FBX-101

FBX-101 is an experimental gene therapy delivered via adeno-associated virus rh10 vector to the GALC gene. It is developed by Forge Biologics to treat Krabbe disease.^[1]^[2]^[3]^[4]

References

^ Hordeaux, Juliette; Jeffrey, Brianne A.; Jian, Jinlong; Choudhury, Gourav R.; Michalson, Kristofer; Mitchell, Thomas W.; Buza, Elizabeth L.; Chichester, Jessica; Dyer, Cecilia; Bagel, Jessica; Vite, Charles H.; Bradbury, Allison M.; Wilson, James M. (1 May 2022). "Efficacy and Safety of a Krabbe Disease Gene Therapy". Human Gene Therapy. 33 (9–10): 499–517. doi:10.1089/hum.2021.245. PMC 9142772. PMID 35333110.
^ Bradbury, Allison M.; Bagel, Jessica H.; Nguyen, Duc; Lykken, Erik A.; Pesayco Salvador, Jill; Jiang, Xuntian; Swain, Gary P.; Assenmacher, Charles A.; Hendricks, Ian J.; Miyadera, Keiko; Hess, Rebecka S.; Ostrager, Arielle; ODonnell, Patricia; Sands, Mark S.; Ory, Daniel S.; Shelton, G. Diane; Bongarzone, Ernesto R.; Gray, Steven J.; Vite, Charles H. (10 August 2020). "Krabbe disease successfully treated via monotherapy of intrathecal gene therapy". Journal of Clinical Investigation. 130 (9): 4906–4920. doi:10.1172/JCI133953. PMC 7456224. PMID 32773406.
^ Escolar, Maria; Lugt, Mark Vander; Poe, Michele; Greco, Melissa; Werling, Keith; De Silva, Erandi; Ruiz, Juan; Szabolcs, Paul (2023). "P014: First-in-human phase 1/2 trial of intravenous FBX-101 with cord blood transplantation increases GALC, brain and motor development in infantile Krabbe". Genetics in Medicine Open. 1 (1): 100024. doi:10.1016/j.gimo.2023.100024.
^ Labant, MaryAnn (1 December 2022). "Cell and Gene Therapy Industry Feels Growing Pains: Signs of greater maturity in the industry include scalable platforms, improved plasmids and vectors, streamlined processes, and regulatory discussions". Genetic Engineering & Biotechnology News. 42 (12): 38, 40–42. doi:10.1089/gen.42.12.14. S2CID 254532949.

[1] Hordeaux, Juliette; Jeffrey, Brianne A.; Jian, Jinlong; Choudhury, Gourav R.; Michalson, Kristofer; Mitchell, Thomas W.; Buza, Elizabeth L.; Chichester, Jessica; Dyer, Cecilia; Bagel, Jessica; Vite, Charles H.; Bradbury, Allison M.; Wilson, James M. (1 May 2022). "Efficacy and Safety of a Krabbe Disease Gene Therapy". Human Gene Therapy. 33 (9–10): 499–517. doi:10.1089/hum.2021.245. PMC 9142772. PMID 35333110.

[2] Bradbury, Allison M.; Bagel, Jessica H.; Nguyen, Duc; Lykken, Erik A.; Pesayco Salvador, Jill; Jiang, Xuntian; Swain, Gary P.; Assenmacher, Charles A.; Hendricks, Ian J.; Miyadera, Keiko; Hess, Rebecka S.; Ostrager, Arielle; ODonnell, Patricia; Sands, Mark S.; Ory, Daniel S.; Shelton, G. Diane; Bongarzone, Ernesto R.; Gray, Steven J.; Vite, Charles H. (10 August 2020). "Krabbe disease successfully treated via monotherapy of intrathecal gene therapy". Journal of Clinical Investigation. 130 (9): 4906–4920. doi:10.1172/JCI133953. PMC 7456224. PMID 32773406.

[3] Escolar, Maria; Lugt, Mark Vander; Poe, Michele; Greco, Melissa; Werling, Keith; De Silva, Erandi; Ruiz, Juan; Szabolcs, Paul (2023). "P014: First-in-human phase 1/2 trial of intravenous FBX-101 with cord blood transplantation increases GALC, brain and motor development in infantile Krabbe". Genetics in Medicine Open. 1 (1): 100024. doi:10.1016/j.gimo.2023.100024.

[4] Labant, MaryAnn (1 December 2022). "Cell and Gene Therapy Industry Feels Growing Pains: Signs of greater maturity in the industry include scalable platforms, improved plasmids and vectors, streamlined processes, and regulatory discussions". Genetic Engineering & Biotechnology News. 42 (12): 38, 40–42. doi:10.1089/gen.42.12.14. S2CID 254532949.

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