FDA Platform Technology Designation: Krystal Biotech Leads the Way After Sarepta Setback

In a significant development for the pharmaceutical industry, Krystal Biotech has become the second company—and currently the only active recipient—to receive the FDA's Platform Technology Designation. This designation, aimed at accelerating approvals for therapeutics using similar technologies, was awarded to Krystal's modified herpes simplex virus (HSV) vector used in gene therapies.

Krystal's HSV Vector Platform: A New Frontier in Gene Therapy

Krystal Biotech's platform, which utilizes a non-replicating HSV-1 viral vector, has garnered attention for its potential to revolutionize gene therapy delivery. The company's approved topical therapy, Vyjuvek, indicated for the skin condition dystrophic epidermolysis bullosa, serves as the foundation for this designation.

The HSV-1 vector technology takes advantage of the virus's natural characteristics, including high payload capacity and ability to evade the immune system, while aiming to minimize toxicities. This approach has positioned Krystal at the forefront of gene therapy innovation, with implications reaching beyond dermatological applications.

Implications for Future Drug Development

The Platform Technology Designation is expected to streamline the development and regulatory review processes for Krystal's pipeline. Of particular interest is KB801, the company's investigational eye drop formulation currently in Phase I/II trials for neurotropic keratitis, a rare corneal disease.

William Blair analyst Sami Corwin noted the unique aspect of Krystal's designation, stating, "Krystal is interesting because [KB801] uses the same HSV [vector] but the route of administration is totally different." This highlights the potential for the platform to be applied across various administration methods, potentially accelerating the development of diverse therapeutic modalities.

Industry Landscape and Regulatory Considerations

The pharmaceutical sector is closely watching the implications of this designation, especially in light of recent events. Sarepta Therapeutics, the first recipient of the Platform Technology Designation for its AAV vector platform, had its designation revoked in July 2025 following patient deaths associated with the technology.

Sarfaraz Niazi, an adjunct professor at the University of Illinois' College of Pharmacy, emphasized the FDA's focus on safety: "The PTD designation implies that the FDA has found the HSV-1 platform sufficiently well-characterized to warrant cross-application review efficiencies, but it does not pre-clear ocular-specific risks."

As the industry adapts to this new regulatory tool, questions remain about its application across different administration routes and therapeutic areas. The coming months will likely provide clarity on how the FDA intends to leverage this designation to accelerate drug development while maintaining rigorous safety standards.