A local biotech company's drug candidate that is looking to treat a rare lung and liver genetic disorder has received approval from the U.S. Food and Drug Administration to begin Phase 1 clinical trials.
Krystal Biotech Inc., a gene therapy-focused firm based in the South Side, announced that its KB408 product had its Investigative New Drug (IND) application approved by the FDA for the treatment of alpha-1 antitrypsin deficiency (AATD), which lacks a cure.
To be inhaled via nebulization, Krystal said KB408 is designed to help those who have a certain gene mutation that causes the body to make misfolded proteins that can become trapped in the liver, which can then lead to lower levels of this protein in the blood.
Left untreated, Krystal said this can lead to inflammation and damage to the lungs that could result in life-threatening pulmonary impairments.
"We are excited to advance KB408, our investigational gene therapy for patients with alpha-1 antitrypsin deficiency, into the clinic in our Serpentine-1 study," Dr. Hubert Chen, senior vice president of clinical development at Krystal Biotech, said in a prepared statement. "This IND acceptance represents an important milestone for us as we work to address a serious lung disease with limited treatment options, and also allows us to demonstrate the potential of our platform to deliver genes repeatedly to epithelial cells of the lung.”
Current treatment practices for severe cases of AATD require weekly infusions, the biotech company said.
As part of the clinical trials, Krystal will see its KB408 candidate used as part of a single-dose escalation study to evaluate the drug's safety, tolerability and efficacy. Phase 1 trials are expected to begin by Q1 2024.
