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Peer-reviewedCC BY 4.0International Journal of Molecular Sciences

Fidanacogene Elaparvovec Gene Therapy: Budesonide Treatment of a Late Decline in Factor IX Expression in a Patient with Hemophilia B

  • Kerstin Herbst1
  • Behnaz Pezeshkpoor1
  • Claudia Klein1,2
  • Thilo Albert1
  • Philipp Lutz3
  • Christian Strassburg3
  • Ulrich Spengler3
  • Georg Goldmann1,2
  • Johannes Oldenburg1,2
  1. 1Institute of Experimental Hematology and Transfusion Medicine, University of Bonn, 53127 Bonn, Germany
  2. 2Center for Rare Diseases Bonn (ZSEB), University Clinic Bonn, 53127 Bonn, Germany
  3. 3Department of General Internal Medicine I, University of Bonn, 53127 Bonn, Germany
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Abstract

Fidanacogene elaparvovec is an adeno-associated virus (AAV)-based gene therapy that enables endogenous factor IX (FIX) expression in hemophilia B, potentially eliminating the need for prophylactic FIX replacement. Post-treatment monitoring focuses on FIX durability, hepatic safety, and immune-mediated loss of expression. A 40-year-old man with severe hemophilia B received a single intravenous dose of fidanacogene elaparvovec in the phase 3 BENEGENE-2 trial (NCT03861273). FIX activity was assessed by one-stage and chromogenic assays. Liver enzymes, clinical parameters, and adverse events were monitored. Prednisolone was initiated for liver enzyme elevation and later switched to budesonide in response to declining FIX levels. Follow-up continued for 4 years. FIX activity peaked at week 6 (~30 IU/dL) and remained stable until week 16. Moderate ALT elevation (peak ~70 U/L) responded to prednisolone. Between weeks 22–28, FIX levels declined without ALT elevation. Budesonide (9 mg/day) was started at week 25 for 3 months and gradually tapered, stabilizing FIX activity at 10-20 IU/dL. This level has been maintained for 4 years without bleeding episodes or FIX replacement. Fidanacogene elaparvovec provided sustained, clinically meaningful FIX expression. Budesonide may offer an effective hepatocyte-targeted alternative to systemic corticosteroids while supporting long-term transgene expression.

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Cite

BibTeXRIS
BibTeX
@article{herbst2026fidanacogene,
  title = {{Fidanacogene Elaparvovec Gene Therapy: Budesonide Treatment of a Late Decline in Factor IX Expression in a Patient with Hemophilia B}},
  author = {Kerstin Herbst and Behnaz Pezeshkpoor and Claudia Klein and Thilo Albert and Philipp Lutz and Christian Strassburg and Ulrich Spengler and Georg Goldmann and Johannes Oldenburg},
  journal = {International Journal of Molecular Sciences},
  year = {2026},
  month = oct,
  doi = {10.3390/ijms27209049},
  url = {https://www.mdpi.com/1422-0067/27/20/9049},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Fidanacogene Elaparvovec Gene Therapy: Budesonide Treatment of a Late Decline in Factor IX Expression in a Patient with Hemophilia B
AU  - Kerstin Herbst
AU  - Behnaz Pezeshkpoor
AU  - Claudia Klein
AU  - Thilo Albert
AU  - Philipp Lutz
AU  - Christian Strassburg
AU  - Ulrich Spengler
AU  - Georg Goldmann
AU  - Johannes Oldenburg
PY  - 2026
DA  - 2026/10/12
JO  - International Journal of Molecular Sciences
DO  - 10.3390/ijms27209049
UR  - https://www.mdpi.com/1422-0067/27/20/9049
AB  - Fidanacogene elaparvovec is an adeno-associated virus (AAV)-based gene therapy that enables endogenous factor IX (FIX) expression in hemophilia B, potentially eliminating the need for prophylactic FIX replacement. Post-treatment monitoring focuses on FIX durability, hepatic safety, and immune-mediated loss of expression. A 40-year-old man with severe hemophilia B received a single intravenous dose of fidanacogene elaparvovec in the phase 3 BENEGENE-2 trial (NCT03861273). FIX activity was assessed by one-stage and chromogenic assays. Liver enzymes, clinical parameters, and adverse events were monitored. Prednisolone was initiated for liver enzyme elevation and later switched to budesonide in response to declining FIX levels. Follow-up continued for 4 years. FIX activity peaked at week 6 (~30 IU/dL) and remained stable until week 16. Moderate ALT elevation (peak ~70 U/L) responded to prednisolone. Between weeks 22–28, FIX levels declined without ALT elevation. Budesonide (9 mg/day) was started at week 25 for 3 months and gradually tapered, stabilizing FIX activity at 10-20 IU/dL. This level has been maintained for 4 years without bleeding episodes or FIX replacement. Fidanacogene elaparvovec provided sustained, clinically meaningful FIX expression. Budesonide may offer an effective hepatocyte-targeted alternative to systemic corticosteroids while supporting long-term transgene expression.
SN  - 1422-0067
ER  -