Intellia Therapeutics Inc. (NASDAQ:NTLA) has presented the first demonstration of human alpha-1-antitrypsin protein levels in non-human primates after a single administration. The company presented the data at the 20th Alpha-1 Foundation’s Gordon L. Snider Critical Issues Workshop: The Promise of Gene-Based Intervention of Alpha-1 Antitrypsin Deficiency.

Intellia on the path to provide protein restoration therapy

When compared to the normal adeno-associated virus (AAV) gene therapy, the company’s targeted liver gene insertion tech has the potential of achieving therapeutic protein expression levels durably and stably with a single-course therapy. Intellia’s CEO John Leonard indicated that the new data reinforces the company’s promise to provide a potential cure to a range of rare diseases that require functional protein restoration in the liver with just a single-course treatment. Leonard added that they have demonstrated the Intellia platform’s translatability and modularity to several targets through gene insertion. This resulted in the production of unprecedented protein levels in non-human primates for AAT deficiency and hemophilia B.

Leonard added that besides advancing to clinical treatment for other chronic diseases, the company will continue with preclinical studies to validate its wholly-owned CRISPR-based AAT deficiency treatment approaches to achieve normal AAT protein levels. The company is advancing multiple genome editing strategies with the potential of treating liver and lung manifest6atioons of AAT deficiency occurring because of SERPINA1 gene mutations. Most importantly the normal human AAT protein levels the company attained after targeted human SERPINA1 gene insertions were stable across the 11 study weeks in the ongoing NHP study.

AAT protein levels could restore protease inhibition to protect the lungs and liver

Interestingly the human AAT protein levels observed could be therapeutically adequate in restoring protease inhibition in protecting the liver and lings from inappropriately regulated neutrophil elastase activity. These latest results build on the past results demonstrating that consecutive in vivo genome editing achieved therapeutically significant results in AATD mouse models.

The data reinforce previous data that showed the use of the same proprietary gene insertion tech for targeted Factor 9 gene insertion led to circulating human Factor 9 in hemophilia B patients.