The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and the Gene Therapy Advisory Committee (GTAC) have approved a clinical trial application (CTA) submitted by University College London (UCL) to initiate a phase I/II trial of BGT-OTCD, Bloomsbury Genetic Therapies Ltd.’s liver-targeted AAV-LK03 gene therapy, in pediatric patients with ornithine transcarbamylase deficiency (OTCD).
Glucagon-like peptide 1 (GLP-1) receptor agonists have been reported by Terns Pharmaceuticals Inc. in a recent patent. They are described as potentially useful for the treatment of diabetes and liver diseases.
The most ambitious objective of any treatment is to eradicate the disease, acting on its origin to cure it instead of treating its symptoms. This is the purpose of the gene therapy against type 2 diabetes (T2D) and obesity that Fractyl Health Inc. is developing. Scientists from the Lexington, Mass.-based company have designed a strategy based on glucagon-like peptide-1 (GLP-1) to transform pancreatic cells and reverse the disease.
Cgenetech (Suzhou, China) Co. Ltd. has described glucagon-like peptide 1 (GLP-1) receptor agonists reported to be useful for the treatment of diabetes.
Shanghai Meiyue Biotech Development Co. Ltd. has disclosed serine/threonine-protein kinase SIK2 (QIK) inhibitors reported to be useful for the treatment of metabolic, autoimmune diseases, cardiovascular, dermatological, endocrine disorders, transplant rejection, fibrosis and inflammatory disorders.
Astrazeneca AB has synthesized octahydrofuro[3,4-b]pyrazines acting as glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, cardiovascular and metabolic disorders.
Cells of Saccharomyces cerevisiae, a yeast used as a model for human mitosis, age in two ways. Both genomic instability and the decline of mitochondria cause cells to degenerate and die. The choice of one type or another depends on a network of genes that can be adjusted by bioengineering.
A recent Astrazeneca AB patent describes 2,5-diazabicyclo[4.2.0]octanes and octahydrofuro[3,4-b]pyrazines acting as glucagon-like peptide 1 receptor (GLP-1R) agonists potentially useful for the treatment of type 2 diabetes.
2-Oxo-3-azabicyclo[3.1.0]hexane derivatives acting as ketohexokinase (KHK) inhibitors have been reported in a recent Hua Medicine Ltd. patent as potentially useful for the treatment of diabetes, fibrosis, obesity, neurological, metabolic, gastrointestinal, cardiovascular and renal diseases, among others.