
Oral Enzymatic Management of PhenylketonuriaAward last edited on: 5/4/2020
Sponsored Program
STTRAwarding Agency
NIH : NIDDKTotal Award Amount
$225,000Award Phase
1Solicitation Topic Code
847Principal Investigator
Anil P D'MelloCompany Information
Phase I
Contract Number: 1R41DK122897-01Start Date: 9/17/2019 Completed: 8/31/2020
Phase I year
2019Phase I Amount
$225,000Public Health Relevance Statement:
Phenylketonuria is an inborn error of metabolism, characterized by mutations in the enzyme phenylalanine hydroxylase causing a pathological elevation of phenylalanine in the blood. Orally active enzyme replacement therapy has shown initial promise but limited efficacy as native processes break down the exogenous enzymes resulting in a transient reduction in phenylalanine levels. Microencapsulation of enzymes in a porous and non-toxic membrane represents an innovative way to prolong stability and functionality in the destructive environment of the gastrointestinal tract and has the potential to help alleviate the enormous burden experienced by patients.
Project Terms:
Address; Adult; alternative treatment; Amino Acids; aqueous; Biological; Blood; Cellulose; Chemical Structure; Child; chymotrypsin; cinnamic acid; Classical phenylketonuria; Consumption; design; Development; Diet; Diet therapy; dietary control; Dietary Proteins; dietary restriction; Dose; Emulsions; Encapsulated; Ensure; Environment; Enzyme Kinetics; enzyme replacement therapy; Enzymes; Essential Amino Acids; Excision; Exhibits; experience; experimental study; Food; food restriction; Food Supplements; Formulation; Future; Gastrointestinal tract structure; Genetic Diseases; Goals; Guidelines; High Pressure Liquid Chromatography; In Vitro; in vitro Model; Inborn Errors of Metabolism; Incubated; Infant; innovation; Intestines; Left; Legal patent; Life; Liquid substance; medical food; Membrane; Metabolic Diseases; Methylene Chloride; Microcapsules drug delivery system; Microencapsulations; Modeling; Mutation; Neonatal Screening; Neurologic Deficit; new technology; non-compliance; off-patent; Oral; Oral Administration; Organic solvent product; Pathologic; Patients; Pepsin A; Peptide Hydrolases; Pharmacologic Substance; Phase; Phenylalanine; Phenylalanine Ammonia-Lyase; Phenylalanine Hydroxylase; Phenylalanine Metabolism Pathway; Phenylketonurias; Plants; Plasma; Polymers; preservation; Problem behavior; Procedures; Process; process optimization; Production; Property; protective effect; Regulation; Research; resilience; Resistance; Schedule; Seizures; severe intellectual disability; Small Intestines; stem; Stomach; Structure; symptom management; Techniques; Temperature; Time; Trypsin; United States
Phase II
Contract Number: ----------Start Date: 00/00/00 Completed: 00/00/00