Synlogic Presents at Global PKU Patient Meeting
- Results of patient and caregiver survey highlight unmet need in PKU, burden of disease, and benefits from addition of natural protein to diet -
The presentation, "Synthetic Biotic Medicines for the Treatment of PKU," was delivered by Dr.
- Two-thirds of adult and one-third of pediatric PKU patients do not have Phe levels within the target range, despite a restrictive low phenylalanine diet and treatment with currently available therapies.
- Greater than 90% of patients and 95% of pediatric caregivers would like to increase the amount of natural protein in their diet.
- More than 50% of adult respondents and more than 80% of pediatric caregiver respondents found 2-3 grams of additional natural protein per day "meaningful."
- One slice of bread or one medium sized potato contains 2-3 grams of natural protein.
The survey was conducted through partnerships with the
Phenylketonuria (PKU) is an inherited metabolic disease that manifests at birth and is marked by an inability to break down phenylalanine (Phe), an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic and can lead to serious neurological and neuropsychological problems affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, infants are screened at birth in many countries to ensure early diagnosis and treatment to avoid intellectual disability and other complications.
SYNB1618 is an investigational oral drug for the treatment of Phenylketonuria (PKU) composed of an engineered Synthetic Biotic designed to lower plasma phenylalanine (Phe) levels by consuming Phe in the GI tract. A solid oral lyophilized formulation of SYNB1618 was found to be safe and well-tolerated, and consumes Phe in the GI tract of healthy volunteers.
Synlogic™ is bringing the transformative potential of synthetic biology to medicine. With a premiere synthetic biology platform that leverages a reproducible, modular approach to microbial engineering,
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