Chronic kidney disease (CKD) during diabetes may manifest several phenotypes, including diabetic nephropathy (DN), nondiabetic renal disease (NDRD) or a mixed form.
Alpha-1 antitrypsin deficiency (AATD) is a recessive genetic disorder caused by single nucleotide variants (SNV) in the SERPINA1 gene encoding for alpha-1 antitrypsin (AAT), with the most common mutation being the E342K mutation (Z allele) that introduces an amino acid change from glutamic acid (E) coding for M-AAT to a lysine (K) coding for Z-AAT.
Researchers from Duke University presented findings from a study that aimed to assess the physiological role of Piezo1, a mechano-gated ion channel that is highly expressed in pancreatic acinar cells, in the exocrine pancreas.
Prologue Medicines Inc. has launched to develop therapeutics created from the viral proteome, which are proteins produced across all viruses. It’s a newer twist on harnessing the power of the proteins that regulate biology. The company was founded on the premise of peering beyond the human proteome to see if there were viral proteins that could become therapeutics, Theonie Anastassiadis, Prologue’s founding president, told BioWorld.
Sixpeaks Bio AG has emerged from stealth with $30 million in series A financing and a pipeline of therapies for healthy weight loss, including a dual-specific antibody targeting activin type IIA and B receptors for preservation of skeletal muscle mass.
The FDA has cleared an IND application for GC-1130A, a treatment for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) being jointly developed by GC Biopharma Corp. and Novel Pharma Inc.
CSPC Pharmaceutical Group Ltd.’s anti-βKlotho monoclonal antibody drug JMT-202 has received clearance from China’s National Medical Products Administration (NMPA) to enter clinical trials to lower triglyceride levels in patients with hypertriglyceridemia.
The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.
Centogene NV and Evotec SE have announced the discovery of a new small molecule with potential to treat patients with type 2 and type 3 Gaucher disease, or neuronopathic Gaucher disease.