Cedric Hermans: Advances in Gene Therapy for Haemophilia A
Cedric Hermans, Co-Chair FVIII/FIX SSC ISTH 2025, shared a post on LinkedIn:
” Gene editing in haemophilia A: renewed reasons for optimism.
Attending the World Federation of Hemophilia’s 19th New Technology and Gene Therapy Workshop in Montreal, marking 30 years of scientific innovation, I was particularly inspired by the presentations on targeted FVIII gene integration.
The investigational MGX-001 approach inserts a FVIII gene into the albumin locus in hepatocytes, harnessing the native albumin promoter to drive expression.
The ambition is sustained FVIII production through stable genomic integration.
Publicly reported preclinical findings are encouraging: FVIII expression sustained for approximately 19 months in nonhuman primates using a surrogate FVIII construct, and activity reaching the normal range with optimized delivery in separate dose-ranging studies.
These remain preclinical results. Human studies must establish whether this approach can deliver lasting efficacy with acceptable genomic and immunological safety.
Nevertheless, they provide a compelling scientific basis for renewed optimism about the future of gene-based treatment for haemophilia A.
An important next step is now approaching: on 10 November 2026, Metagenomi announced FDA clearance of its Investigational New Drug application for MGX-001.
The company plans to initiate the first-in-human phase 1/2 INTEGR8 trial in the first half of 2027, in adults with moderately severe to severe haemophilia A.
For those of us who have followed this field for decades, it is inspiring to see new strategies addressing the challenges of durability.
The ambition of lasting freedom from haemophilia continues to drive meaningful scientific progress.
My thanks to the WFH, the researchers and everyone contributing to these essential discussions.
About the WFH NTGT Workshop
MGX-001 IND-clearance announcement”
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