Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full length dystrophin in Becker muscular dystrophy.
In a recently published article, researchers from the University of Central Florida and collaborators reported developing a novel multiorgan microphysiological system (MPS) to simulate acute opioid overdose and recovery, addressing key challenges in opioid crisis research.
Scientists at Universidade Federal Rural do Rio de Janeiro (UFRRJ) have reported a novel multitarget-directed ligand (MTDL) targeting butyrylcholinesterase (BChE) with potential for development as an antioxidant and metal-chelating agent for the treatment of Alzheimer’s disease.
Rappta Therapeutics Oy has signed an exclusive global license agreement with Springworks Therapeutics Inc. for RPT-04402, a first-in-class molecular glue of specific protein phosphatase 2A (PP2A) complexes. Springworks will be responsible for global development and commercialization of RPT-04402, now named SW-3431, and expects to file an IND application by year-end.
Pulsesight Therapeutics SAS has submitted a clinical trial authorization (CTA) to the French authority ANSM seeking to conduct a first-in-human phase I trial of PST-611 for dry age-related macular degeneration (AMD)/geographic atrophy (GA).
Major depressive disorder (MDD) is a leading cause of disability worldwide and unfortunately no treatment without side effects is available. Previous reports have found unbalanced glutamate-glutamine cycle in the medial prefrontal cortex of mice with depression, which led to the interest in glutamine synthetase as a potential therapeutic target for treating MDD, since this enzyme is a regulator of this cycle.
An experimental tuberculosis (TB) vaccine with a dual mission – self-destruction after inducing immunity – improved the design of the Bacillus Calmette-Guérin (BCG) immunotherapy, a vaccine also used against cancer. Scientists at the University of Pittsburgh engineered this strain with a double break, which is effective and safer after an intravenous administration, according to their results in nonhuman primates and mice.
Fourteen global pharma companies are getting together to conduct the largest proteomics study to date, analyzing 600,000 blood samples held in the UK Biobank to assess the levels of 5,400 different proteins.
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