Axovant Gene Therapies (NASDAQ: AXGT) announced the grant of an Orphan Drug Designation for AXO-AAV-GM1, the Company’s investigational gene therapy for the treatment of GM1 gangliosidosis. AXO-AAV-GM1 is capable of restoring the β-galactosidase enzyme activity by delivering a functional copy of the dysfunctional GLB1 gene,...
CV Sciences (OTCQB: CVSI), the manufacturer of PLUSCBD range of hemp-based CBD oils, the #1 selling hemp CBD supplement in North America, announced the signing of a new distribution agreement with Southeastern Grocers, Inc. The agreement will enable the Company to launch its PlusCBD oil...
Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig’s disease is a progressive neurodegenerative disease, characterized by the atrophy of nerve cells in brain and spinal cord, leading to hardening and scarring in the affected region. As the disease progresses, patients gradually lose the functioning of muscles,...
Pompe disease is characterized by the build-up of complex sugars named glycogens in the body’s cell, due to the lack of an enzyme called acid alfa glucosidase (GAA), which is responsible for breaking down the sugars. This results in the breakdown of organs, muscles and...
Sandhoff Disease also referred to as GM2-gangliosidosis Type ll is an autosomal recessive genetic lipid storage disorder that is caused by an abnormal gene for the beta subunit of the hexosaminidase B enzyme. The dysfunction of this gene causes accumulation of lipids called GM2 gangliosides in the...
Neurotrope Inc, (NASDAQ: NTRP) announced a setback, with Bryostatin, its lead candidate for the treatment of Alzheimer’s, failing to meet its clinical endpoints of its mid-stage study. The Topline results from the confirmatory Phase 2 study of Bryostatin-1, in moderate to severe Alzheimer’s, failed to achieve...
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...
Seelos Therapeutics (NASDAQ: SEEL), a clinical-stage biotechnology Company, announced the acceptance of its Investigation New Drug (IND) application for SLS-005 (trehalose) by the FDA. The company is planning to initiate a combined Phase IIb/III, multicenter study to assess the safety, tolerability and efficacy of trehalose...
Niemann–Pick is a rare autosomal recessive inherited disease that is characterized by excessive accumulation of cholesterol and lipids in cells, leading to apoptosis and neural degeneration. The disease affects numerous body parts such as the brain, nerves, liver, spleen, bone marrow and lungs. Those suffering...
The Cannabis plant has an estimated 113 different compounds, out of which THC and CBD are the two main constituents. These affect the receptors in the brain to alter an individual’s physical and mental state. THC is mainly responsible for the psychoactive effects or the...
