What question did this study set out to answer?

The aim is to explore strategies to modulate neutralizing antibody responses against therapeutic transgenes in hemophilia B gene therapy.

May 8, 2012Molecular TherapyOpen Access

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

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Authors

FMFederico MingozziInserm YCYifeng ChenUniversity of Mons SMSamuel L. MurphyIntegrated BioTherapeutics (United States)

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Implication

Randomized trial demonstrates eradication of neutralizing antibodies in a nonhuman primate model, suggesting B cell-targeting therapies may enhance gene transfer safety.

Key Points

The aim is to explore strategies to modulate neutralizing antibody responses against therapeutic transgenes in hemophilia B gene therapy.
Used anti-B cell monoclonal antibody rituximab with cyclosporine A in rhesus macaques
Assessment after liver gene transfer with AAV8 vectors expressing human F.IX
Evaluated antibody responses and F.IX transgene product levels post-immunosuppression.
Anti-F.IX neutralizing antibodies eradicated in treated macaques with restored plasma F.IX levels
In one macaque, anti-AAV6 antibodies dropped below detection, facilitating vector readministration
Achieved high levels of F.IX transgene product in plasma (60% of normal).

Cite This Study

Mingozzi et al. (2012) studied this question.

synapsesocial.com/papers/6a6273aec72d59d08cf56269 https://doi.org/10.1038/mt.2012.84

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Implication

Randomized trial demonstrates eradication of neutralizing antibodies in a nonhuman primate model, suggesting B cell-targeting therapies may enhance gene transfer safety.

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

Discussion

Member takes

Implication

Key Points

Cite This Study

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

Discussion

Member takes

Implication

Key Points

Cite This Study

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