物医药赞助方并推进临床工具研究

FSHD Industry Collaborative Expands with Four Biopharma Sponsors and Reports Rapid Advances Across Multiple Muscular Dystrophy Research Programs
- The growing multi-stakeholder Collaborative brings together biopharma sponsors, academic experts, technology partners, and funders to advance facioscapulohumeral muscular dystrophy (FSHD) research
- Altay Therapeutics, Epicrispr Biotechnologies, Sarepta Therapeutics, and Scholar Rock have joined the FSHD Industry Collaborative
- Sponsors support further advancing clinical endpoint data analysis, blood biomarker discovery, biomarker assay validation, and novel MRI analytics to accelerate FSHD drug development
- The Collaborative calls on additional pharmaceutical and biotech company sponsors, technology partners, researchers, philanthropic investors, and mission-aligned donors to join
ABINGTON, Mass. and VANCOUVER, BC, July 30, 2026 /PRNewswire/ -- The FSHD Society, SOLVE FSHD, and the FSHD Clinical Trial Research Network (FSHD CTRN) today announced significant momentum for the FSHD Industry Collaborative, including the addition of four new biopharma sponsors and meaningful early progress across all active research workstreams. Launched in April 2026, the Collaborative is a first-of-its-kind, pre-competitive initiative generating insights that will improve how FSHD clinical trials are designed, executed, and evaluated, supporting the approximately one million people worldwide living with FSHD.
The Collaborative welcomes new sponsor members Altay Therapeutics, Epicrispr Biotechnologies, and Sarepta Therapeutics. Together with foundational funding support from Solve FSHD, and initial biopharma sponsor Scholar Rock, the Collaborative commits to advancing FSHD therapeutic development through shared investment in pre-competitive research, demonstrating a collective drive to find treatments for FSHD.
"Scholar Rock is proud to be the initial sponsor of the FSHD Industry Collaborative. It represents a model of how to integrate existing data sources in the rare disease space to accelerate development of therapies for patients," said John Staropoli, MD, PhD, Vice President of Clinical Research at Scholar Rock.
A Growing Coalition for FSHD
Rather than supporting a single research focus, the Industry Collaborative is designed to address common challenges facing all FSHD therapeutic developers, including biomarker qualification, understanding endpoint sensitivity, patient stratification, and regulatory readiness. Each new Collaborative sponsor brings one or more active programs in FSHD, and all are contributing funding, expertise, and strategic guidance toward shared initiatives that benefit the broader FSHD community while reducing development risk for individual programs. The Industry Collaborative provides a mechanism to collectively generate evidence, validate tools, and establish best practices that can benefit multiple development programs simultaneously.
"The pace of progress we can impact through the FSHD Industry Collaborative demonstrates what is possible when industry, researchers, clinicians, patients, and funders come together around a shared goal. We are excited to contribute to the work addressing challenges that affect every FSHD drug developer. By working collaboratively in a pre-competitive environment, we can accelerate the development of therapies and help bring effective treatments to people living with FSHD faster." Wendy Erler, Senior Vice President, Patient Affairs at Sarepta Therapeutics
Significant Early Progress Across Workstreams
"One of the strengths of the Collaborative is its ability to rapidly launch and execute projects by combining funding, expertise, patient samples, and longitudinal datasets," said Karen Lam, PhD, Vice President, Business Development and Partnerships at SOLVE FSHD. "Within months of launching, we initiated the development of shared resources that will accelerate progress for the entire field."
Since its launch, the Collaborative has moved rapidly to achieve key initial milestones:
- Novel Blood Biomarker Discovery from MOVE Natural History Study: Data and patient samples provided by founding Collaborative member, the FSHD CTRN, has expedited the generation of new high-dimensional proteomic biomarker discovery data from participants enrolled in the MOVE natural history study. This work is identifying candidate blood-based biomarkers that can be linked to imaging, functional, and patient-reported outcomes, with the aim of establishing sensitive and objective measures of disease progression for use in clinical trials.
- Rapid Progress on Blood Biomarker Assay Development and Validation: The Collaborative has made significant early progress in the development and analytical validation of novel FSHD blood biomarker assays in partnership with Immunologix Laboratories. These assays are being developed to regulatory-ready standards, positioning them for use in future clinical development programs. The rapid pace of this work reflects one of the core strengths of the Collaborative: direct access to funding, expertise, patient samples, and clinical datasets enables research projects to be launched and executed with great efficiency.
- New Analysis of Fulcrum's ReDUX4 Clinical Trial Data: We now have access to one of the largest longitudinal MRI datasets ever generated in FSHD. Using clinical trial data generously donated by Fulcrum Therapeutics, collaborative partner Analysis Group has initiated work on a large MRI dataset generated by AMRA Medical. Preliminary findings from this work were presented by Analysis Group at the FSHD Society's 2026 FSHD International Research Congress (IRC) in Chicago on June 25-26, 2026. The goal of this work is to improve endpoint selection and trial design.
- Launch of Novel MRI Analysis with Springbok Analytics: A new MRI analysis workstream has been launched in partnership with Springbok Analytics, with a particular focus on characterizing disease-related changes detectable at early clinical trial timepoints. This work aims to identify sensitive imaging endpoints capable of capturing treatment effects within shorter trial windows — a critical need for the field given FSHD's variable disease progression and the urgency shared by trial participants and developers.
"One of the greatest challenges in FSHD drug development has been the lack of sensitive, validated tools to measure disease progression and treatment benefit," said Amber Salzman, Ph.D., Chief Executive Officer, Epicrispr Biotechnologies. "We're proud to join the FSHD Industry Collaborative and contribute to efforts advancing blood biomarker discovery, assay validation, MRI analytics, and clinical data integration. As we advance EPI-321, the first investigational epigenetic therapy designed to address the root cause of FSHD by silencing DUX4 expression, these initiatives are helping strengthen the tools and infrastructure needed to evaluate therapeutic impact, accelerate clinical development, and ultimately bring meaningful treatments to patients faster."
"Altay Therapeutics is delighted to join the Industry Collaborative," said James Breitmeyer, MD, PhD, CEO of Altay Therapeutics, "as we collaborate with the FSHD Society and SOLVE FSHD to develop our novel oral DUX4 inhibitor DX5057 to initiate clinical trials later this year."
Call for Additional Collaborators
As additional sponsors join, the Collaborative will expand its ability to generate datasets, validate biomarkers, advance imaging analytics, and address emerging challenges in FSHD clinical development. New sponsors have the opportunity to help shape future workstreams while benefiting from the shared infrastructure and insights. The Collaborative continues to welcome additional pharmaceutical and biotech company sponsors, technology partners, researchers, philanthropic investors, and mission-aligned donors. Research priorities are established through a multi-stakeholder governance process involving founding members, industry sponsors, academic experts, and patient-focused organizations.
About the FSHD Industry Collaborative
The FSHD Industry Collaborative is a pre-competitive, multi-stakeholder initiative jointly led by the FSHD Society, SOLVE FSHD, and the FSHD Clinical Trial Research Network (FSHD CTRN). The Collaborative brings together biopharma sponsors, academic experts, technology partners, and funders to generate datasets and insights that reduce clinical development risk, improve patient selection, advance the standardization of clinical measures, and support a unified regulatory narrative for FSHD programs.
About the FSHD Society
The FSHD Society is a patient-driven organization advancing research and clinical development in facioscapulohumeral muscular dystrophy (FSHD). As the world's largest FSHD patient advocacy organization, the FSHD Society funds research, supports patients and families, advocates with regulatory agencies, and accelerates the path to effective treatments. More information is available at fshdsociety.org. The Global FSHD Innovation Hub, a wholly owned subsidiary of the FSHD Society, provides strategic advisory, project management, and contracting services for the Industry Collaborative as well as support for clinical trial execution and design for FSHD trial sponsors.
About SOLVE FSHD
SOLVE FSHD is a venture philanthropic organization dedicated to accelerating treatments for FSHD. SOLVE FSHD provides foundational and strategic funding to de-risk and catalyze the most promising pathways to effective therapies. More information is available at solvefshd.com.
About the FSHD Clinical Trial Research Network (FSHD CTRN)
The FSHD CTRN is a large academic consortium comprising more than 30 academic centers across 10 countries, with deep expertise in FSHD natural history studies and clinical trial design and execution.
SOURCE FSHD Society