Quralis Inc. raised $88 million in series B round to fund clinical development of its two lead programs in amyotrophic lateral sclerosis (ALS) and to take forward earlier-stage pipeline projects in ALS and frontotemporal dementia.
Treatment with a cell-penetrating peptide that prevented nuclear export of unprocessed C9ORF72 RNA and its subsequent translation into neurotoxic dipeptide repeat proteins reduced motor neuron damage and death both in fruit fly models of amyotrophic lateral sclerosis (ALS), and in patient-derived induced neuronal precursor cells (iNPCs). The work suggests that targeting nuclear export could be a therapeutic option in ALS, and possibly also frontotemporal dementia (FTD), where C9ORF72 mutations also play a role.
Fresh off an end-of-year IPO, Coya Therapeutics Inc. is gearing up for clinical testing with its lead Treg-enhancing biologic in neurodegenerative disease, aiming to build on a wealth of academic-generated data highlighting the potential of Treg therapy to attack the neuroinflammation underlying diseases such as amyotrophic lateral sclerosis and Alzheimer’s disease.
Amyotrophic lateral sclerosis (ALS) is the most frequent adult-onset motor neuron disease, and it is pathologically related with frontotemporal dementia (FTD). Genetic studies have identified C9ORF72 as a major genetic cause of ALS/FTD. Further genetic analyses and validation studies have identified some other genes associated with ALS risk, highlighting among them the NUP50 gene, which encodes nuclear pore complex protein Nup50.
Vigil Neuroscience Inc. has synthesized triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of frontotemporal dementia, multiple sclerosis, rheumatoid arthritis, stroke, prion infections, Parkinson's, Alzheimer's and Nasu-Hakola diseases.
Bit Bio Ltd. has announced the addition to its portfolio of ioGlutamatergic Neurons MAPT N279K and ioGlutamatergic Neurons MAPT P301S disease models for frontotemporal dementia (FTD), and early access to its ioGABAergic Neurons for neurological diseases, including epilepsy, schizophrenia, autism and Alzheimer's disease.
Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are two devastating neurodegenerative diseases, with clinical, pathological and genetic overlap, without effective therapy.
Biohaven Therapeutics Ltd. has divulged compounds targeting TAR DNA-binding protein 43 (TDP43) reported to be useful for the treatment of amyotrophic lateral sclerosis, traumatic encephalopathy, sporadic inclusion body myositis, frontotemporal dementia and Alzheimer's disease.
Cumulus Neuroscience Ltd. published a study demonstrating its home use electroencephalogram (EEG) can turn in more precise results than supervised laboratory-based assessments.