Krystal Biotech said that the FDA has cleared an IND submitted by the company at the beginning of July 2022 and that it now intends to initiate a Phase 1 trial of its KB407 nebulized re-dosable gene therapy for the treatment of cystic fibrosis sometime before the end of this year. The company had previously announced a Phase 1 study of KB407 taking place in Australia. The new Phase 1 SAD study in the US is expected to enroll up to 20 adult CF patients and will evaluate 3 doses of the nebulized gene therapy.
According to Krystal, KB407 delivers two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to lung cells using a modified HSV-1 vector and is for use in CF patients no matter the type of mutation underlying their CFTR malfunction. The company’s pipeline is mostly focused on gene therapies for skin conditions, but it also has an inhaled gene therapy for the treatment of alpha-1 antitrypsin deficiency (KB408) in preclinical development.
Krystal Biotech Senior VP of Clinical Development Hubert Chen said, “We are excited to advance KB407, our investigational gene therapy for patients with CF, into the clinic. It is designed to treat the root cause of the disease regardless of an individual patient’s mutation by giving the body instructions to produce its own functional protein. In addition, this IND acceptance represents an important milestone for us as it allows us to demonstrate the power of our platform to deliver genes, beyond skin cells, to respiratory cells.”
Read the Krystal Biotech press release.
