Sarepta pauses Elevidys shipments
Sarepta Therapeutics has voluntarily paused Elevidys shipments in the US for non-ambulatory Duchenne muscular dystrophy (DMD) patients. The decision follows an informal request from the FDA and reflects safety concerns, especially liver-related risks. The company initially halted dosing for non-ambulatory patients in June for an expert advisory committee to assess enhanced immunosuppressive protocols and to align with the FDA on label updates. CEO Doug Ingram said, “As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of Elevidys was a painful one, as individuals with Duchenne are losing muscle daily and in need of disease-modifying options. It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the Elevidys label supplement process.”
FDA RMAT designation for gene therapy
The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Genascence Corporation’s gene therapy candidate GNSC-001 for knee osteoarthritis. GNSC-001 is a first-in-class, recombinant AAV vector delivering an optimized human interleukin-1 receptor antagonist (IL‑1Ra) via a single intra‑articular injection. By blocking IL‑1, GNSC-001 has been shown to sustain long-term therapeutic levels of IL‑1Ra. Previously awarded Fast Track status in late 2024, GNSC‑001 has shown promising safety and biomarker results in two trials: a phase I dose-escalation in nine subjects, and the DONATELLO phase Ib trial. A phase IIb/III trial designed in collaboration with FDA is expected to start in 2026. The RMAT designation offers benefits including early FDA guidance, rolling review, and potential priority and accelerated approvals.
Catapult selects Spliceor for funding boost
UK-based biotech spin‑out Spliceor has received investment from the Cell and Gene Therapy Catapult under the inaugural Cross‑Catapult Investment Pilot. This represents the first seed-stage award through the initiative, designed to bolster early-stage UK advanced therapy firms. The funding will support Spliceor’s trans-splicing gene therapy platform, currently focused on treating hepatocellular carcinoma (liver cancer), and help expand Spliceor’s team and advance pre-clinical validation of its precision gene-repair approach.
A multidisciplinary committee selected Spliceor based on its science, therapeutic aim, and potential patient impact.
Matthew Durdy, CGT Catapult CEO, said, “Spliceor is an innovative advanced therapies company with the potential to develop a life-changing gene therapy to benefit patients. By working with Innovate UK to invest in promising companies, like Spliceor, that have strong technologies and clear applications, we hope to accelerate their growth, boost the advanced therapies ecosystem in the UK, and ultimately lead to more therapies entering clinical trials and commercial use.”
Partnership to boost Australian CGT landscape
Australia-based organizations Viral Vector Manufacturing Facility and Cell Therapies have signed a memorandum of understanding to strengthen the country’s capabilities in developing and manufacturing Advanced Therapy Medicinal Products. This partnership aims to enhance patient access to innovative cell and gene therapies, support clinical trials, and bolster Australia's competitiveness in the global advanced therapies market. The agreement focuses on fostering a robust and export-ready value chain, leveraging the complementary strengths of both companies. VVMF specializes in viral vector production, while Cell Therapies brings extensive experience in GMP manufacturing of various cell-based therapies.
Cell Therapies CEO Bev Menner said, “This collaboration advances efforts to secure a fully integrated onshore value chain for advanced therapies in Australia—enhancing patient access to cutting-edge treatments, reinforcing clinical trial and translational capabilities, and enabling export-readiness across the APAC region. Strategic investment in sovereign manufacturing capacity for CGTs and critical inputs positions Australia to compete in the global advanced therapies market.”
Key Genes Suppress Colon Cancer Growth
A study by researchers at the University of Fukui, Japan, reveals that the homeobox transcription factors CDX1 and CDX2 play a crucial tumor-suppressive role in colon cancer by repressing cancer stemness. Loss of CDX1, or combined loss of CDX1 and CDX2, enhanced stem cell-like features and invasiveness in colon tumors. Mechanistically, CDX1/2 reduced expression of key cancer stemness genes like LGR5, CD44, and c-MYC by binding downstream of the LGR5 transcription start site. Although chromatin remained open, CDX1/2 disrupted the β-catenin-mediated assembly of the RNA polymerase II (Pol II) transcriptional machinery, specifically the DSIF and PAF1C complexes. This repression was mediated through the CDX1/2 homeodomains, independently of their transactivation functions. The findings suggest that DSIF and PAF1C serve as a regulatory hub integrating oncogenic (β-catenin) and tumor-suppressive (CDX1/2) inputs to control colon cancer stemness, offering insight into transcriptional control of tumor progression.
In Vivo prime editing takes on AHC
Researchers at the Broad Institute and the Jackson Laboratory have demonstrated that in vivo prime editing can correct ATP1A3 mutations responsible for alternating hemiplegia of childhood, a rare and severe neurodevelopmental disorder. Researchers employed both prime and base editing to fix five prevalent mutations in human cells and two mouse models. In patient-derived induced pluripotent stem cells, editing efficiencies ranged from 43-90 percent, with minimal off-target effects. Using adeno-associated virus vectors, prime editing was delivered to newborn mice, achieving up to 85 percent correction in brain regions, restoring ATPase activity, and dramatically improving survival and neurological function. Treated mice exhibited reduced seizure-like episodes, enhanced motor coordination, and improved cognitive behaviors. Notably, this one-time treatment outperformed traditional gene therapy approaches.
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