Capsida Biotherapeutics Inc. has obtained IND clearance from the FDA for CAP-003, an intravenously administered gene therapy, for Parkinson’s disease associated with GBA mutations (PD-GBA). A phase I/II trial will begin dosing in the third quarter of this year.
Octant Inc. has received a $4.9 million grant from the Gates Foundation to support a new therapeutics program focused on human papillomavirus (HPV)-related cancers. The initiative will develop affordable, broad-spectrum, small-molecule antiviral drugs designed to disrupt the interaction between hundreds of variants of the HPV E6 protein and the tumor suppressor protein p53.
Epilepsy affects more than 70 million worldwide and while several drugs have proven effective for controlling seizures, around 30% of patients do not respond to them and their side effects can be intolerable.
Regulatory T cells (Tregs) maintain immune homeostasis by inhibiting excessive immune responses. Dysregulation of Tregs, characterized by reduced cell numbers or impaired suppressive function, is implicated in the pathogenesis of autoimmune diseases. Low-dose interleukin-2 (IL-2) therapy expands Tregs and enhances their suppressive capacity while minimizing the activation of T cells and natural killer (NK) cells.
4M Therapeutics Inc. has released promising results from completed GLP safety studies for its lead asset, 4MT-2001, supporting its potential to offer a safer treatment option to patients who are underserved by lithium and atypical antipsychotics.
The ionic and metabolic impairment observed in chronic kidney disease (CKD) leads to vascular calcification, which can induce cardiovascular events and mortality. Several factors may impact the progression of vascular calcification, where inorganic pyrophosphate plays a crucial inhibitory role.
Researchers at the Buck Institute for Research on Aging have developed an epigenetic clock that could predict an individual’s intrinsic capacity score, a composite score that is a measure of healthy aging. The clock, which has only limited overlap with other epigenetic clocks, could be “a surrogate for aging that can be used for clinical trials,” senior author David Furman told BioWorld. And even more basically, it could help address a basic conundrum: that aging is the major risk factor for most causes of death, but not itself a disease.
Is fecal microbiota transplant effective? Is it really safe? And is it really all the same? Scientists at the University of Chicago have investigated the regional differences in gut environments to question these interventions to analyze the microbiome differences and their effects after transplantation form different intestine areas. The results show how host-microbe mismatches after these interventions could affect gut health.
Work at Sensorium Therapeutics Inc. has led to the identification of new serotonin transporter (SERT) inhibitors reported to be useful for the treatment of depression, anxiety and stress disorders.
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