Myrobalan Therapeutics Nanjing Co. Ltd. has divulged uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-like) antagonists reported to be useful for the treatment of multiple sclerosis, amyotrophic lateral sclerosis, Alzheimer’s disease and Parkinson’s disease.
Sichuan Kelun Pharmaceutical Research Institute Co. Ltd. and Suzhou Kelun Pharmaceutical Co. Ltd. have identified carboxylic acid ester compounds reported to be useful for anesthesia.
Parkinson’s disease (PD) is a progressive disease characterized by loss of dopaminergic neurons in the substantia nigra, which lead to motor symptoms. Aromatic-L-amino-acid decarboxylase (AADC) converts levodopa into dopamine and glial cell line-derived neurotrophic factor (GDNF) promotes the survival, growth and regeneration of dopaminergic neurons. VGN-R09b is an adeno-associated viral vector (AAV)-based AADC and GDNF combination gene therapy that is delivered to the striatum for the treatment of PD.
Casma Therapeutics Inc. has developed bicyclic heteroaromatic compounds acting as mucolipin-1 (MCOLN1; TRPML1) activators reported to be useful for the treatment of Duchenne muscular dystrophy, neurodegeneration, atherosclerosis, cancer, infections, liver, lysosomal storage and inflammatory bowel diseases.
Oligomeric forms of α-synuclein are increasingly recognized as the primary neurotoxic species in Parkinson’s disease (PD) and other synucleinopathies, contributing to synaptic dysfunction, mitochondrial impairment and the prion-like propagation of pathology. Targeting these early aggregates represents a promising strategy for disease modification.
The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments.
Cala Health Inc.’s KIQ wearable device delivers transcutaneous afferent patterned stimulation through nerves at the wrist that modulate neural activity in the brain and reduces hand tremors in individuals with essential tremor, two recent studies show.
In a deal that could top $2 billion, China-based Innocare Pharma Ltd. licensed the exclusive worldwide development and commercialization rights to the BTK inhibitor orelabrutinib to Zenas Biopharma Inc. for multiple sclerosis and other indications aside from oncology.
Northern Illinois University and Stream Neuroscience Inc. have patented benzothiazole derivatives acting as phosphodiesterase‑4 (PDE4) inhibitors and reported to be useful for the treatment of cancer, cognitive, inflammatory and mood disorders.