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Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications

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Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74.

Rachael A Potter¹, Ellyn L Peterson¹, Danielle Griffin¹

¹Sarepta Therapeutics, Inc., Cambridge, MA 02142, USA.

Molecular Therapy. Methods & Clinical Development

|February 8, 2024

Summary

Plasmapheresis effectively reduces anti-adeno-associated virus (AAV) antibodies in nonhuman primates, enabling gene transfer therapy. This pretreatment is safe and may allow redosing or overcoming pre-existing immunity in future human studies.

Keywords:

Duchenne muscular dystrophy adeno-associated virus antibodies delandistrogene moxeparvovec gene therapy immunosuppression plasmapheresis pre-existing antibodies redosing safety

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Area of Science:

Biomedical research
Gene therapy
Immunology

Background:

Pre-existing immunity to adeno-associated virus (AAV) prevents patients from receiving gene transfer therapies.
Developing strategies to overcome AAV immunity is crucial for expanding gene therapy accessibility.

Purpose of the Study:

To investigate immunosuppression strategies for AAV gene transfer therapy safety and efficacy.
To evaluate plasmapheresis as a method to circumvent pre-existing AAV immunity or enable redosing.

Main Methods:

Nonhuman primates received delandistrogene moxeparvovec (SRP-9001) gene therapy.
Different immunosuppression regimens (prednisone, rituximab, sirolimus) were tested.
Plasmapheresis was performed before redosing in select cohorts to assess its impact on anti-AAV antibodies.

Main Results:

Plasmapheresis successfully reduced circulating anti-AAVrh74 antibodies after 2-3 exchanges.
Redosing after plasmapheresis was well-tolerated without adverse clinical or immunological events.
A cohort redosed without plasmapheresis experienced hypersensitivity reactions, which were managed.

Conclusions:

Plasmapheresis is a safe and effective pretreatment to lower anti-AAV antibody levels in nonhuman primates.
This approach shows promise for enabling gene transfer therapy in individuals with pre-existing immunity or requiring redosing.
Findings may guide human clinical trials for AAV-based gene therapies.