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.
Cerevance Inc. has expanded its series B financing with an additional close of US$51 million, bringing the total series B financing to US$116 million. The proceeds will be used to advance Cerevance's therapeutics for central nervous system (CNS) diseases developed using its proprietary Nuclear Enriched Transcript Sort sequencing (Netsseq) platform.
Updated MRI results from Clene Inc.’s phase II Visionary-MS trial testing gold nanocrystal therapy CNM-Au8 bolsters the company’s premise that targeting energy metabolism could protect neuronal function in patients with multiple sclerosis and lays the groundwork for a phase III trial as soon as a strategic partner is found.
The suppression of the SYF2 factor could be a new therapeutic strategy for the treatment of the different types of amyotrophic lateral sclerosis (ALS). According to a study from the University of Southern California, SYF2 acts on the TDP-43 protein, improving the survival of motor neurons affected by this disease. “We wanted to find something that would improve neuron survival across many different iPSC lines for ALS,” Justin Ichida told BioWorld.
Biogen Inc. and C4 Therapeutics Inc. have patented proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety coupled to interleukin-1 receptor-associated kinase 4 (IRAK-4)-targeting moiety through a linker acting as IRAK-4 degradation inducers reported to be useful for the treatment of cancer, inflammation, autoimmune and metabolic diseases, Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis and multiple sclerosis, among others.
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.
Researchers at the Dana-Farber Cancer Institute have been able to identify proteins that were released from muscles during exercise in relatively small quantities. Using their method, the team was able to demonstrate that the neurotrophic factor prosaposin was produced during exercise. Prosaposin is “a well-known CNS neurotrophic factor, but has never been seen to come out of muscle or fat,” Bruce Spiegelman told BioWorld. Spiegelman is a researcher at the Dana-Farber Cancer Institute and Stanley J. Korsmeyer Professor of Cell Biology and Medicine at Harvard Medical School.