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.
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.
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.
Alceptor Therapeutics Inc. has patented new pyrazol-5-amine α2B-adrenoceptor agonists reported to be useful for the treatment of pain and hypertension.
Innervation by the sympathetic nervous system is typically a boon to tumors. But researchers from Weill Cornell Medicine and colleagues have shown that in some cases, the relationship between tumors and the nervous system is more complex. Depending on context, innervation can either assist or obstruct tumor growth. “The nervous system typically has been considered as a driver of cancer growth, but here we’ve found that it can be a brake on cancer growth in some contexts,” said David Simon, an assistant professor of biochemistry and biophysics at Weill Cornell Medicine.
Rett syndrome (RTT) is a rare neurodevelopmental condition affecting multiple organ systems and is most often driven by mutations in the X-linked MECP2 gene. Researchers at Shanghai Duomirui Biological Technology Co. Ltd. have developed a new class of trofinetide prodrugs aimed at addressing limitations related to drug administration and pharmacokinetic properties.
Acurastem Inc. has been awarded $7.5 million in grant funding by the California Institute for Regenerative Medicine (CIRM) to support the advancement of lead clinical candidate AS-241 toward first-in-human testing.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have prepared and tested new compounds acting as triggering receptor expressed on myeloid cells 2 (TREM2) agonists. As such, they are believed to be potentially useful for the treatment of rheumatoid arthritis, amyotrophic lateral sclerosis, frontotemporal dementia, multiple sclerosis, prion disease, stroke, Parkinson's and Alzheimer's disease.
In previous work, researchers from Kawasaki Medical School and collaborating institutions engineered a modified HEXB construct, modHexB, to improve GM2 ganglioside (GM2) recognition and GM2-activating protein (GM2A) interaction. The team has now combined these previous advancements to develop a new gene therapy strategy for Sandhoff disease.
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