An experimental gene therapy based on the prime editing technique could become an effective treatment for alternating hemiplegia of childhood, a severe and currently incurable rare disease. David Liu’s lab at the Broad Institute, the inventor of this gene edition methodology, together with scientists from The Jackson Laboratory, successfully reversed the effects of five mutations associated with this disorder in a mouse model.
After Recognify Life Sciences Inc.’s phase IIb failure in treating the cognitive impairment in those with schizophrenia, the indication is no closer to a U.S. FDA approved therapy. The placebo-controlled study of inidascamine missed its primary endpoint to improve cognition, joining a list of companies that have tried and failed to find a successful treatment.
Researchers from Biotiche Drug Discovery Srl and collaborators have discovered that modulating the activity of a specific family of potassium channels, known as Kv3 channels, can have beneficial effects on the progression of amyotrophic lateral sclerosis (ALS) in a mouse model of the disease. The study, published in the journal Acta Neuropathologica Communications, provides new insights into the role of these channels in skeletal muscle function and their potential as a therapeutic target for ALS.
Dewpoint Therapeutics Inc. has described TAR DNA-binding protein 43 (TARDBP; TDP-43) modulators reported to be useful for the treatment of traumatic brain injury, frontotemporal dementia and amyotrophic lateral sclerosis.
Stroke is the third leading cause of disability worldwide, and its incidence is expected to increase as the global population ages. Idebenone can promote recovery after stroke, but it is less effective during the acute phase of stroke.
Congruence Therapeutics Inc. has received a research grant of $5 million from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to advance its GCase-targeting small molecules for GBA1 Parkinson’s disease. Mutations of the GBA1 gene, encoding the enzyme GCase, represent the single largest genetic risk factor for Parkinson’s disease.
Current treatments for Alzheimer’s disease have limited effects. While they can slow cognitive decline or alleviate symptoms, they do not reverse this complex neurodegenerative condition caused by multiple factors. Researchers from the Gladstone Institutes and the University of California, San Francisco (UCSF) have screened FDA-approved drugs in search of agents that could potentially modify the disease.
Roche Holding AG received CE mark for its Elecsys pTau181 test to help rule out Alzheimer’s disease. The minimally invasive blood test, developed in collaboration with Eli Lilly and Co., measures phosphorylated Tau 181 protein, a biomarker indicative of amyloid pathology, a hallmark of Alzheimer’s.
Neurogene Inc.’s disclosure June 30 of its registrational trial design in Rett syndrome (RS) added spice to the space, where Taysha Gene Therapies Inc. is another closely watched player. The New York-based firm said Embolden will test NGN-401, a gene therapy for the treatment of females age 3 and older with RS. U.S. regulators have signed off regarding key aspects of Embolden’s structure.
Researchers from Peking University and the China-Japan Friendship Hospital have gained new insights into the role of triggering receptor expressed on myeloid cells-1 (TREM-1) in traumatic brain injury (TBI). Cerebral damage resulting from external mechanical impacts causes TBI, which is a leading cause of morbidity and mortality worldwide.
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