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”
More from BIC 2025 featured in Hemostasis Today.
-
Nov 3, 2025, 08:23The 1st Participant in A New CHECK-NIRS Clinical Study is Enrolled!
-
Nov 3, 2025, 08:15Bert van Meurs Shares the New Late-breaking Results from the iMODERN Trial
-
Nov 3, 2025, 07:10Lisa Shea Shares Key Takeaways from NORD Summit 2025
-
Nov 3, 2025, 06:49Darshan Doshi on Results of iMODERN and ILIAS ANOCA Studies Presented at TCT 2025
-
Nov 2, 2025, 14:172019 ESC/EAS Guidelines for the Management of Dyslipidaemias Discussion with Lale Tokgözoğlu and Jeanine Roeters van Lennep
-
Nov 3, 2025, 09:13Claudio Carrubba on How Timing Truly Matters in Trauma Embolization
-
Nov 3, 2025, 08:46Rachel Lamerton and Colleagues on Activation of Platelets in Flow Cytometry
-
Nov 3, 2025, 04:56Nirupama Ramadas and Erica Sparkenbaugh on Thromboinflammation in SCD
-
Nov 2, 2025, 14:21Claude Franceschi: I Advise You to Understand the Hemodynamic Pathophysiology of Venous Diseases Rather than Blindly Applying Ablation Techniques or Venous Stent Placement
-
Nov 2, 2025, 14:15Krishna Aragam: Why are (DCM) and Heart Failure About Twice as Common in Individuals of Self-Identified Black Race?
-
Oct 29, 2025, 07:31The Use of Artificial Intelligence to Improve Detection of Acute Incidental Pulmonary Emboli - JTH
-
Oct 29, 2025, 07:05Shrinidhi Nathany: AI in Diagnostic Medicine
-
Oct 28, 2025, 07:02Aya Berman: AI Model for Predicting Bacteremia
-
Oct 28, 2025, 06:55The New Era of Genomics, AI and Cell Therapy in Hematology and Infectious Diseases
-
Oct 28, 2025, 06:49Lukas Gaats: Researchers Are Building Reproductive Organoids
-
Nov 3, 2025, 09:24Stephen Cornelissen: It’s Not Just About Emergencies — It’s About Everyday Care, Chronic Conditions and Hope
-
Nov 3, 2025, 06:57Maria Elisa Mancuso Reflects on 2025 Annual Congress of the Italian Association of Hemophilia Centres
-
Nov 3, 2025, 05:46Chandra Viswanathan: When Blood Safety Fails, We All Bleed - A Call for Reform and Responsibility
-
Nov 2, 2025, 14:17Anamaria B: Your Mother Still Runs Part of Your DNA
-
Nov 2, 2025, 10:57Paul Bolaji on Launching The First Centralised Nigerian National Stroke Registry