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FDA Veteran Paz Vellanki, MD, joins Precision to become one of 3 ex-FDA oncology leaders supporting clients

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Gene Therapy Immunogenicity and IVD Regulatory Services

Unmatched expertise in NAb/TAb assays and in managing global regulatory engagements

Precision for Medicine provides a suite of services for adeno-associated virus (AAV) immunogenicity assays from preclinical development through gene therapeutic commercialization.

Whether your development path includes a companion diagnostic (CDx) or not, Precision experts—who are shaping and guiding this field—help determine the optimal development route for your assays, ensuring the most efficient and compliant path to market.

Proven expertise in AAV immunogenicity assays, from preclinical development through commercialization

Precision for Medicine has extensive experience with AAV immunogenicity assays for rare diseases like hemophilia, Fabry, Gaucher, muscular dystrophies, and non-rare diseases like age-related macular degeneration (AMD) and heart failure.

The assays employed encompass anti-capsid NAb and TAb, anti-transgene anti-drug antibody (ADA), and ELISpot assays targeting both anti-capsid and anti-transgene responses.

Notably, Precision for Medicine developed and serves as the lab for the single-site laboratory-developed test (LDT) anti-AAV5 NAb assay for HEMGENIX®.

25
AAV gene therapy programs supported
3
FDA-approved IDEs (+4 in progress)
40
AAV assay validations (GxP & CLIA)
175
patients tested using Precision developed commercial NAb assay for HEMGENIX®
PFM-Extensive Experience with AAV Immunogenicity Assays

Delivering NAb, TAb, and ELISpot assays for diverse capsid types at every stage

Precision for Medicine's scientific team has developed and validated NAb, TAb and ELISpot assays in both humans and non-human primates (NHPs) for several capsid types across multiple stages:

  • Preclinical/GLP
  • Clinical trial GxP
  • Clinical Trial CLIA
  • CDx
Risk assessment and regulatory requirements for CTAs

Our experienced regulatory consulting group supports AAV gene therapy assays from LDTs to IVDs and more

Precision's regulatory team provides comprehensive support at each critical step of the development timeline, from advising on requirements and design through analytical and clinical protocol development and execution. In parallel, Precision for Medicine will draft the required regulatory documentation (SRD, IDE, Annex XIV, ITA) to support clinical trials and, if needed, final commercialization (PMA, CE Mark, UKCA Mark) while communicating with the regulatory bodies to ensure compliance and streamlined review of submissions.

Purposefully integrated to support all aspects of gene therapy approvals

In addition to immunogenicity assay development and integrated regulatory consulting, Precision supports gene therapy development with a broad set of services to streamline development and avoid delays to market.

Explore our supporting gene therapy services:

Leading the conversation: Precision’s contributions to American Association of Pharmaceutical Scientists (AAPS) Guidelines on AAV NAb, TAb, and ELISpot Assays

Experts from Precision for Medicine guide and shape the gene therapy field, actively contributing to the AAPS recommendations for the development of gene therapy NAb, TAb, and ELISpot assays. Explore our peer-reviewed articles that outline best practices in this area.
  • Research Article

    AAPS Assessment of Immune Responses Against AAV Encoded Transgene Products (2025)

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    AAPS Assessment of Immune Responses Against AAV Encoded Transgene Products (2025)
  • Research Article

    AAPS Evaluation of Cellular Immune Response to Adeno-Associated Virus Based Gene Therapy (2023)

    Download Article
    AAPS Evaluation of Cellular Immune Response to Adeno-Associated Virus Based Gene Therapy (2023)
  • Research Article

    AAPS Evaluation of the Humoral Response to Adeno-Associated Virus Based Gene Therapy Modalities Using Total Antibody Assays (2021)

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    AAPS Evaluation of the Humoral Response to Adeno-Associated Virus Based Gene Therapy Modalities Using Total Antibody Assays (2021)

Precision has years of recognized thought leadership in gene therapy

The expert teams at Precision continue to be at the forefront of current thinking and best practices across gene therapy assays and global regulatory frameworks

Future-proofing gene therapy assays Validation strategies for long-term success

Webinar

Future-proofing Gene therapy Assays: Validation Strategies for Long-term Success

PFM - Gene therapy assays_ Achieving regulatory compliance with a unified immunogenicity strategy- Webinar

Webinar

Gene Therapy Assays: Achieving Regulatory Compliance with a Unified Immunogenicity Strategy

PFM - Strategies for Immunogenicity Assays in AAV Gene Therapy_ Development to CDx Approval - Webinar_

Webinar

Strategies for Immunogenicity Assays in AAV Gene Therapy: Development to CDx Approval

PFM - Companion Diagnostics_ Strategies for Effective Co-Development with Therapeutics - Webinar

Webinar

Companion Diagnostics: Strategies for Effective Co-development with Therapeutics

Optimizing GTx and CDx development in the US_Thumbnail

White paper

Optimizing Successful Development of Viral Vector Gene Therapies, Trials, and CDx

PFM-The Regulation of Companion Diagnostics-Scientific Article 2013

Research article

The Regulation of Companion Diagnostics: A Global Perspective. (2013)

Additional insights on gene therapy

Explore more insights
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Beyond AAV immunogenicity assays, Precision is a full-service CRO with expertise in gene, cell, and novel therapies

Precision’s transformational approach to biomarker and assay development, and clinical trials, ensures comprehensive services and expertise for every gene therapy development challenge, from IND-enabling assays to commercialization.

50 rare disease clinical trials and trials for 100+ orphan projects
12 sample processing locations worldwide
350 IVD and CDx global regulatory submissions and registrations
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