As gene therapy research continues to advance, understanding who may be eligible for a particular treatment remains an important challenge for the Duchenne and Becker community. PPMD is committed to supporting research that addresses these challenges and helps move the field forward.
Today, we’re sharing an update on a research project funded through PPMD’s Gene Therapy Initiative to develop a more standardized approach to antibody screening for adeno-associated virus (AAV) gene therapy. In a collaborative agreement, PPMD and a number of gene therapy sponsors co-funded a post-doctoral fellowship to support a project conducted by Dr. Michelle Rengarajan at Massachusetts General Hospital titled ‘Development of a Rapid, Scalable, and Standardized Universal Antibody Screening Tests for Adeno-Associated Virus Gene Therapy’.
Why Does Antibody Screening for AAV Matter?
AAV is a commonly used delivery vehicle for gene therapies. However, some people have pre-existing antibodies to certain AAV serotypes, which may affect their eligibility for a particular gene therapy. After receiving an AAV gene therapy, the body can also produce high levels of antibodies, which presents challenges for potential re-dosing.
Currently, different gene therapy developers may use different antibody screening tests for AAV vectors. Because these tests are not necessarily comparable, it can be difficult for families and clinicians to understand how antibody results relate to eligibility across different AAV serotypes, such as AAV8, AAV9, and AAVrh74.
A standardized screening approach could help address this challenge by making it easier to evaluate antibody levels across multiple AAV serotypes. Over time, this work could support more consistent testing and help inform gene therapy development and regulatory review.
Supporting Progress Toward a Universal Screening Test for AAV
In August 2025, PPMD awarded funding to support a postdoctoral fellowship for Dr. Camille Wouters, who is conducting this work under the guidance of Dr. Michelle Rengarajan at Massachusetts General Hospital.The fellowship builds on a larger project led by Dr. Rengarajan and supported by the Massachusetts Life Sciences Center. The project evolved from discussions convened with PPMD’s support at the Broad Institute through our Gene Therapy Initiative. Those discussions brought together stakeholders to examine challenges related to AAV antibodies, including how pre-existing antibodies can affect treatment eligibility and how antibodies generated after treatment may limit future dosing options.
One key takeaway was the need for more consistent antibody testing across AAV types.
Progress To Date
The team has evaluated multiple antibody screening assays and developed a protocol that can reliably and sensitively measure antibody levels across different AAV types. An important part of the protocol is that it relies only on cost-effective equipment that is widely available in clinical laboratories. This is a critical consideration for translating a research approach into a tool that could be used in clinical development and potentially support regulatory needs. The team is currently partnering with a clinical lab to begin validating the test for wider, clinical use.
Developing a standardized, scalable approach will take time, and further work is needed to understand how these tests can be used in practice. However, evaluating available methods is an important step toward addressing a challenge that affects the broader gene therapy field.
Looking Ahead
PPMD is proud to support collaborative research that addresses shared challenges in Duchenne and Becker research and care. Improving our understanding of AAV antibodies and developing more consistent screening methods could help researchers and clinicians better interpret antibody results as the gene therapy landscape evolves.
We will continue to follow this project and share updates with the community as the work progresses.
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