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.
All patients dosed in a phase IIa trial with Tryptamine Therapeutics Ltd.’s (Tryp) oral psilocybin (TRP-8802) trial reported an improvement in fibromyalgia pain severity, sleep, and pain interference.
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.
The U.S. FDA recently granted Medtronic plc approval for its deep brain stimulation (DBS) system to be used to treat Parkinson’s disease or essential tremor while a patient is asleep, under general anesthesia. The approval gives patients another option for DBS therapy which can transform their quality of life, Amaza Reitmeier, vice president and general manager for Medtronic brain modulation, told BioWorld in an interview.
A new brain-computer interface (BCI) developed at UC Davis Health is able to translate brain signals into speech with up to 97% accuracy – the most accurate system of its kind.
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.
RSS
