Mutations that activate phosphorylation of certain Rab GTPases by LRRK2 have been linked to a subset of genetic cases of Parkinson’s disease, and PPM1M is one of the phosphatases responsible for reversing phosphorylation by LRRK2.
Researchers at Sanofi SA have developed a promising gene therapy approach targeting the microtubule-associated protein tau (MAPT) for the treatment of Alzheimer’s disease.
Elutia Inc. agreed to sell its Elupro and Cangaroo bioenvelopes for implantable medical devices to Boston Scientific Corp. for $88 million in cash. Elutia will use the funds to further development of NXT-41x, an antibiotic biomatrix designed to reduce post-surgical complications in breast reconstruction.
Argenica Therapeutics Ltd. saw its stock drop 56% following mixed results for its ARG-007 phase II Seancon trial in acute ischemic stroke patients undergoing endovascular revascularization, or thrombectomy.
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
Myotonic dystrophy type 1 (DM1) is a rare, progressive genetic disease that causes severe muscle weakness and other debilitating symptoms, such as compromised respiration and cardiac conduction abnormalities. No disease-modifying therapy exists for DM1, so care focuses on managing symptoms like arrhythmia, myotonia, hypertension, cataracts, respiratory issues and sleep disorders.
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
Medicxi Ventures’ asset-focused investment model has scored another hit, in a $450 million deal with Servier SA, for KER-0193, an oral small molecule that is ready for phase II development in the treatment of the inherited autism spectrum disorder, fragile X.
Robust efficacy, competitive tolerability and ease of administration. Those are the qualities for a potential blockbuster antiseizure medication, according to Abe Ceesay, CEO of Rapport Therapeutics Inc., which reported a successful phase IIa trial testing RAP-219 in patients with drug-resistant focal onset seizures and aims to move into a large-scale phase III program in 2026.
Otsuka Pharmaceutical Co. Ltd. has identified new piperidine- and morpholine-carboxylate compounds acting as sortilin (SORT; NT3; Gp95) antagonists. As such, they are described as potentially useful for the treatment of amyotrophic lateral sclerosis, frontotemporal dementia, Alzheimer’s and Parkinson’s disease.
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