A phase 1/2 safety and efficacy study of TAK‐754 gene therapy: The challenge of achieving durable factor VIII expression in haemophilia A clinical trials
Sustained FVIII expression remains a challenge in haemophilia A AAV gene therapy trials. Mechanisms of transgene expression loss require further study as clinical studies enter long term follow-up periods.
Authors
- Mila Ayash‐Rashkovsky
- Marcin von Grotthuss (ORCID: https://orcid.org/0000-0002-2850-1152)
- Francisco José López‐Jaime (ORCID: https://orcid.org/0000-0003-2407-3901)
- Matt Wagoner
- Kavitha Rajavel
- María Teresa Álvarez Román (ORCID: https://orcid.org/0000-0003-3296-4288)
- Jon Kenniston
- María Eva Mingot‐Castellano (ORCID: https://orcid.org/0000-0001-9083-855X)
- Dorothée Diogo (ORCID: https://orcid.org/0000-0003-2913-5913)
- Antoine Rauch (ORCID: https://orcid.org/0000-0002-1182-4131)
- John Chapin (ORCID: https://orcid.org/0000-0003-3365-7757)
- Sophie Susen (ORCID: https://orcid.org/0000-0001-5953-163X)
- Caterina Maggiore
- Qin Wang (ORCID: https://orcid.org/0000-0002-8384-7703)
Institutions
- Hospital Universitario La Paz (ES)
- Hospital Regional Universitario de Málaga (ES)
- IQVIA (United States) (US)
- Instituto de Biomedicina de Sevilla (ES)
- Takeda (United States) (US)
- AstraZeneca (United States) (US)
- Hospital Universitario Virgen del Rocío (ES)
- Universidad Autónoma de Madrid (ES)
Publication Details
- Journal
- Haemophilia
- Published
- 2024-12-23
- DOI
- https://doi.org/10.1111/hae.15121
- Citations
- 8
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 3.64
Funders
- Takeda Pharmaceuticals International