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BioWorld - Thursday, April 9, 2026
Home » amyotrophic lateral sclerosis

Articles Tagged with ''amyotrophic lateral sclerosis''

Brain with handshake and cityscape
Neurology/psychiatric

Transition Bio and Voyager Therapeutics partner in ALS and FTD

Nov. 11, 2025
No Comments
Transition Bio Inc. and Voyager Therapeutics Inc. have entered into a drug discovery collaboration and license option agreement for novel, selective small molecules for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) with TDP-43 pathology.
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Chris Lai, cofounder and CEO, Metis at Nanoforge launch

Metis plans China NDA submission for orally dissolving PBA drug

Oct. 28, 2025
By Marian (YoonJee) Chu
No Comments
Based on positive phase III study results, Metis Techbio is planning to file an NDA for its AI-derived orally disintegrating tablet drug candidate for pseudobulbar affect, MTS-004, in China next year.
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Chris Lai, cofounder and CEO, Metis at Nanoforge launch

Metis plans China NDA submission for orally dissolving PBA drug

Oct. 27, 2025
By Marian (YoonJee) Chu
No Comments
Based on positive phase III study results, Metis Techbio is planning to file an NDA for its AI-derived orally disintegrating tablet drug candidate for pseudobulbar affect, MTS-004, in China next year.
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Neurology/psychiatric

Study identifies REST as biomarker and siRNA target in ALS

Oct. 21, 2025
No Comments
Restrictive element-1 silencing transcription factor (REST) has key roles in neuronal differentiation, structural remodeling and plasticity, contributing to neuronal homeostasis in postnatal neurons. It acts as a suppressor of neuronal gene expression in stem and progenitor cells, and abnormal accumulation of it has been linked to several neurological disorders, like Huntington’s disease, epilepsy and stroke.
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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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