Our approach to efficacious biotherapeutics

The ability of our technology to improve multiple protein properties in parallel led us to discover potential enzyme and gene therapy candidates optimized for specific therapeutic indications. Bringing together semi-rational library designs with application-focused high throughput screens allows us to generate large data sets, that with the aid of our bioinformatics software, yield therapeutic candidates that address needs in safety, efficacy, and manufacturability.

Biotherapeutic focus

Our focus is on discovering safe and efficacious enzymes that are orally administered for function in the GI or that are administered as the transgene of a gene therapy for treatment of Inborn Errors of Metabolism (IEMs) and other metabolic diseases. Two of our therapeutic enzyme candidates are in Phase 1 clinical trials – CDX-6114 for potential treatment of phenylketonuria, and CDX-7108 for exocrine pancreatic insufficiency. CDX-6512 is currently in IND-enabling studies and has been granted orphan drug designation by the FDA.

Biotherapeutics pipeline

We are committed to the advancement of therapeutic candidates with best-in-class efficacy through self-funded initiatives and in close collaboration with our valued partners.

Category Program Discovery Pre-Clinical Research IND-Enabling Phase 1 Partner
Oral Enzyme Therapies CDX-6114 (Phenylketonuria)
Discovery Phase complete
Pre-Clinical Research Phase complete
IND-Enabling Phase complete
Phase 1 Phase in progress
1
CDX-7108 (Exocrine Pancreatic Insufficiency)
Discovery Phase complete
Pre-Clinical Research Phase complete
IND-Enabling Phase complete
Phase 1 Phase in progress
2
CDX-6512 (Homocystinuria)
Discovery Phase complete
Pre-Clinical Research Phase complete
IND-Enabling Phase in progress
Phase 1 Phase not started
Wholly Owned
CDX-6210 (Maple Syrup Urine Disease)
Discovery Phase complete
Pre-Clinical Research Phase in progress
IND-Enabling Phase not started
Phase 1 Phase not started
Wholly Owned
Gluten Management
Discovery Phase in progress
Pre-Clinical Research Phase not started
IND-Enabling Phase not started
Phase 1 Phase not started
Wholly Owned
Undisclosed GI Program
Discovery Phase complete
Pre-Clinical Research Phase in progress
IND-Enabling Phase not started
Phase 1 Phase not started
2
Undisclosed GI Program
Discovery Phase in progress
Pre-Clinical Research Phase not started
IND-Enabling Phase not started
Phase 1 Phase not started
2
Gene Therapies Fabry Disease Transgene (CDX-6316)
Discovery Phase complete
Pre-Clinical Research Phase in progress
IND-Enabling Phase not started
Phase 1 Phase not started
3
Pompe Disease Transgene (CDX-6417)
Discovery Phase complete
Pre-Clinical Research Phase in progress
IND-Enabling Phase not started
Phase 1 Phase not started
3
Blood Factor Disorder Transgene
Discovery Phase complete
Pre-Clinical Research Phase in progress
IND-Enabling Phase not started
Phase 1 Phase not started
3
Lysosomal Storage Disorder Transgene
Discovery Phase in progress
Pre-Clinical Research Phase not started
IND-Enabling Phase not started
Phase 1 Phase not started
3
Rare Disease Gene Therapy
Discovery Phase in progress
Pre-Clinical Research Phase not started
IND-Enabling Phase not started
Phase 1 Phase not started
Wholly Owned
Gene Therapy Technology
Discovery Phase in progress
Pre-Clinical Research Phase not started
IND-Enabling Phase not started
Phase 1 Phase not started
Wholly Owned

1 Nestlé Health Science licensed CDX-6114 for world-wide development ; 2 Co-development by Nestlé Health Science and Codexis ; 3 World-wide rights licensed to Takeda

The unmet need

Homocystinuriais a rare inherited genetic disorder that affects the metabolism of the amino acid methionine, causing its metabolite homocysteine to reach toxic levels in the body. Although doctors have been able to test for HCU in newborns for more than 50 years, treatment options remain limited to dietary restrictions, which leads to many untreated HCU patients experiencing intellectual disability, seizures, and mental disorders.

Our approach

Using our proprietary CodeEvolver® technology platform, we engineered CDX-6512, an orally administered, GI-stable therapeutic enzyme to potentially treat HCU by compensating for the absence of the patient’s methionine-metabolizing enzymes. In animal models, CDX-6512’s stability in the harsh GI environment allows it to remove methionine and homocysteine from the body before they reach toxic levels, solving a problem that has vexed scientists for decades. This program is now in IND-enabling studies.

News release

The unmet need

Fabry disease is a lysosomal storage disorder that affects the metabolism of a type of fatty molecule, globotriaosylceramide, causing it to build up inside cells and damage them. While enzyme replacement therapies with recombinant human alpha-galactosidase are effective, patients may continue to experience symptoms, especially if anti-drug antibodies arose, necessitating tailored, highly effective, long-term treatments.

Our approach

Using our proprietary CodeEvolver® technology platform, we engineered CDX-6311, a unique variant of human alpha-galactosidase tailored for improved stability, activity, and immunogenicity profile, as a potential more efficacious alternative to treating Fabry disease. In collaboration with Takeda, Codexis continues to generate novel gene sequences encoding enzyme variants with enhanced efficacy to be used as transgenes in a variety of gene therapies, thereby pioneering next-generation, long-term treatments for rare genetic disorders.

News release

Latest News

Codexis Presents Pre-Clinical Data Highlighting Gene Therapy Programs at the ASGCT 25th Annual Meeting

Codexis Announces FDA Orphan Drug and Rare Pediatric Disease Designations for CDX 6512 for the Treatment of Homocystinuria

Scientific Highlights

Towards improving the treatment of Hemophilia A with directed evolution of transgene

Marcus Rohovie, Sr. Scientist at Codexis, presented a poster at the ASGCT Annual Meeting that highlights the potential of engineered transgenes to offer improved efficacy of Recombinant Factor VIII (FVIII) therapies in patients with Hemophilia A. The CodeEvolver® technology platform was used to identify variants of a B-domain deleted FVIII (FVIII-BDD) with superior properties as compared to the wild-type enzyme.

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