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BioWorld - Wednesday, December 17, 2025
Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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AI-generated illustration of spinal cord
Neurology/psychiatric

Dewpoint and Mitsubishi ink $480M ALS research collaboration

Dec. 10, 2024
By Tamra Sami
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an undisclosed up-front payment and is eligible to receive R&D-based milestone payments up to $480 million. Upon reaching those milestones, Osaka, Japan-based MTPC will have an exclusive option to license the program and assume responsibility for global clinical development and commercialization. Dewpoint will also receive tiered royalties on net sales.
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Paradigm's AU$16M moves Zilosul to pivotal osteoarthritis trials

Dec. 9, 2024
By Tamra Sami
Paradigm Biopharmaceuticals Ltd. raised AU$16 million (US$10.31 million) in a placement that will allow the company to start a global phase III trial with its lead product, Zilosul (injectable pentosan polysulfate/iPPS), to treat osteoarthritis.
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AI-generated illustration of spinal cord

Dewpoint and Mitsubishi ink $480M ALS research collaboration

Dec. 6, 2024
By Tamra Sami
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an undisclosed up-front payment and is eligible to receive R&D-based milestone payments up to $480 million. Upon reaching those milestones, Osaka, Japan-based MTPC will have an exclusive option to license the program and assume responsibility for global clinical development and commercialization. Dewpoint will also receive tiered royalties on net sales.
Read More
D-rendered image showing atlas of human embryonic skeletal development
Drug design, drug delivery & technologies

More than 100M cells included in the human cell atlas

Nov. 26, 2024
By Mar de Miguel
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.
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Illustration of muscle tissue anatomy
Musculoskeletal

Dystrophies affect not just muscles; can RNA editing help?

Nov. 26, 2024
By Mar de Miguel
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.
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Celltrion wins Korea approvals of Prolia, Xgeva biosimilars

Nov. 26, 2024
By Marian (YoonJee) Chu
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.
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Sarepta ‘secures future’ with massive Arrowhead collaboration

Nov. 26, 2024
By Jennifer Boggs
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.
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Illustration of man holding magnifying glass to human body model showing muscle anatomy
Musculoskeletal

In muscular dystrophies, gene therapies race the clock

Nov. 25, 2024
By Mar de Miguel
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.
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Immune

Seed funding at Autoimmunity Biosolutions to advance immuno-corrective therapy for autoimmune diseases

Nov. 22, 2024
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.
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Celltrion wins Korea approvals of Prolia, Xgeva biosimilars

Nov. 22, 2024
By Marian (YoonJee) Chu
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.
Read More
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