Abeona Therapeutics Inc. and Beacon Therapeutics Ltd. have signed an agreement for evaluation by Beacon of Abeona’s patented AAV204 capsid for the development and commercialization of potential gene therapies for select ophthalmology indications.
Scientists at Stanford University School of Medicine have discovered a new compound that increases the effects of naloxone, a drug that is used to treat overdoses from opioids like fentanyl, a narcotic that has triggered a public health crisis.
Sirnaomics Ltd. has completed IND-enabling studies for STP-125G, a single-stranded siRNA therapeutic targeting apolipoprotein C3 (ApoC3), based on its proprietary Galahead mxRNA technology.
Targeting the endocannabinoid system and, specifically, inhibiting the degradation of endocannabinoid 2-arachidonoylglycerol (2-AG) has demonstrated a neuroprotective effect in multiple sclerosis (MS). In this sense, monoacylglycerol lipase (MAGL), the enzyme that regulates 2-AG in the brain, has been proposed as a therapeutic target for MS.
The word “niche” implies a specialized environment. But to Fiona Doetsch, the stem cell niche is anything but. For brain stem cells, “the whole organism is the niche,” Doetsch told the audience at the third plenary session of the International Society for Stem Cell Research (ISSCR) annual meeting in Hamburg this week. It’s a surprising idea at first, given the brain’s protection from many circulating substances via a series of barriers, including the blood-brain barrier and the blood-cerebrospinal fluid barrier.
Additional early-stage research and drug discovery news in brief, from: Lisata Therapeutics, Ovid Therapeutics, Recce Pharmaceuticals, Virpax Pharmaceuticals.
Gasherbrum Bio Inc. has disclosed GLP-1 receptor agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, hyperglycemia, hypertension, obesity, stroke, myocardial infarction and gout, among others.
Hangzhou Zhongmei Huadong Pharmaceutical Co. Ltd. has described proteolysis targeting chimera (PROTACs) compounds comprising an E3 ubiquitin ligase-binding moiety covalently linked to a mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1)-targeting moiety. They are reported to be useful for the treatment of cancer.
Sironax Ltd. has discovered (+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, diabetic neuropathy, multiple sclerosis, Parkinson's disease, chemotherapy-induced peripheral neuropathy and traumatic brain injury.
Haisco Pharmaceutical Group Co. Ltd. has identified phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors reported to be useful for the treatment of breast cancer.