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.
A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.
Suzhou Genhouse Bio Co. Ltd. has described histone deacetylase 6 (HDAC6) inhibitors reported to be useful for the treatment of cancer, asthma, Alzheimer’s disease, diabetes, amyotrophic lateral sclerosis, multiple sclerosis, pulmonary fibrosis and psoriasis, among others.
Adlai Nortye Biopharma Co. Ltd. and Adlai Nortye Pte Ltd. have divulged cyclin-dependent kinase (CDK) inhibitors reported to be useful for the treatment of cancer, inflammatory disorders and autoimmune diseases.
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