Krystal Biotech Receives FDA Platform Technology Designation for HSV-1 Viral Vector Used in KB801 for Neurotrophic Keratitis
The US FDA has granted platform technology designation to Krystal Biotech's genetically modified, non-replicating herpes simplex virus type 1 (HSV-1) viral vector, which is used in KB801, a redosable eye drop gene therapy currently being evaluated for the treatment of neurotrophic keratitis (NK)25.
The platform technology designation is intended to streamline drug development and regulatory review for products using the designated technology—including efficiencies in manufacturing, earlier engagement with the FDA, and leveraging prior nonclinical safety and manufacturing data from previously approved products (such as VYJUVEK)25.
Krystal’s HSV-1 platform has previously been used in VYJUVEK®, the company’s FDA-approved gene therapy for dystrophic epidermolysis bullosa, and the new designation recognizes the reproducibility and scalability of the HSV-1 vector across different administration methods15.
KB801 is currently being evaluated in a randomized, placebo-controlled trial for neurotrophic keratitis, a rare degenerative corneal disease leading to eye injury and vision loss25.
This is only the second FDA platform technology designation awarded since the program’s inception, and comes after the FDA withdrew the previously assigned designation from Sarepta Therapeutics' adeno-associated virus vector platform due to safety concerns1.
The designation may allow Krystal Biotech to accelerate the development of its genetic medicine pipeline by applying knowledge and regulatory data from previous products using the HSV-1 vector to new drug applications23.
Sources:
1. https://www.biospace.com/fda/fdas-second-platform-designation-goes-to-krystal-after-sarepta-withdrawal
2. https://www.globenewswire.com/news-release/2025/10/14/3166102/0/en/Krystal-Biotech-Receives-FDA-Platform-Technology-Designation-for-HSV-1-Viral-Vector-Used-in-KB801-for-the-Treatment-of-Neurotrophic-Keratitis.html
3. https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-krys/krystal-biotech/news/does-fdas-platform-technology-designation-redefine-the-innov
5. https://www.pharmanow.live/latest-news/fda-grants-krystal-platform-designation