To be eligible for platform technology designation, the technology must be incorporated in or used by an FDA-approved drug or biologic, and preliminary evidence must demonstrate its potential for use across multiple products without compromising quality, manufacturing, or safety. Sponsors granted designation may leverage existing data from the platform in subsequent applications, potentially reducing the need for redundant studies.

 

In June 2025, the FDA granted Sarepta Therapeutics’ recombinant adeno-associated virus serotype rh74 (rAAVrh74) vector the first-ever Platform Technology Designation for a viral delivery system. The FDA’s Platform Technology Program recognizes well-characterized, reproducible technologies that can be used across multiple products to streamline drug development.  By enabling the reuse of clinical and manufacturing data for gene therapies that utilize rAAVrh74, the program can significantly reduce redundant preclinical testing and speed up regulatory filings for subsequent therapies. Standardized production processes and shared safety profiles also help lower research and development costs.

What is a Designated Platform Technology

A Designated Platform Technology is a platform officially recognized under section 506K of the Federal Food, Drug, and Cosmetic Act (FD&C Act) and section 351 of the Public Health Service (PHS) Act. To qualify, a platform must be part of at least one FDA-approved drug or licensed biological product. It must also show preliminary evidence that it can be used across multiple drugs without negatively impacting product quality, manufacturing, or safety. Additionally, data must indicate that the platform is likely to bring significant efficiencies to drug development, manufacturing, and regulatory review.

Sponsors Using Existing FDA-Approved Platform Technologies Can Request Designation

Sponsors developing drugs that incorporate or use an existing platform technology, previously included in an FDA, approved NDA, BLA, or ANDA, can request designation of that technology under section 506K of the FD&C Act. This request, ideally submitted during the IND phase, enables the sponsor to leverage the platform in future applications. To qualify, the sponsor must demonstrate how the technology meets the eligibility criteria outlined in the law, including its potential to bring efficiencies to development and regulatory review. The FDA will review designation requests within 90 days and issue a written determination. Importantly, designation does not confer rights to third-party data unless the requester holds full rights of reference. Once designated, sponsors can reuse supporting data across their own NDAs, BLAs, or EUAs. Use of previously submitted platform data in subsequent applications by other sponsors is only allowed if a full right of reference is granted.

Differences Between a Designated Platform Technology and a Platform Technology

A Platform Technology, as defined in section 506K(h)(1) of the FD&C Act, is a well-understood and reproducible technology, such as a nucleic acid sequence, delivery method, vector, or mechanism of action, essential to a drug’s structure or function. It must be adaptable for use across multiple drugs with shared structural elements and facilitate standardized processes in manufacturing or development. However, not all platform technologies qualify as designated platform technologies. Only those that meet specific statutory criteria and receive FDA designation gain the formal benefits of the program, including regulatory efficiencies and the ability to leverage prior data in future submissions1.

Examples of Platform Technologies Eligible for Designation

Technologies suitable for designation include:

  • Lipid nanoparticle (LNP) platforms used in mRNA vaccines or gene therapies, characterized by consistent composition and robust manufacturing processes.
  • Monoclonal antibody platforms, which rely on standardized cell substrates and purification processes applicable across multiple products.
  • siRNA platforms using chemically defined targeting moieties, especially when sequence modifications do not impact product safety or quality.
  • LNP platforms encapsulating oligonucleotides, provided sequence differences do not affect product quality or manufacturing consistency.

These examples show how standardized, reproducible technologies that maintain quality across products can qualify for designation.

Eligibility for Designated Platform Technology Requires Meeting the Platform Technology Definition

To be eligible, a technology must first meet the definition of a Platform Technology: it must be well-understood, reproducible, essential to a drug’s structure or function, usable across multiple drugs with common features, and facilitate standardized manufacturing or development. For formal designation, the technology must be incorporated in or used by an FDA-approved drug, have preliminary evidence of safe and effective cross-product use, and demonstrate potential for significant efficiencies in development, manufacturing, and regulatory review.

Sponsors must provide evidence that the technology can be used in new drugs without compromising quality, safety, or manufacturing. This includes demonstrating minimal differences between the approved and investigational drugs in structure, mechanism, biological effects, and manufacturing. Supporting evidence may include data on structural similarities, comparable formulations, and shared manufacturing methods. Sponsors must also submit summary data from all products using the platform and justify why these data are adequate for leveraging prior studie

Benefits of Platform Technology Designation for Sponsors and Subsequent Applications

A platform technology designation offers several potential benefits to sponsors, provided the subsequent application is either from the original sponsor or one with full rights of reference. These benefits may include early and enhanced interactions with the FDA to discuss the platform’s application, particularly regarding safety, purity, potency, or quality. Sponsors may receive timely regulatory advice and prioritized engagement, especially for products with significant public health impact. The designation also allows leveraging of prior data from related products, such as batch and stability data, nonclinical safety data, or inspectional findings, which can reduce the need for duplicative testing. However, the designation does not automatically grant priority review or expedited approval; eligibility for those pathways is determined separately.

Considerations and Limitations

To be eligible for platform technology designation, the technology must be incorporated in or used by an FDA-approved drug or biologic, and preliminary evidence must demonstrate its potential for use across multiple products without compromising quality, manufacturing, or safety. Sponsors granted designation may leverage existing data from the platform in subsequent applications, potentially reducing the need for redundant studies. However, this benefit does not extend to expedited programs such as Fast Track or Breakthrough Therapy Designation, which require separate applications. Additionally, only the original sponsor or a party with full rights of reference can request a designation or leverage it in future submissions; third parties without such rights are not permitted to do so.

Reference

FDA- Platform Technology Designation Program for Drug Development

 

About Marin Biologic Laboratories

Our Recent Publication/Meeting Presentation on Gene Therapy

1. Development of VNX-101, an Adeno-Associated Virus with Less Immunogenicity and Efficient Long-Term Expression of a CD19 T-Cell Engager. Molecular Therapy Methods & Clinical Development, published online July 24, 2025.

2. Development of a Pharmacokinetic (PK) Mouse Serum GLP ELISA for an Anti–CD19–AntiCD3 Diabody
bioRxiv 2025.03.19.644217; doi: https://doi.org/10.1101/2025.03.19.644217.

3. Cell-Based Potency Assay for Anti-CD3-Anti-CD19 Diabody. bioRxiv 2025.04.15.648836v1 https://www.biorxiv.org/content/10.1101/2025.04.15.648836v1.

4.  American Society of Hematology (ASH) Annual Meeting 2024.
Abstract link: Using Gene Therapy to Solve Challenges with CAR-T Cell Immunotherapy: Lead Selection and Preclinical Development of an Adeno-Associated Virus with Reduced Immunogenicity  Exhibiting Efficient and Long-Term Expression of an Anti-CD19 T-Cell Engager.

Comprehensive Assay Solutions for In Vitro and Cell Based Potency Assays and Pharmacokinetics Studies- Our Expertise

With 30 years of expertise in cell culture, cell-based assays, and preclinical/clinical PK/PD analysis, we specialize in offering assay services essential for a wide variety of therapeutic drug development programs, preclinical studies, IND/BLA applications, and commercialization. Our comprehensive services include both preclinical non-GLP and GLP assays, as well as non-GMP and GMP assays, providing critical support throughout the entire development pipeline.

Watch the following video and explore our latest presentation on the development and validation of potency and pharmacokinetic (PK) assays for AAV vectors, highlighting innovative methodologies and industry-leading expertise.

 

 

Download the full presentation: Development of Custom Cell Based and In vitro Potency and Pharmacokinetics (PK) Assays for AAV vectors- Marin biologic Laboratories

 

Development of Cell-Based Potency Assays: Case Studies and Blogs from Marin Biologic Laboratories (MarinBio)

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