TDP-43

Reduced activity of the TDP-43 protein, a hallmark of amyotrophic lateral sclerosis (ALS), leads to changes in the DNA of nerve cells, which alters the activity of important genes, a new study reports. These findings may help explain how problems with this protein can contribute to the death of…

Dewpoint Therapeutics has received a grant from the Target ALS Foundation to advance the development of small molecules called c-mods, which will target the TDP-43 condensates thought to drive most cases of amyotrophic lateral sclerosis (ALS). Condensates are borderless compartments in cells that can cause cellular processes…

VectorY Therapeutics has raised about $138 million in funding aiming to bring into clinical testing VTx-002, its lead treatment candidate for amyotrophic lateral sclerosis (ALS). The therapy equips nerve cells to produce antibodies against the damaging TDP-43 protein clumps that are an ALS hallmark. “The investment…

Takeda has obtained an exclusive, worldwide license to AcuraStem’s investigational therapies targeting the PIKfyve enzyme, including AS-202, a potential treatment for amyotrophic lateral sclerosis (ALS) that ultimately aims to lower toxic TDP-43 protein buildup in nerve cells. While Takeda will be responsible for clinical development,…

When the TDP-43 protein is less able to form two-protein complexes called dimers, it appears to be more likely to form the toxic clumps seen in amyotrophic lateral sclerosis (ALS), a study reports. As these clumps are found in about 97% of all ALS cases, a better understanding of the…

AC Immune has been awarded more than $500,000 in nonprofit grants by the Michael J. Fox Foundation (MJFF) and the Target ALS Foundation to support its programs to detect abnormal forms of the TDP-43 protein via imaging scans and fluid samples. “It is an honor to have…

Amydis is developing a test to monitor the buildup of TDP-43 in the eye’s retina of people with amyotrophic lateral sclerosis (ALS), which could offer a noninvasive way to diagnose the neurodegenerative disease sooner and better monitor its progression. The research will be conducted with support from a…

A protein called NOVA1 may be involved in the early disruption of protein production in amyotrophic lateral sclerosis (ALS), even before the characteristic buildup of the toxic TDP-43 protein occurs. The finding is a step toward identifying ways to treat the disease in its earlier stages, before nerve cells…

TDP-43 protein abnormalities characteristic of most amyotrophic lateral sclerosis (ALS) cases contribute to the loss of motor neurons mostly by limiting the function of a protein called stathmin-2 (STMN2), a study reported. Results indicate that boosting STMN2 levels may be a useful approach in treating ALS, according to its…

Stealth BioTherapeutics’ investigational therapy SBT-272 eased inflammation and upper motor neuron degeneration in a mouse model of amyotrophic lateral sclerosis (ALS) associated with the accumulation of toxic TDP-43 protein clumps, the company announced. Motor neurons are nerve cells that control voluntary movement and progressively die in people with ALS.