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.
The pathogenesis of multiple sclerosis (MS) has been tied to ineffective immune control of Epstein-Barr virus-driven autoimmune responses. Patients with MS are deficient in protective adaptive natural killer cells (pNK cells) in contrast to healthy individuals. These pNK cells are positive for NKG2A, NKG2C and NKG2D and recognize and kill autoreactive B cells in a selective and efficient manner.
Taho Pharmaceuticals Ltd. announced an NDA submission to the U.S. FDA for TAH-3311, developed as the world’s first oral dissolving film formulation of apixaban, marking a regulatory milestone for the Taipei-based biotech.
Lexeo Therapeutics Inc. feels like it’s in a faster lane to a BLA for its Friedreich ataxia cardiomyopathy gene therapy after talking with the U.S. FDA. The agency told Lexeo that LX-2006 could be on the accelerated approval path if there is a mingling of the company’s data and studies.
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