Codexis uses its proprietary CodeEvolver® discovery platform to uniquely tailor next-generation biotherapeutics with transformative properties. We combine bioinformatics from thousands of protein variants with directed evolution to create intentionally designed proteins, specifically optimized for their disease indications.
We focus on developing life-changing biologic and gene therapy candidates that address the critical unmet needs of patients. Our current priorities are inborn errors of metabolism, gastrointestinal disorders, lysosomal storage diseases, and other debilitating genetic disorders with pivotal patient need.
Our pipeline is committed to the continuous advancement of therapeutic candidates with best-in-class efficacy through self-funded initiatives or in close collaboration with our valued partners.
The unmet need:
Phenylketonuria, is a rare inherited genetic disorder that affects the metabolism of the amino acid phenylalanine, causing it to reach toxic levels in the body. Although doctors have been able to test for PKU in newborns for more than 50 years, treatment options primarily remain limited to dietary restrictions, which leads to many untreated PKU patients experiencing intellectual disability, seizures, and mental disorders.
Using our proprietary CodeEvolver®platform, we engineered CDX-6114, an administered, GI-stable therapeutic enzyme to potentially treat PKU by actively compensating for the absence of the patient’s phenylalanine-metabolizing enzymes. CDX-6114’s stability in the harsh GI environment allows it to potentially remove phenylalanine from the body before it reaches toxic levels, solving a problem that has vexed scientists for decades. The program was licensed to Nestlé Health Science after meeting all of its Phase 1a study endpoints.
The unmet need:
Fabry disease is a rare, lysosomal storage disorder that affects the metabolism of a type of fat called globotriaosylceramide (Gb3 or GL-3), causing it to build up inside cells and damage them. While enzyme replacement therapies with alpha-galactosidase may reduce patient symptoms, patients may still experience multiple symptoms, necessitating more specifically tailored, long-term treatments
Using our proprietary CodeEvolver® platform, we engineered CDX-6311, a unique protein variant specifically tailored with an improved stability, activity, and immunogenicity profile to potentially treat Fabry disease. In collaboration with Takeda, Codexis continues to generate novel gene sequences encoding protein variants with enhanced efficacy to be used as improved transgenes in a variety of gene therapies, pioneering next-generation, long-term treatments for rare genetic disorders.
The November issue of Nature’s Biopharma Dealmakers includes an article that highlights how our CodeEvolver® technology platform can overcome the suboptimal properties and limitations of natural proteins by generating novel biological variants with safety and efficacy profiles specifically tailored to target diseases.