#USA #Barcelona #Congruence Therapeutics #CGX-195 #AAT Deficiency

Congruence Therapeutics Reveals Key Findings on CGX-195

Congruence Therapeutics, known for its innovative approach in the biotech industry, is poised to share significant advancements concerning CGX-195 at the prestigious European Respiratory Society (ERS) Alpha-1 Antitrypsin Conference 2026, taking place on October 23rd and 24th in Barcelona, Spain. This event, in partnership with the European Alpha-1 Research Collaboration (EARCO), gathers elite researchers from around the globe to discuss the latest breakthroughs in alpha-1 antitrypsin deficiency (AATD).

The presentation will showcase compelling preclinical data demonstrating CGX-195's efficacy in enhancing levels of functional Z-A1AT while simultaneously decreasing polymerized forms of the protein. These findings are based on cellular assays and a PiZ transgenic mouse model, suggesting that CGX-195 effectively tackles the fundamental issues associated with AATD.

Significance of AAT Deficiency

Alpha-1 antitrypsin deficiency is a hereditary condition resulting from mutations in the SERPINA1 gene. This gene is responsible for producing A1AT, a vital protease inhibitor primarily synthesized in the liver, which safeguards the lungs from damage caused by neutrophil elastase. The most common mutation, known as the Z variant, leads to the misfolding and subsequent polymerization of A1AT within liver cells. This misfolding gives rise to a two-pronged health threat: insufficient levels of functional A1AT expose the lungs to protease damage, resulting in early-onset emphysema and chronic obstructive pulmonary disease (COPD); meanwhile, the buildup of polymerized proteins in the liver can precipitate liver fibrosis or even hepatocellular carcinoma.

CGX-195's Mechanism of Action

The innovation behind CGX-195 lies in its capacity as an orally administered small molecule corrector targeting the misfolded Z variant of A1AT. Developed utilizing the proprietary Revenir™ computational platform of Congruence Therapeutics, CGX-195 is engineered to bind to and stabilize this Z variant, thereby promoting proper folding and enhancing the secretion of functional A1AT. In preclinical models, CGX-195 has demonstrated a significant reduction in the formation of toxic polymers, addressing both lung and liver manifestations of AATD.

Sharath Hegde, Ph.D., Chief Scientific Officer at Congruence, expressed enthusiasm regarding the upcoming presentation, stating, “We are honored that our preclinical results with CGX-195 were selected for a podium presentation at the field's leading scientific meeting on alpha-1 antitrypsin deficiency... By correcting the misfolding of the Z variant at its source, CGX-195 has the potential to provide a comprehensive treatment for AATD.”

Presentation Details

• - Title: CGX-195, a novel orally active small molecule corrector for Alpha-1 Antitrypsin Deficiency (AATD), increases functional secreted Z-A1AT and reduces polymerized Z-A1AT in cellular assays and in the PiZ mouse model.
• - Abstract Number: OP01
• - Session: Oral Presentations (Chairs: Didier Cataldo and Jeanine D'Armiento)
• - Presenter: Anzhelika Kabro, Senior Director of Chemistry at Congruence Therapeutics
• - Date and Time: October 23, 2026, from 4:50 PM to 5:00 PM CEST (10:50 AM to 11:00 AM ET)
• - Location: ERS Alpha-1 Antitrypsin Conference 2026, Barcelona, Spain

Future Prospects

With ongoing IND-enabling activities, Congruence Therapeutics is diligently preparing to advance CGX-195 toward clinical development in 2027. This ambitious goal reflects the organization's commitment to not only addressing AATD but also developing therapies for other genetically validated diseases — including GBA-driven Parkinson’s disease and genetic obesity. As the company continues to navigate the complex biotech landscape, the upcoming presentation at the ERS conference marks another pivotal step in their journey to provide novel treatments for patients in need.

For more details about Congruence Therapeutics, visit their official website at www.congruencetx.com.