Several recent Janssen Pharmaceutica NV patents describe new NLRP3 inflammasome inhibitors reported to be useful for the treatment of traumatic brain injury, multiple sclerosis, amyotrophic lateral sclerosis, dementia, Huntington’s, Parkinson’s, Alzheimer’s and motor neuron diseases.
Purdue Research Foundation has discovered compounds for inhibiting protein aggregation potentially useful for the treatment of wild-type transthyretin amyloidosis, type 2 diabetes, amyotrophic lateral sclerosis and more.
People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases.
Daiichi Sankyo Co. Ltd. has divulged compounds acting as NLRP3 inflammasome inhibitors reported to be useful for the treatment of inflammation, major depression, multiple sclerosis, multiple system atrophy, schizophrenia, autoimmune disease, Alzheimer’s disease and Parkinson’s disease, among others.
Disarm Therapeutics Inc. has identified NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, multiple sclerosis, diabetic neuropathy and chemotherapy-induced peripheral neuropathy.
IGC Pharma Inc. has reported preclinical research demonstrating the therapeutic potential of IGC-1C, a cyclic dipeptide-based small-molecule modulator, in neurodegenerative disease due to its modulation of tau protein, which is involved in the formation of neurofibrillary tangles, a key hallmark of Alzheimer’s disease.
Ractigen Therapeutics Co. Ltd.’s small activating RNA (saRNA) therapeutic, RAG-18, has been awarded U.S. orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy.
Scientists at Harvard Medical School have shown that in mice lacking amyloid beta (Aβ), the fundamental hallmark of Alzheimer's disease (AD), neurons died from the effect of the most harmful mutation of this neurodegenerative disease. They showed that presenilin (PS) could be behind the origin of the disease without the need for Aβ. They maintain that it is time to update theories and redirect efforts.
Immpact Bio USA Inc. has obtained IND clearance from the FDA for IMPT-514, a CD19/CD20 bispecific chimeric antigen receptor (CAR) T-cell therapy for the treatment of adult patients with multiple sclerosis (MS). A phase I trial will focus on patients who have suboptimal disease control despite prior treatment with high efficacy disease-modifying therapies in all forms of MS.
After sparking further interest from investors after the close of its series A, Vandria SA has extended the round and now has the means to advance its lead mitophagy inducer program as far as phase Ib/IIa development in the treatment of mild cognitive impairment.