Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the sarcolemma and protects muscle cells during contraction.
As in other muco-obstructive diseases, the airways in cystic fibrosis (CF) are characterized by goblet cell and glandular hyperplasia, with overproduction of mucins MUC5 and MUC5AC, resulting in viscous mucus, respiratory blockade and recurrent infections and inflammation.
A team of scientists from the Perelman School of Medicine at the University of Pennsylvania and Aum Biotech LLC have described the development of a novel cancer immunotherapy designed to target FOXP3-positive T regulatory cells (Tregs) with a next generation of antisense oligonucleotides (ASOs), termed FOXP3 Aumsilence ASO. In contrast to previous ASOs, FOXP3 Aumsilence ASOs do not require delivery agents, and are capable of highly specific RNA silencing of previously undruggable targets.
Acurastem Inc. has secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to facilitate the development of its UNC13A program toward clinical trials for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
Flamingo Therapeutics BV and Ionis Pharmaceuticals Inc.’s FTX-001 (also known as FLM-7523) is a potential first-in-class antisense oligonucleotide (ASO) targeting the human metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) long noncoding RNA (lncRNA); it is being developed for the treatment of cancer.
Bolden Therapeutics Inc. has closed a $1.5 million pre-seed convertible note financing. This financing, together with National Institutes of Health (NIH) small business grants, will support preclinical development of Bolden's antisense oligonucleotides to promote neurogenesis.
Lysophosphatidylcholine acyltransferase 3 (LPCAT3), highly expressed in the liver, intestine, and adipose tissues, is an enzyme that preferentially incorporates polyunsaturated fatty acyl chain into lysophospholipids. Previous work showed that LPCAT3 and phospholipid remodeling play a crucial role in regulating glucose metabolism and contribute to the development of insulin resistance in type 2 diabetes.
Vanda Pharmaceuticals Inc. and Olipass Corp. have entered into a research and development collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on Olipass' proprietary modified peptide nucleic acids.
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