Isabella Presch/LinkedIn
Oct 30, 2026, 15:40
Isabella Presch: The Practicalities of Gene Therapy Adoption in Hemophilia
Isabella Presch, Senior Medical Director of Rare Diseases, Advanced Therapies and Pediatrics at Fortrea, shared a post on LinkedIn:
“Interesting talks this morning European Society of Gene and Cell Therapy ESGCT 2026 about uptake of gene therapy, hurdles and learnings in hemophilia.
Wolfgang Miesbach shared key insights, success factors and achievements from Germany:
What worked well:
- Patient organisations involved early
- Consensus guidance on centres, hub-and-spoke care and SOPs (WFH, EAHAD, ISTH, GTH)
- Independent registries
- Documentation platform: GENTS
Education and shared decision-making tools.
Key learnings:
- Communicate ranges and non-responders (not cure)
- Patient-relevant endpoints and standardised factor assays early in development
- Broader trial populations
- Early dialogue with HTA bodies
- Funding for centre infrastructure, monitoring and 15-year follow-up
- Follow-up regardless of commercial fate; early notice of market exits
Amit Nathwani described considerations involved in decision making in the UK:
- For Patients: Why change? Will it last? Monitoring burden
- For clinical teams: Eligibility assessment, Follow-up capacity, Coordinated care
- For NHS: Contracts and liability, Service readiness, Reliable supply.”
Other posts featuring Isabella Presch on Hemostatis Today.
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Oct 30, 2026, 15:12Edward C.C. Wong: Navigating the Implications of the 2026 ASH Guidelines for Blood Donation
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Oct 30, 2026, 14:53Pall T. Onundarson: Anticoagulation Guidelines Under the Influence of Pharmaceuticalisation
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Oct 30, 2026, 14:27Isabella Presch: From Innovation to Accessibility in Cell and Gene Therapy
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Oct 30, 2026, 14:00Abdul Mannan: A Negative PCR Does Not Exclude β-Thalassaemia
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Oct 30, 2026, 13:53Matteo Foschi: Can Thrombolysis Safely Treat Stroke After Cardiac Procedures?
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Oct 30, 2026, 13:42Wolfgang Miesbach: Gene Therapy-Associated Leukaemia With Clonal Persistence Over 16 Years
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Oct 30, 2026, 12:47Louise St. Germain Bannon: Turning Thrombosis Awareness into Stroke Prevention
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Oct 30, 2026, 12:36Aryabhatta Sadhu: Sickle Cell Crisis Recognising When Treatment Must Change
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Oct 30, 2026, 12:22Fabrice Cognasse: The Future of Blood Banks From Transfusion Support to Precision Medicine