Dravet syndrome is a rare, severe, lifelong developmental and epileptic encephalopathy that begins in infancy and is marked by prolonged, often fever-triggered seizures that are difficult to control. It is usually caused by mutations in the SCN1A gene and is associated with developmental delay, cognitive and behavioral impairment, and reduced life expectancy.
BLR Bio LLC has presented new data highlighting the potential of BLR-200 for the treatment of systemic sclerosis (scleroderma). The company also presented data on its research on CCN proteins as potential noninvasive biomarkers for systemic sclerosis.
Latus Bio Inc. has closed a $97 million series A financing to support its broad therapeutics pipeline based on novel AAV capsid variants. Proceeds from the financing are expected to fund operations through milestones that include initial clinical data from the company’s two most advanced programs: LTS-201 for Huntington’s disease and LTS-101 for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
A new vaccination strategy designed to induce antibodies that recognize the apex of the HIV Env protein uses Env trimers displayed on liposomes to increase their density and orient them correctly. This presentation enhanced apex-focused antibody responses in macaques, and the monoclonal antibodies isolated after immunization showed binding modes and structural features resembling human broadly neutralizing antibodies (bNAbs), indicating that the vaccine can steer the antibody response toward this vulnerable site.
Beijing Innocare Pharma Tech Co. Ltd. has synthesized new antibody-drug conjugates comprising antibody or antigen-binding fragment covalently linked to eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1) degradation inducer potentially useful for the treatment of cancer.
Ono Pharmaceutical Co. Ltd. has synthesized bridged bicyclic compounds acting as endosomal/lysosomal proton channel TMEM175 activators. As such, they are described as potentially useful for the treatment of inflammatory disorders, lysosomal storage diseases, autoimmune disease and Parkinson’s disease.
University of Western Australia has identified new lysergic acid diethylamide (LSD) analogues acting as 5-HT2A and 5-HT2B modulators reported to be useful for the treatment of neurological and psychiatric disorders.
Work at Pheon Therapeutics Ltd. has led to the development of new antibody-drug conjugates comprising an antibody or antigen binding fragment targeting CUB domain-containing protein 1 (CDCP1) covalently linked to cytotoxic drug. They are intended for use in the treatment of cancer.
Alceptor Therapeutics Inc. has patented new pyrazol-5-amine α2B-adrenoceptor agonists reported to be useful for the treatment of pain and hypertension.
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