Earlier this month, the FDA granted orphan-drug status for KB408 in AATD. The first patient in the planned Phase I trial is expected to receive treatment in Q1 2024. The Phase I trial, involving adult patients with AATD and the PI*ZZ genotype, will assess the safety, tolerability, and efficacy at three planned dose levels.
KB408 is a gene therapy consisting of a modified herpes simplex virus 1 (HSV-1) derived vector, which delivers two full-length copies of the SERPINA1 gene to enable AAT expression. The therapy is delivered via nebulisation.
Senior Vice President of Clinical Development at Krystal Biotech Hubert Chen said, “This IND acceptance represents an important milestone for us as we work to address a serious lung disease with limited treatment options and allows us to demonstrate the potential of our platform to deliver genes repeatedly to epithelial cells of the lung.”
According to GlobalData, the AATD market in the US and Germany is expected to see significant growth in the next few years, with its market size reaching $3.48bn in 2031.
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.