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BioWorld - Sunday, January 4, 2026
Home » amyotrophic lateral sclerosis

Articles Tagged with ''amyotrophic lateral sclerosis''

Neurology/psychiatric

Highly selective HDAC6 inhibitor against neurodegenerative disease

Oct. 14, 2025
No Comments
Inhibiting histone deacetylase 6 (HDAC6) has therapeutic potential against several neurodegenerative disorders. A collaboration including researchers from Eikonizo Therapeutics Inc., spanning the U.K., U.S. and France, developed EKZ-438, which has shown strong potential against amyotrophic lateral sclerosis and frontotemporal dementia in preclinical studies.
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Brain-DNA illustration
Rare Genetic Disease Symposium

More stem cell, gene therapies progressing in clinic for ALS

Sep. 23, 2025
By Marian (YoonJee) Chu
No Comments
Amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disorder with no known cure. While three therapies have gained U.S. FDA approvals to date, including Rilutek (riluzole), Radicava/Radicava ORS (edaravone) and tofersen (BIIB-067, the lack of a disease-modifying drug has spurred the continual search for novel therapies.
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Illustration of green and yellow antibodies, DNA
Neurology/psychiatric

Vectory and Shape Therapeutics sign option and license agreement

Sep. 19, 2025
No Comments
Vectory Therapeutics BV and Shape Therapeutics Inc. have entered into an option and license agreement granting Vectory an exclusive option to evaluate Shape’s deep brain penetrating AAV capsid, SHP-DB1, for vectorized antibody payloads against three therapeutic targets.
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Neurology/psychiatric

Ono and Wuxi Apptec disclose new S1P3 receptor antagonists

Sep. 16, 2025
Ono Pharmaceutical Co. Ltd. and Wuxi Apptec Co. Ltd. have divulged sphingosine 1-phosphate S1P3 receptor antagonists reported to be useful for the treatment of cancer, liver diseases, neurodegeneration, and metabolic, endocrine, cardiovascular and respiratory disorders.
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Neil Miller, CEO, NRG Therapeutics
Neurology/psychiatric

NRG raises $67M to test mitochondrial pore inhibitor in ALS

Sep. 9, 2025
By Nuala Moran
No Comments
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
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Neil Miller, CEO, NRG Therapeutics

NRG raises $67M to test mitochondrial pore inhibitor in ALS

Sep. 8, 2025
By Nuala Moran
No Comments
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
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Brain as light bulb filament
Neurology/psychiatric

Financing to advance Leal Therapeutics’ neuro-metabolic pipeline

Aug. 28, 2025
No Comments
Leal Therapeutics Inc. has raised $30 million in series A financing to advance its neuro-metabolic pipeline of therapeutics that aim to correct metabolic imbalances in the brain in patients with neuropsychiatric or neurodegenerative disorders.
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Neural network
Neurology/psychiatric

Targeted antibody intervention restores PGRN levels and TDP43 localization in ALS models

Aug. 1, 2025
No Comments
GSK plc scientists have presented findings from studies on the therapeutic potential of GSK-5862611, an anti-Sortilin antibody, in addressing neurodegenerative disease mechanisms linked to the TDP43 G298S mutation.
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Illustration of motor neuron connecting to muscle fiber
Neurology/psychiatric

Preclinical study confirms positive modulation of Kv3 channels as therapeutic option for ALS

July 28, 2025
No Comments
Researchers from Biotiche Drug Discovery Srl and collaborators have discovered that modulating the activity of a specific family of potassium channels, known as Kv3 channels, can have beneficial effects on the progression of amyotrophic lateral sclerosis (ALS) in a mouse model of the disease. The study, published in the journal Acta Neuropathologica Communications, provides new insights into the role of these channels in skeletal muscle function and their potential as a therapeutic target for ALS.
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Brain and neural networks
Biomarkers

Proteomics finds surprise commonalities as well as differences in neurodegenerative diseases

July 15, 2025
By Nuala Moran
No Comments
The switch will be flicked today to make the world’s largest dementia-related proteomics dataset freely available to researchers, at the same time as members of the consortium which compiled it publish the proteomics signatures of major neurodegenerative diseases that they uncovered in a first trawl of the data.
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