Somite Therapeutics Inc.’s lead program, SMT-M01, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD). The program leverages the company’s proprietary Alphastem artificial intelligence (AI) platform to develop a novel cell replacement therapy for DMD.
The map of the human brain is not a static image of the cellular architecture of the central nervous system (CNS). Throughout life, all living beings are born, develop and age. The brain of pregnant mothers reflects a dynamic that modifies this general picture. A collaboration of scientists from the University of California has studied the changes in the adult brain during pregnancy and observed that a large part of it adapts during the development of the fetus.
Voyager Therapeutics Inc. has announced the selection of a development candidate in a gene therapy program for the treatment of an undisclosed neurological disease under its collaboration with Neurocrine Biosciences Inc.
By looking at the electrical activity of tumor cells, rather than the neurons that innervate them, investigators at Baylor College of Medicine have added both basic and translational insights to the emerging field of cancer neuroscience. In their studies, which were published in Cancer Cell on Sept. 5, 2024, the researchers identified the cell of origin for IDH-mutated gliomas.
Silo Pharma Inc. has completed a pre-IND meeting with the FDA regarding the company’s development plan for SPC-15, an intranasal prophylactic treatment for post-traumatic stress disorder (PTSD) and stress-induced anxiety disorder.
Stealth Biotherapeutics Inc. has been awarded the Kyle Bryant Translational Research Award from the Friedreich’s Ataxia Research Alliance (FARA) to evaluate mitochondria-targeted molecule SBT-589 in Friedreich’s ataxia (FA).
Neuroinflammation underlies neurodegeneration in Alzheimer’s disease (AD) and other brain disorders. The stimulation of nicotinic α7 receptors, involved in the regulation of neuroinflammation, has shown efficacy in improving memory and learning in AD models.
A novel gene therapy that leads to cellular rejuvenation could restore vision after non-arteritic anterior ischemic optic neuropathy (NAION) and glaucoma. The technique is based on a reprogramming process that reverses the epigenetic DNA alterations caused by aging. Preclinical studies in glaucoma mice and nonhuman primates (NHP) models for this stroke-like disorder that affects the eye, showed an improvement of vision and restoration of the damaged axons of the optic nerve.
Immunotherapy based on T cells is the vanguard of cancer treatments. Researchers from Washington University in St. Louis have shown that similar approaches using T cells could be applied for treating injuries of the central nervous system (CNS). They reported their findings in Nature on Sept. 4, 2024.
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