Brian O Mahony Shares Amit Nathwani’s Research from BIC 2025 on FVII and FIX Gene Therapies for Hemophilia
Brian O Mahony, CEO of Irish Haemophilia Society, Former President of WFH and EHC, shared on X:
”Nathwani from UCL BIC 2025 on their original FIX Gene Therapy trial.
First PWH now 15 years post. Average 13 years.
Stable expression of 2-5%FIX.ABR decreased from average 14 to 1.5. 7 of 10 off prophylaxis.
Reduction in ABR cumulative with decreased microbleeds and synovial inflammation over time.
2 unrelated malignancies. 1) PWH had liver biopsy at 10 years post- showed stable FIX transgene in liver in approximately 5% of hepatocytes transduced”
”Nathwani BIC 2025 on UCL plans to begin FVII Gene Therapy clinical trial in 2026.
Similar vector design as haemophilia B. cDNA small size at 1.4Kb so fits easily into AAV.Dose low at 6E11 vg/kg.
Eligible- severe FVII grade 3, at least 16 years of age, male or female
Interestingly, looking at cost only reimbursement model supported by foundations in UK and US so should be affordable if development works”
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