5-Hydroxyindoleacetic acid

Homology Medicines Announces Peer-Reviewed Publication of HMI-102 Investigational Gene Therapy Demonstrating Restoration of Normal Metabolic Pathway in PKU Disease Model

Retrieved on: 
Monday, March 16, 2020
Amino acids, Branches of biology, Carboxylic acids, Hydroxyarenes, Metabolism, Intellectual disability, Phenylketonuria, RTT, Neurotransmitter, Metabolite, Tyrosine, 5-Hydroxyindoleacetic acid

The data also demonstrated a concomitant increase in tyrosine (Tyr), a metabolite of Phe and precursor to neurotransmitters, indicating enzymatic activity.

Key Points: 
  • The data also demonstrated a concomitant increase in tyrosine (Tyr), a metabolite of Phe and precursor to neurotransmitters, indicating enzymatic activity.
  • Additionally, brain levels of Phe, 5-HIAA (downstream serotonin metabolite) and coat color were normalized, further indicating restoration of the Phe metabolic pathway.
  • We developed a robust preclinical data package for our investigational HMI-102 gene therapy, which supported the initiation of our ongoing Phase 1/2 pheNIX clinical trial for adults with PKU, stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines.
  • These published data demonstrated that a single dose of HMI-102 was able to restore the normal biochemical pathway in the established PKU model on normal protein diet.

Lexicon Pharmaceuticals Announces Real-World Data on Telotristat Ethyl’s Antiproliferative Effects in Patients With Carcinoid Syndrome

Retrieved on: 
Friday, January 24, 2020
Medical specialties, Medicine, Organ systems, Gastrointestinal cancer, Neuroendocrinology, Carcinoid syndrome, Carcinoid, Neuroendocrine tumor, Endocrine oncology, Diarrhea, 5-Hydroxyindoleacetic acid, VIPoma

Such preclinical data and the results of this observational study support the conduct of clinical trials to examine telotristat ethyls effect on neuroendocrine tumors.

Key Points: 
  • Such preclinical data and the results of this observational study support the conduct of clinical trials to examine telotristat ethyls effect on neuroendocrine tumors.
  • Discovered using Lexicons unique approach to gene science, XERMELO is the first and only approved oral therapy for carcinoid syndrome diarrhea.
  • Carcinoid syndrome is a rare condition that occurs in patients living with mNETs and is characterized by frequent and debilitating diarrhea.
  • XERMELO targets the overproduction of serotonin inside mNET cells, providing an additional treatment option for patients suffering from carcinoid syndrome diarrhea.