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20 | InTouch AUTUMN 2026 RESEARCH NEWS Preclinical results indicate up to 40% exon skipping efficiency. Initial discussions were held regarding a New Zealand–based clinical trial, though the company is currently prioritizing the U.S. and Europe, with potential for Oceania involvement if early trials succeed. Keros Therapeutics A clinical-stage biopharmaceutical company developing treatments for diseases linked to dysfunctional TGF-β superfamily signalling. Pipeline includes KER-065, a Phase I program targeting neuromuscular diseases, with readouts expected in 2026. A collaboration pathway has been established to explore bringing a Phase I/II regenerative drug trial to New Zealand, with NDAs currently underway. Satellos Bioscience A clinical-stage drug development company focused on small-molecule therapies that restore muscle regeneration by correcting defects in muscle stem-cell polarity, with lead candidate SAT-3247 in clinical development for DMD. Actively conducting clinical work in Australia. Sarepta Therapeutics Maintains an industry-leading genetic medicine pipeline including RNA-based technologies and gene therapies for rare neuromuscular conditions. Active siRNA programs include SRP- 1001 (FSHD1), SRP-1003 (DM1), and SRP-1005 (Huntington’s disease), several of which are entering Phase I/II cohorts in 2026. Scholar Rock A late-stage biopharmaceutical company pioneering therapies that selectively inhibit myostatin, enabling muscle-targeted treatments for SMA and broader neuromuscular disorders. Apitegromab has shown statistically significant motor-function benefits in Phase 3 SMA studies, positioning myostatin inhibition as a potential new standard of care. Vertex Pharmaceuticals A biopharmaceutical company pursuing programs targeting the underlying genetic mechanism of DM1 through via VX-670. VX-670 is in Phase 1/2 clinical evaluation for DM1, assessing safety, pharmacokinetics, and pharmacodynamics across global study sites. A cell-therapy concept for Duchenne is in early research. A new era: Gene editing for neuromuscular diseases Precision BioSciences is a clinical-stage gene editing company leveraging its proprietary ARCUS® nuclease platform to develop one-time in-vivo gene editing therapies. Its DMD program, PBGENE-DMD, aims to permanently correct mutations in exons 45–55, addressing ~60% of DMD patients and restoring production of near full-length dystrophin. Recently received regulatory clearance to proceed with a gene-editing clinical trial (PBGENE-DMD) for Duchenne muscular dystrophy, following announcements made at conference events. Summary: A New Zealand voice The TREAT-NMD Conference in Lisbon provided a significant platform to represent MDANZ and advocate for the needs of people living with neuromuscular conditions. Key themes included evolving clinical trial practices, challenges in managing patient and investor expectations, and the growing importance of preclinical research and transparent communication. Discussions with global biopharmaceutical companies highlighted promising therapeutic pipelines and opportunities for future New Zealand involvement. Engagement with international advocacy groups also opened potential collaborations aimed at strengthening patient representation, improving trial readiness, and ensuring New Zealand’s inclusion in emerging global treatment pathways. Following my attendance at the conference, I have been asked to be a Committee Member on the Neuromuscular Disease Advisory Committee (NMDAC) and the Standard Operating Procedures (SOP) Library, representing New Zealand with a scientific voice in the global research arena. The TREAT-NMD Conference in Lisbon provided a significant platform to represent MDANZ and advocate for the needs of people living with neuromuscular conditions. Angus Lindsay, PhD Principal Investigator, Neurology of Myopathies (NoM) Lab Sir Charles Hercus Health Research Fellow, University of Canterbury Chairperson, Neuromuscular Research Foundation Trust