Aurigene Oncology Ltd. has identified proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase protein binding moiety covalently linked to probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) and/or transcription activator BRG1 (SMARCA4; BAF190A; SNF2-β) binding moieties through a linker reported to be useful for the treatment of cancer.
Essential tremor is a movement disorder marked by involuntary, rhythmic shaking in the hands but sometimes affecting the head, voice and other areas. Its exact cause is still unknown. Recent research suggests that dysfunction in the neurotransmitter systems, particularly involving γ-aminobutyric acid (GABA), contributes significantly to the disorder.
Neurocrine Biosciences Inc. has disclosed vesicular monoamine transporter 2 (VMAT2) inhibitors reported to be useful for the treatment of neurological and psychiatric disorders, among others.
Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating protein that operates postsynaptically. Current therapies can mitigate symptoms but not cure the underlying disease. Researchers at the Allen Institute and collaborators have demonstrated a potential genetic cure for SynGAP1 deficiency.
Septic cardiomyopathy is a common and serious complication of sepsis, affecting up to 60% of patients and markedly worsening survival outcomes. It is characterized by left ventricular systolic dysfunction, but its underlying mechanisms remain poorly understood despite extensive research.
Tr1x Inc. has obtained IND clearance from the FDA for TRX-319, paving the way for initiation of a phase I/IIa study in progressive multiple sclerosis (MS) early next year. TRX-319 is designed to combine targeted B-cell control with active anti-inflammatory signaling and T-cell regulation, with the goal of restoring immune balance.
CXC chemokine receptor 4 (CXCR4) is a receptor overexpressed in several tumor types and associated with tumor aggressiveness and risk of metastasis, resistance and recurrence. A novel PET radiopharmaceutical tracer and CXCR4 ligand, [68Ga]NOTA-R-54, was developed and tested for potential use in the treatment of lung cancer.
Corventum Inc. has gained IND clearance from the FDA for CVT-130 for the prevention of anthracycline-related cardiotoxicity. In cancer patients, the use of anthracyclines as chemotherapy is effective but limited by cumulative heart damage. Protecting against this chemotherapy-induced cardiac injury could enable retreatment for patients with recurrent cancer.
Frontier Medicines Corp. has unveiled FMC-242 as its newest development candidate. FMC-242 is a covalent allosteric inhibitor that breaks the interaction between PI3Kα and RAS proteins to inhibit downstream effector signaling in tumors without impacting normal functions and while minimizing the toxicities commonly associated with the broader class of inhibitors.
Scientists at Indiana University and Purdue Research Foundation have synthesized inositol polyphosphate-5-phosphatase D (SHIP-1; INPP5D) inhibitors reported to be useful for the treatment of mild cognitive impairment, Alzheimer’s disease, vascular cognitive impairment, Lewy body dementia and frontotemporal dementia.
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