Acellena

Acellena Therapeutics was a biotechnology company focused on developing small molecule pharmacological chaperones for the treatment of lysosomal storage disorders and neurodegenerative diseases. The company's primary research programs targeted the glucocerebrosidase (GBA) enzyme, which is frequently mutated in patients with Gaucher disease and is a significant genetic risk factor for Parkinson's disease. By utilizing a proprietary platform to identify molecules that stabilize misfolded proteins, Acellena aimed to restore enzymatic activity and reduce the accumulation of toxic substrates. The company was founded by experts in protein folding and rare disease drug development to address significant unmet needs in genetic medicine. Its lead candidate, ACE-101, was designed as a next-generation chaperone with optimized pharmacokinetic properties for systemic and central nervous system penetration. Although the company is no longer active, its work contributed to the advancement of chaperone-based therapeutic strategies.