Sr. VP, Strategic Development, Codexis
Pictured above: Codexis employees participated in the company’s 2020 Rare Disease Day event.
Effective partnerships between experts in protein engineering and drug marketers will result in better treatments for rare diseases reaching patients faster.
Phenylketonuria (PKU) is a genetic condition that causes intellectual disability when left untreated and treatment typically imposes a very restricted, life-long diet. New treatments stand to revolutionise outcomes for this condition as, by the time people with PKU reach adulthood, typically only 10% are sticking with the recommended, demanding diet. In an example of how collaborative thinking can benefit patients, protein engineering expert, Codexis, has teamed up with pharmaceutical marketing giant, Nestlé Health Science, to develop an oral treatment for phenylketonuria (PKU).
Getting treatments to rare disease patients sooner
Another rare disease, Fabry disease, causes problems in the heart and kidney and, left untreated, can greatly reduce life expectancy. To generate a better treatment option for Fabry patients, Codexis is working with Takeda to generate better versions of the enzyme whose activity is missing in these patients, for administration as gene therapy.
Dr Gjalt Huisman, Codexis senior vice-president, strategic development, and general manager of Codexis’ Biotherapeutics business, says: “Unfortunately, there are many rare diseases where there is either no treatment, or if there is, they may not be all that efficacious.” Teaming up with leading pharmaceutical companies, means that Codexis’ drug discovery engine can bring more, and more effective treatments to market, and sooner.
Teaming up with leading pharmaceutical companies, means that Codexis’ drug discovery engine can bring more, and more effective treatments to market, and sooner.
Building and replacing missing enzymes for patients
There are ~6,000 different hereditary rare diseases, which means that they are caused by genetic changes. Codexis’ focus is to develop new drug candidates that are best- or first-in-class for disorders with, currently, suboptimal treatments. Candidates for novel enzyme therapeutics may also include treatments for non-hereditary indications, for instance gluten and other gastric intolerances, which also demand strict, life-long dietary adherence.
Dr Huisman explains: “If a vital enzyme is missing or dysfunctional, harmful chemical molecules can start to accumulate in the body, and this can create havoc.” In the sphere of gene therapy, the focus is on developing an optimally-functioning replacement for a dysfunctional or absent gene.
Codexis’ focus in the Biotherapeutics world is two-fold: locally-administered enzyme therapeutics and gene therapies. These therapeutics involve replacing non-functional enzymes in the body, with functional ones, to ensure that the chemical processes in the body can proceed unencumbered. Dr Huisman says: “This is a rapidly developing field, with an improving safety and efficacy record and in-market experience for treating such disorders; however, better treatments continue to be needed to truly improve patients’ quality of life.”