At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
The Ministry of Food and Drug Safety (MFDS) approved South Korea’s first denosumab (Prolia/Xgeva) biosimilars, developed by Celltrion Inc. under the brand names of Stoboclo/Osenvelt (CT-P41) for respective indications, a move the company hopes will help secure first-mover advantage for the drugs, currently under review in the U.S. and Europe.
In the largest collaboration of 2024, Sarepta Therapeutics Inc. and Arrowhead Pharmaceuticals Inc. entered a sprawling global licensing deal that includes a swath of clinical and preclinical candidates targeting rare genetic diseases. Under the terms, Sarepta gains access to existing and potential future compounds derived from the RNAi platform developed by Arrowhead, with the latter eligible for payments potentially exceeding $11 billion.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
Autoimmunity Biosolutions has closed a seed financing to support its work advancing a next-generation, immuno-corrective therapy for autoimmune diseases. The company’s approach targets a branch of the interleukin-7 (IL-7)/interleukin-7 receptor (IL-7R) pathway controlled by the soluble IL-7R (sIL-7R), a critical amplifier of autoimmune reactions.
The Ministry of Food and Drug Safety (MFDS) approved South Korea’s first denosumab (Prolia/Xgeva) biosimilars, developed by Celltrion Inc. under the brand names of Stoboclo/Osenvelt (CT-P41) for respective indications, a move the company hopes will help secure first-mover advantage for the drugs, currently under review in the U.S. and Europe.
An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.
Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
The bromodomain and extra terminal domain (BET) family of proteins, including BRD4, regulates the transcription of multiple proinflammatory and immunoregulatory genes. BRD4 is involved in immune-related disorders such as rheumatoid arthritis (RA).
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