Articles Tagged with ''amyotrophic lateral sclerosis''

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
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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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Tree in the shape of human head losing leaves
Neurology/psychiatric

Encoding an entire mAb against TDP-43 into AAV to treat neurodegenerative diseases

June 30, 2025
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Monoclonal antibodies show strong therapeutic potential against neurodegenerative diseases, but a major challenge is ensuring that they can arrive unharmed in the brain at sufficient concentrations. One way around this challenge is to vectorize the monoclonal antibody: encode it within a vector that can infect target cells in the brain and express the antibody in situ.
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Neuronata-R

Corestemchemon preps US BLA filing for stem cell ALS therapy

June 10, 2025
By Marian (YoonJee) Chu
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Corestemchemon Inc. is planning to file a BLA for Neuronata-R (lenzumestrocel) by the end of 2025 to gain accelerated approval from the U.S. FDA, company officials confirmed to BioWorld during a June 2 interview. Neuronata-R is an autologous bone marrow-derived mesenchymal stem cell therapy that first gained approval in South Korea in 2014 to delay the progression of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder also known as Lou Gehrig’s disease.
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Neuronata-R

Corestemchemon preps US BLA filing for stem cell ALS therapy

June 9, 2025
By Marian (YoonJee) Chu
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Corestemchemon Inc. is planning to file a BLA for Neuronata-R (lenzumestrocel) by the end of 2025 to gain accelerated approval from the U.S. FDA, company officials confirmed to BioWorld during a June 2 interview. Neuronata-R is an autologous bone marrow-derived mesenchymal stem cell therapy that first gained approval in South Korea in 2014 to delay the progression of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder also known as Lou Gehrig’s disease.
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Conceptual 3D illustration showing degradation of motor neuron
Neurology/psychiatric

Alchemab and Lilly sign licensing agreement for ATLX-1282

May 6, 2025
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Alchemab Therapeutics Ltd. has entered into a licensing agreement with Eli Lilly and Co. for ATLX-1282, Alchemab’s first-in-class IND-ready program targeting a novel receptor and mechanism for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions.
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Neurosense phase II combo to hail new Paradigm in ALS?

April 9, 2025
By Randy Osborne
As developers continue to search for better amyotrophic lateral sclerosis (ALS) therapies, Neurosense Therapeutics Ltd. turned up some hopeful findings from its phase IIb Paradigm trial with PrimeC. The drug, a combination therapy (ciprofloxacin and celecoxib) designed to target multiple ALS pathways, is having salutary effects on microRNA modulation (miRNA), Neurosense said, with the study showing a “profound and consistent” downregulation of 161 mature miRNAs across all time points in the double-blind period of the experiment.
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