Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the treatment of optic neuropathy in familial dysautonomia and BPN-36964 for systemic treatment of familial dysautonomia.
Cardiopulmonary resuscitation (CPR) after cardiac arrest (CA) is often a cause of secondary neurological impairment, which results in considerable morbidity and mortality. Suppression of protein degradation of key blood-brain barrier (BBB) components after CPR could maintain the stability of the BBB function, and as such minimize secondary neurological damage and improve long-term prognosis after ischemia reperfusion injury.
Icelandic genomics company Arctic Therapeutics has closed a €26.5 million (US$27.6 million) series A, enabling it to assess if its lead drug AT-001, designed to treat a rare inherited amyloid disease, also could be used to treat more common forms of dementia, including Alzheimer’s disease. The program is currently in a phase IIb/III European trial in cystatin C amyloid angiopathy, an ultra-rare disease found only in Iceland that is caused by the L68Q mutation in the cystatin C gene.
Tris Pharma Inc.’s positive top-line data from a pivotal phase III study of cebranopadol in pain relief has the company thinking about an NDA submission later in the year. The company’s goal is to avoid the problems opioids have brought to treating pain.
Standing for live visualization of brain circuits, LVIS Corp launched the cloud-based Neuromatch Access platform as a software as a service to help neurologists and medical professionals review, interpret and analyze electroencephalogram (EEG) data.
The activation of the adenosine A2A receptor, which is mediated by the inhibition of adenosine A1 receptor, has been associated with depression-like behavior and anhedonia. High levels of cortisol, increased oxidative stress and antioxidant enzyme reduction are also contributors to the pathophysiology of depression.
A series of disappointments that drove Sage Therapeutics Inc.’s stock down by 85% since August of 2023 has evolved into an unsolicited takeout offer by partner Biogen Inc., followed by a lawsuit filed by Sage a week later. The two parties, both of Cambridge, Mass., first partnered in a $1.52 billion deal in 2020, primarily to develop Zurzuvae (zuranolone) for depression. The deal included Biogen taking a 10.2% equity stake in Sage, paying $104.14 per share, or $650 million total.
The largest analysis to date of patients taking GLP-1 receptor agonists (GLP-1RAs) has investigated their effects on nearly 175 diseases, and found that compared to three other classes of diabetes medications, individuals with a prescription for GLP-1RAs had a reduced risk of 42 diseases, and an increased risk of 19.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have divulged triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of amyotrophic lateral sclerosis, Alzheimer’s disease, frontotemporal dementia, multiple sclerosis, Nasu-Hakola disease, Parkinson’s disease, rheumatoid arthritis and stroke.
Northeastern University has disclosed cannabinoid CB1 receptor allosteric modulators and monoglyceride lipase (MGLL; MAGL) inhibitors reported to be useful for the treatment of pain, inflammation, anxiety, psychosis, traumatic brain injury, post-traumatic stress disorder, epilepsy and neurodegenerative diseases.
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