Amyotrophic lateral sclerosis (ALS) is a disorder that leads to progressive muscle weakness and loss of muscle control due to affectation of the motor neurons. Increasing evidence points to defects in the nuclear envelope, which leads to disease progression.
Researchers from Ipsen Ltd. and affiliated organizations presented the discovery and preclinical characterization of a novel NTCP inhibitor, A-7387, being developed for the treatment of HBV and HDV infections.
Researchers from The Brigham and Women's Hospital presented data from a study that aimed to evaluate the role of somatic variants in drug-resistant mesial temporal lobe epilepsy (MTLE). High-coverage whole-exome sequencing was conducted using DNA samples derived from the hippocampus and paired brain tissue and/or blood samples.
Omega Therapeutics Inc. has presented preclinical data on hepatocyte nuclear factor 4-alpha (HNF4A) modulation in fibrotic liver disease models to enhance its key role and suggest it as a potent therapeutic target.
Surmodics Inc. reported positive two-year data from the SWING trial, the first-in-human study to look at the safety of its Sundance Sirolimus drug-coated balloon (DCB) when used to treat occlusive disease of the infrapopliteal arteries. Results presented at the VEITHsymposium in New York showed that the Sundance Sirolimus DCB demonstrated an excellent safety profile and achieved primary patency of 71.4% at 24 months in the per protocol analysis population.
Eli Lilly and Co. shared insights into an inhibitor targeting ANGPTL3, named solbinsiran. Solbinsiran, a N-acetylgalactosamine (GalNAc) conjugated Dicer-substrate small interfering RNA (DsiRNA), is designed to target ANGPTL3 expression in the liver.
Researchers from Kyverna Therapeutics Inc. have presented data on their anti-CD19 CAR T-cell therapy, KYV-101, for the treatment of B-cell-driven autoimmune diseases.
Researchers from Aligos Therapeutics Inc. presented preclinical data for ALG-001075, a novel capsid assembly modulator leading to the formation of empty capsids (CAM-E), being developed for the treatment of hepatitis B.
Researchers from Apeximmune Therapeutics Inc. presented the discovery and preclinical evaluation of AIM-104, a novel potent pan-allele anti-SIRPα antibody for the treatment of cancer.
Bitterroot Bio Inc. presented data on an anti-CD47 hybrid protein, BRB-002, tested in a murine model of atherosclerosis. BRB-002 was tested at 2.5 or 10 mg/kg i.p. 3x/week after a high-fat diet. Firstly, BRB-002 was seen to bind CD47 with high affinity in several cell lines, but not in Jurkat CD47-knockout cells.
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