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BioWorld - Wednesday, December 31, 2025
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Neurology/psychiatric

Satellos files in Australia to begin clinical testing of SAT-3247 for Duchenne muscular dystrophy

July 12, 2024
Satellos Bioscience Inc. has submitted a clinical research proposal to a Human Research Ethics Committee (HREC) in Australia seeking regulatory authorization under the Therapeutic Goods Administration (TGA)’s clinical trial notification scheme to conduct a first-in-human phase I trial of SAT-3247.
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Ocular

Beacon Therapeutics to evaluate Abeona’s AAV204 capsid for ophthalmology indications

July 12, 2024
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.
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Illustration of how Naloxone works at the cellular level
Substance use & poisoning

Compound enhances naloxone’s effect in reversing opioid overdose

July 12, 2024
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.
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Heart scientific overlay
Cardiovascular

Sirnaomics completes IND-enabling studies with STP-125G for cardiovascular diseases

July 12, 2024
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.
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Neurology/psychiatric

Novel MAGL inhibitor exerts neuroprotection in multiple sclerosis models

July 12, 2024
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.
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Stem cells
Neurology/psychiatric

ISSCR 2024: Expanding niche definition gives insights into stem cells

July 12, 2024
By Anette Breindl
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.
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Other news to note for July 11, 2024

July 11, 2024
Additional early-stage research and drug discovery news in brief, from: Lisata Therapeutics, Ovid Therapeutics, Recce Pharmaceuticals, Virpax Pharmaceuticals.
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Endocrine/metabolic

Gasherbrum Bio patent details new GLP-1 receptor agonists

July 11, 2024
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.
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Cancer

New PROTACs reported in Hangzhou Zhongmei Huadong Pharmaceutical patent

July 11, 2024
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.
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Neurology/psychiatric

Sironax identifies SARM1 modulators for axonal degeneration

July 11, 2024
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.
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