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Home » amyotrophic lateral sclerosis

Articles Tagged with ''amyotrophic lateral sclerosis''

EU flag, syringe, capsules

CHMP nods for Korjuny, Alhemo; no, again, for Translarna, masitinib

Oct. 21, 2024
By Nuala Moran
The first bispecific antibody to win regulatory approval is about to make a comeback 10 years after being taken off the market in Europe for commercial reasons. Catumaxomab, then called Removab, and now reborn with the brand name Korjuny, received a positive opinion for the treatment of malignant ascites from the EMA’s Committee for Medicinal Products for Human Use (CHMP,) at its monthly meeting Oct. 14 to 17.
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Brain and DNA

Aviadobio, Astellas ink $2B+ frontotemporal dementia gene therapy deal

Oct. 8, 2024
By Nuala Moran
Aviadobio Ltd. has entered a potential $2.18 billion license and commercialization agreement for its frontotemporal dementia gene therapy, AVB-101, with Astellas Pharma Inc. Astellas is making a $20 million equity investment in London-based Aviadobio and will pay up to $30 million up front in advance of deciding whether or not to exercise the exclusive option to worldwide rights.
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Illustration of human brain and dna
Neurology/psychiatric

Reverse engineering opens path for precisely targeted gene therapies for ALS and FTD

Oct. 4, 2024
By Nuala Moran
Researchers in the U.K. have succeeded in reverse engineering the defective cryptic splicing that drives amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) to enable precisely targeted delivery of transgenes and therapeutic protein expression in diseased neurons. The technique is compatible with conventional adeno-associated viral vectors that are approved for gene therapy, and can readily be adapted for different transgenes. ALS, FTD and other neurogenerative diseases are underpinned by loss of function of the RNA-binding protein TDP-43 (transactive response DNA-binding protein 43), that normally functions as a key regulator of splicing, protecting the transcriptome from toxic cryptic exons.
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Neurology/psychiatric

Poltreg studies CAR-Treg cells in neuroinflammatory mouse model

Sep. 26, 2024
Poltreg SA has initiated a preclinical study with CAR-Treg cells in a humanized mouse model of neuroinflammatory disease.
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Global currency
Neurology/psychiatric

Grants support Mabylon’s programs for ALS and inflammatory bowel disease

Sep. 25, 2024
Mabylon AG has been awarded three grants totaling more than CHF1.3 million (US$1.5 million) from Innosuisse Swiss Innovation Agency, Target ALS and the ALS Association.
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Illustration of motor neuron connecting to muscle fiber
Neurology/psychiatric

ARGX-119 prevents early postnatal lethality and reverses disease relapse in model of congenital myasthenia

Sep. 23, 2024
Argenx SE’s ARGX-119 is a monoclonal antibody (MAb) targeting the muscle-specific kinase (MuSK) Frizzled (Fz)-like domain, and has entered early clinical development for the treatment of neuromuscular diseases.
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Brain illustration
Neurology/psychiatric

QED-701 results in significant phenotypic improvements in models of neurodegenerative disease

Sep. 9, 2024
It has been previously demonstrated that the activation of spleen tyrosine kinase (SYK) contributes to processes that are central to the pathogenesis of neurodegenerative diseases, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD).
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Illustration of motor neuron connecting to muscle fiber
Neurology/psychiatric

Grant supports study of NRG Therapeutics’ mPTP inhibitors for ALS

Sep. 5, 2024
NRG Therapeutics Ltd. has been awarded a grant from Target ALS Foundation to support its discovery program for a treatment for amyotrophic lateral sclerosis (ALS).
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Biotech deal illustration
Neurology/psychiatric

CIRM grant supports Acurastem’s UNC13A program for ALS and FTD

Sep. 4, 2024
Acurastem Inc. has secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to facilitate the development of its UNC13A program toward clinical trials for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
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Cell research illustration
Musculoskeletal

ISSCR 2024: iPS cell line panels can be isogenic and diverse

July 15, 2024
By Anette Breindl
The big advantage of cell culture to model diseases is its throughput. “You can play the disease over and over again in the dish,” Clive Svendsen told the audience at the International Society of Stem Cell Research (ISSCR) Annual Meeting held in Hamburg last week. That high throughput, however, is not particularly useful if the cell lines themselves do not accurately model the disease. Cancer cell lines are used in many cell culture experiments far beyond cancer for their ability to grow. But they are “highly abnormal,” Bill Skarnes told the audience at an innovation showcase, as well as quite unstable. “I don’t think the [HEK-293] cell line is the same in your lab as it is in the lab next door,” Skarnes said.
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