The biotechnology company Synlogic announced positive data from its most recent Phase 1/2a clinical trial of its experimental product SYNB1618. This trial did not test the therapeutic action of the drug, but was instead meant to demonstrate that the product was safe and well tolerated in healthy individuals. SYNB1618 is in development for the treatment of phenylketonuria, a rare metabolic disorder. Synlogic is committed to the development of new therapies utilizing synthetic biology.
About Phenylketonuria
Phenylketonuria is a congenital metabolic disorder in which the body is unable to process a certain amino acid called phenylalanine. The disease is the result of mutations affecting the PAH gene which prevents the enzyme that would normally process phenylalanine from functioning. Symptoms of phenylketonuria can include mental disorders, pale skin, a distinct musty body odor, intellectual disability, behavioral problems, and seizures. Most countries screen newborns for this disease. Prompt management and treatment is necessary to avoid symptoms. The disease is able to be managed with a highly precise diet that limits protein intake, and phenylalanine is often found in protein rich foods. There are also some supplements that can be used to compensate for a low protein diet. To learn more about phenylketonuria, click here.
Good Signs For SYNB1618
The study also demonstrated varying levels of specific biomarkers that SYNB1618 is expected to manipulate, demonstrating that the drug’s operative mechanism appears to be working as intended. Overall the study suggests that SYNB1618 could be a safe and effective treatment for phenylketonuria. The therapy has received both Orphan Drug designation and Fast Track designation from the FDA.
Get Involved In The Next Stage of Research
Are you a phenylketonuria patient? You could be a part of the next step of Synlogic’s clinical trial process. The next clinical trial is currently open for recruitment in Salt Lake City, Utah, and other recruitment sites will be in operation soon. The company will compensate for travel expenses, and trial participants will receive the experimental drug, as well as thorough evaluations, monitoring, and follow-up after receiving treatment.
There are several requirements that patients must fulfill in order to be a part of the research.
- Be on a stable diet
- Have a diagnosis of classic phenylketonuria
- Be between the ages of 18 and 64
- Have phe levels that exceed 600 μmol/L
