Krystal Biotech announced that the U.S. FDA has granted platform technology designation to the genetically modified, non-replicating herpes simplex virus type 1 (HSV-1) viral vector used in the company’s redosable eye drop gene therapy, KB801, currently under evaluation for the treatment of neurotrophic keratitis.
The newly-rolled out FDA program is designed to help support rare disease therapies by streamlining drug development, manufacturing, and review processes for drug product applications by incorporating data from designated technologies. To be eligible for the designation, the technology must be a well-understood and reproducible technology — such as a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of any such technologies — and can support the development of multiple drugs or biologics.
Once granted, the designation enables sponsors to leverage prior data from the platform to support INDs, NDAs or BLAs.
“We are excited to work with the FDA under this program to identify potential efficiencies, including opportunities to leverage our prior experience with FDA-approved Vyjuvek to accelerate the development of our genetic medicines pipeline, starting with KB801 for the treatment of NK,” said Suma Krishnan, president of R&D at Krystal Biotech.
KB801 is a redosable eye drop gene therapy, currently in phase 1/2 trials, designed to enable sustained, localized expression and secretion of nerve growth factor (NGF) by epithelial cells in the front of the eye for the treatment of NK, a rare, degenerative corneal disease that leads to corneal epithelial defects, ulcers, and perforation. By enabling the cells of the front of the eye to produce NGF locally, KB801 has the potential to significantly reduce the treatment burden for patients while also maintaining more consistent NGF levels in the front of the eye.
Krystal is the second company to be awarded the platform designation. Back in June, Sarepta Therapeutics was granted the designation for the rAAVrh74 viral vector used in its investigational gene therapy, SRP-9003, for the treatment of limb-girdle muscular dystrophy type 2E/R4. A month later, amid a standoff over Elevidys — which uses the same viral vector — the FDA revoked the designation
Subscribe to our e-Newsletters
Stay up to date with news, articles and insights relevant to cell and gene therapy development and manufacturing. Plus, get special offers from Cell & Gene Therapy Review delivered right to your inbox!
Sign up now!