Amyotrophic lateral sclerosis (ALS)

Neurons
#Amyotrophic lateral sclerosis (ALS) #Neurodegenerative disorders #Neuroscience #NRG Therapeutics

New drug displays anti-inflammatory and neuroprotective effects in ALS

NRG Therapeutics has shared preclinical and mechanistic data of its lead clinical asset NRG5051, a treatment for amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), and Parkinson’s.  NRG disclosed that NRG5051, a first-in-class, orally bioavailable and CNS-penetrant next-generation inhibitor of the mitochondrial permeability transition… Read More

#Editor's Choice #News

AI brain
#Amyotrophic lateral sclerosis (ALS) #Cumulus Neuroscience #Digital health #Frontotemporal Dementia (FTD) #Neuroscience #Research Ireland #Trinity College Dublin

Platform could improve ALS and FTD trial endpoints 

Cumulus Neuroscience, a global digital health company, has published final results from its first-in-class CNS-102 study in Scientific Reports. The study was conducted in collaboration with the PRECISIONALS initiative, a Research Ireland academic–industry collaboration hosted by Trinity College Dublin.  The paper, titled “Longitudinal cognitive assessment using the Cumulus NeuLogiq Platform in… Read More

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#Amyotrophic lateral sclerosis (ALS) #Neuroscience #Phase I trial #Therapeutic Antibodies #VectorY Therapeutics

Vectorised antibody takes ‘innovative approach’ to ALS treatment

VectorY Therapeutics has announced that the first participant has been dosed in its Phase I/II PIONEER-ALS clinical trial of VTx-002 in people with amyotrophic lateral sclerosis (ALS).  VTx-002 is a first-in-class vectorised antibody designed to holistically target TDP-43 pathology in ALS recently granted FDA Fast… Read More

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Neurons
#Amyotrophic lateral sclerosis (ALS) #MND #Neuroscience #NRG Therapeutics #Phase I trial

Disease-modifying treatment for ALS enters Phase I

The first participants have been dosed in the first-in-human Phase I clinical trial of NRG Therapeutics’ lead candidate NRG5051, which is being developed as a treatment for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) and Parkinson’s.   NRG5051 is a first-in-class, orally bioavailable and CNS-penetrant next-generation inhibitor of the mitochondrial… Read More

#News #Trending News

Neurons in the brain
#Amyotrophic lateral sclerosis (ALS) #Appointment #Neuroscience #New appointment #NRG Therapeutics

NRG Therapeutics appoints Paul Thompson as CDO

NRG Therapeutics has expanded its team with the appointment of Paul Thompson PhD as Chief Development Officer (CDO).  In his role at NRG, Thompson will be project lead for NRG5051 which is on track to commence first-in-human studies in early 2026.   NRG5051 is an oral,… Read More

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RNA
#Amyotrophic lateral sclerosis (ALS) #Gene therapy #Neurodegenerative disorders #Tel Aviv University

RNA-based gene therapy offers new hope for ALS treatment

A team of researchers have uncovered a previously unknown molecular mechanism that drives the progression of amyotrophic lateral sclerosis (ALS) and succeeded in neutralising it using RNA-based gene therapy.   The study was conducted in the laboratory of Professor Eran Perlson from the Gray Faculty of Medical &… Read More

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#Amyotrophic lateral sclerosis (ALS) #Biotechnology #Blood-based biomarkers #MND #PLL Therapeutics

Unlocking early detection and treatment of ALS

PLL Therapeutics will present its strategic approach in the early detection and treatment of Amyotrophic Lateral Sclerosis (ALS), a form of motor neuron disease (MND), at the 19th Bioshares Biotech Summit. Bioshares is an Australian biotech stock report. The summit, hosting emerging life science companies listed on the ASX, with… Read More

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Neurons
#Amyotrophic lateral sclerosis (ALS) #Biogen #MHRA #MND #Neuroscience

First new ALS treatment approved in UK in 30 years

Biogen’s Qalsody (tofersen) has been approved in the UK as the first therapy to treat a rare, genetic form of amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND).  The Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorisation for tofersen through the International… Read More

#News #Regulatory #Trending News

Degeneration of dopaminergic neuron in Parkinson's disease
#Amyotrophic lateral sclerosis (ALS) #Dementia #Longitude P #MND #Motor neuron disease #Neuroscience

Award transforming drug discovery with AI

The Longitude Prize on ALS is a new £7.5 million ($10.28 million) global challenge prize to incentivise and reward cutting edge AI-based approaches to transform drug discovery for the treatment of amyotrophic lateral sclerosis (ALS), the most common form of motor neurone disease (MND). Open for entries from 25 June… Read More

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Synsapses
#Amyotrophic lateral sclerosis (ALS) #FDA #Neuroscience #Orphan drugs #Spinogenix

First synaptic regenerative therapy for ALS gets orphan status

The European Medicines Agency (EMA) has granted orphan drug designation (ODD) to Spinogenix’s SPG302 for the treatment of people living with amyotrophic lateral sclerosis (ALS).   SPG302 is being developed as the first synaptic regenerative approach for the treatment of ALS, with the potential to restore function in… Read More

#Editor's Choice #News #Regulatory

Human neuron
#Amyotrophic lateral sclerosis (ALS) #Early access #FDA #Neuroscience #Spinogenix

FDA okays access programme for ALS synaptic regenerative therapy

The Food and Drug Administration (FDA) has authorised Spinogenix to begin its Expanded Access Program (EAP) for SPG302 in the United States for people with amyotrophic lateral sclerosis (ALS) that meet programme eligibility criteria.  SPG302 is being developed as the first synaptic regenerative approach to treating ALS,… Read More

#News #Regulatory #Trending News

#Alchemab Therapeutics #Amyotrophic lateral sclerosis (ALS) #Dementia #Eli Lilly #Neuroscience

ALS drug licensing agreement confirmed

Alchemab Therapeutics has entered into a licensing agreement with Eli Lilly and Company (Lilly) for ATLX-1282, Alchemab’s first-in-class IND-ready programme for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions. The transaction is worth up to a total of $415 million, including an undisclosed upfront payment, potential discovery, development, and commercialisation… Read More

#Business & Investment #News

#Alchemab Therapeutics #Amyotrophic lateral sclerosis (ALS) #Collaboration #Eli Lilly

Lilly and Alchemab collaborate on novel therapies for ALS 

Eli Lilly and Alchemab Therapeutics hope to discover novel therapeutic candidates to treat amyotrophic lateral sclerosis (ALS) as part of a new research collaboration.  Alchemab’s technology platform uses patient samples from individuals with unusually slow rates of disease progression to identify antibodies associated with resilience.  … Read More

#Business & Investment #News

Brain physiology
#Amyotrophic lateral sclerosis (ALS) #Artificial intelligence (AI) #Longitude Prize #Machine learning #Motor neuron disease #Neurology #Neuroscience

Third global Longitude Prize to be launched in 2025 focusing on ALS

The Longitude Prize series from Challenge Works will launch the Longitude Prize on amyotrophic lateral sclerosis (ALS) in Spring 2025. The next installment in the prize series follows the success of the Longitude Prize on antimicrobial resistance (AMR), won in 2024 by Sysmex Astrego’s PA-100 AST system, and the Longitude… Read More

#News

#Amyotrophic lateral sclerosis (ALS) #App note #Human disease models #iPSC #Neuroscience

App Note: Human iPSC-derived disease models for ALS

Despite significant research and funding, the development of therapies for ALS has been challenging due to a lack of readily accessible human cell models, leading scientists to rely on animal models and cell lines that differ considerably from human biology. A new App Note is now available to download entitled… Read More

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#Amyotrophic lateral sclerosis (ALS) #Human disease models #iPSC #Neuroscience

Disease modelling with genetically matched iPSC-derived excitatory neurons

Download bit.bio’s application note to learn more about: Functional characterisation of human iPSC-derived ALS disease models using high throughput MEA. Phenotypic data showing reduced neuronal activity and network formation in ALS disease models. A genetically matched system in ioGlutamatergic Neurons that can be used to identify, measure and rescue disease-related… Read More

#Resources