Precision Biosciences Inc. has received FDA clearance of its IND application enabling clinical trial site activation for a phase I/II trial (FUNCTION-DMD) of PBGENE-DMD for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD). The first site in the U.S. will be activated in the first half of this year.
TYK2, a Janus kinase family member, mediates signaling from pro-inflammatory cytokines such as type I interferons, IL-12 and IL-23. In multiple sclerosis (MS), chronic CNS inflammation involves glial activation and immune cell infiltration, making TYK2 inhibition a potential strategy to reduce neuroinflammation.
Tempest Therapeutics Inc. has outlined plans to advance its newly acquired CAR T assets. While prioritizing development of its clinical-stage dual-targeting CD19/BCMA CAR-T program, TPST-2003 (ERI-2003), the company will also expand its portfolio into next-generation modalities.
Madrigal Pharmaceuticals Inc. has signed an exclusive global license agreement with Suzhou Ribo Life Science Co. Ltd. and its subsidiary Ribocure Pharmaceuticals AB for six preclinical small interfering RNA (siRNA) programs.
Henipaviruses like Nipah (NiV) and Hendra (HeV) are highly lethal, bat-borne zoonotic viruses from the Paramyxoviridae family that cause severe encephalitis and respiratory illness in humans and animals. These viruses are highly transmissible and have notable pandemic potential. Researchers are actively screening and testing numerous small-molecule compounds as potential treatments for henipavirus infections. A team at the University of Illinois Chicago and collaborators used a high-throughput screening approach to identify a focused library of analogues.
Galux closed a ₩42 billion (US$29 million) series B round Feb. 10, led by Yuanta Investment to bring AI-driven “rational design” to the protein drug development process, already heavily influenced by human engineering.
China Pharmaceutical University and Chongqing Innovation Institute China Pharmaceutical University have reported tetrahydronaphthyridine compounds acting as stimulator of interferon genes protein (STING; TMEM173) antagonists.
Fatty acid lipoxygenase ALOX15 inhibitors have been disclosed in a joint Teijin Pharma Ltd. and Transthera Biosciences Co. Ltd. patent. They are reported to be useful for the treatment of amyotrophic lateral sclerosis, asthma, autoimmune disease, cancer, cardiovascular disorders, epilepsy, Parkinson’s disease and allergic rhinitis, among others.
Impact Therapeutics Inc. has divulged heteroaryl compounds acting as ATP-dependent RNA helicase A (DHX9) inhibitors intended for use in the treatment of cancer.
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