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BioWorld - Friday, December 19, 2025
Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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3D illustration of transparent human torso with close up of spinal cord
Musculoskeletal

GRWD-0715 blocks pathogenic T-cell activation in axial spondyloarthritis

July 3, 2025
No Comments
Researchers from Grey Wolf Therapeutics Ltd. presented the preclinical characterization of GRWD-0715, a selective ERAP1 inhibitor developed as a disease-modifying therapeutic approach aimed at preventing CD8+ T-cell activation and the subsequent tissue inflammation and damage associated with autoimmune conditions.
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Deal handshake with coin, chart background

After axing 95% workforce, Vor bets $4B+ on Remegen’s telitacicept

July 1, 2025
By Marian (YoonJee) Chu
No Comments
Just a month after laying off 147 employees and announcing plans to mull “strategic alternatives,” Vor Biopharma Inc. reported raising $175 million in private placement in public equity financing and inking a new $4.23 billion license deal for Yantai Rongchang Biotechnologies (Remegen) Co. Ltd.’s telitacicept, a dual-target fusion protein drug approved in China for three autoimmune indications. The news was disclosed after U.S. market hours June 25. Vor’s shares (NASDAQ:VOR) gained 34 cents, or 60.5%, to close June 26 at 89 cents. The company’s shares had risen for eight consecutive trading days since June 17.
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Illustration of the β2-adrenergic receptor
Endocrine/metabolic

Exercise-mimicking compound offers alternative to GLP-1 therapies

June 27, 2025
By Mar de Miguel
No Comments
An experimental drug for treating diabetes and obesity has been shown to lower blood sugar levels and increase fat burning. It is a β2-adrenergic receptor (β2AR) agonist that mimics the effects of physical exercise by activating skeletal muscle metabolism. Unlike GLP-1-based treatments such as semaglutide and tirzepatide, this new compound, developed by researchers at the Karolinska Institute, Stockholm University, and the biotech company Atrogi AB, does not suppress appetite or cause muscle loss. 
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Limb exam

Edgewise FDA try goes sideways as sevasemten Becker hope lives on

June 26, 2025
By Randy Osborne
No Comments
Edgewise Therapeutics Inc. CEO Kevin Koch speculated that “perhaps a different environment at the FDA” from four months ago led to reviewers’ caution on sevasemten, his firm’s fast skeletal myelin inhibitor for Becker muscular dystrophy and Duchenne muscular dystrophies.
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Deal handshake with coin, chart background

After axing 95% workforce, Vor bets $4B+ on Remegen’s telitacicept

June 26, 2025
By Marian (YoonJee) Chu
No Comments
Just a month after laying off 147 employees and announcing plans to mull “strategic alternatives,” Vor Biopharma Inc. reported raising $175 million in private placement in public equity financing and inking a new $4.23 billion license deal for Yantai Rongchang Biotechnologies (Remegen) Co. Ltd.’s telitacicept, a dual-target fusion protein drug approved in China for three autoimmune indications. The news was disclosed after U.S. market hours June 25. Vor’s shares (NASDAQ:VOR) gained 34 cents, or 60.5%, to close June 26 at 89 cents. The company’s shares had risen for eight consecutive trading days since June 17.
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CBER shakeup points to further FDA realignment

June 20, 2025
By Lee Landenberger
No Comments
The realignment within the U.S. FDA continued with reports of the removal of two high level executives. When asked by BioWorld if the Center for Biologics Evaluation and Research’s (CBER) Office of Therapeutic Products director and deputy director had been forced out and if so, why, an HHS spokesperson responded on background with a single sentence: “Center directors deserve to be supported by managers that are aligned with aggressive goals to expeditiously advance therapeutics for rare diseases using the gold standard of science.”
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Musculoskeletal

New TYK2 inhibitors disclosed in Sudo Biosciences patents

June 19, 2025
Sudo Biosciences Ltd. has divulged non-receptor tyrosine-protein kinase TYK2 (JH2 domain) inhibitors reported to be useful for the treatment of rheumatoid arthritis, multiple sclerosis, psoriasis, systemic lupus erythematosus, radiographic axial spondyloarthritis (ankylosing spondylitis), idiopathic pulmonary fibrosis, Sjögren’s syndrome and type 1 diabetes.
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Test tubes, dropper and capsules
Musculoskeletal

SAN-523 demonstrates anti-inflammatory efficacy in preclinical models of microcrystalline arthropathies

June 18, 2025
No Comments
University of Lausanne has reported promising preclinical data on SAN-523, a first-in-class positive allosteric modulator of cystathionine gamma-lyase (CSE), developed as a potential therapeutic for microcrystalline arthropathies, including gout and calcium pyrophosphate deposition (CPPD) disease.
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Dyne vHOT in DM1 but shares cool on endpoint switch

June 17, 2025
By Randy Osborne
No Comments
The revised trial protocol that means a delay in filing for U.S. approval of DYNE-101 to treat myotonic dystrophy type 1 (DM1) dented shares of Dyne Therapeutics Inc. (NASDAQ:DYN), which closed June 17 at $10.86, down $2.96, or 21%.
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Musculoskeletal

New tracer has diagnostic utility for rheumatoid arthritis

June 17, 2025
No Comments
Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by inflammation and damage of the joints. PET/CT imaging is a useful tool for early disease assessment and monitoring treatment efficacy.
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