Ernexa Therapeutics Inc. is advancing its lead program, ERNA-101, toward an IND submission in the third quarter of this year and the initiation of a first-in-human trial in the fourth quarter for the treatment of platinum-resistant ovarian cancer.
Phase II data disclosed March 31 by Pepgen Inc. in myotonic dystrophy type 1 (DM1) hobbled the stock but might have been much different if not for one outlier in the 5-mg/kg multiple ascending dose cohort of the ongoing phase II Freedom2-DM1 trial – and Wall Street is pondering what the hitch means for the Boston-based firm as well as the competitive space.
Researchers from the China Pharmaceutical University and Guangdong Pharmaceutical University (China) have unveiled the crucial role of the alternative splicing of E2A in myogenic progression and demonstrated that PTBP1, by controlling E2A alternative splicing, is a critical regulator of myogenesis.
Qyuns Therapeutics Co. Ltd. has moved closer to its first commercial product after China’s National Medical Products Administration (NMPA) accepted its NDA for IL-17 antibody crusekitug (QX-002N) for treating ankylosing spondylitis (AS), a chronic inflammatory disease that affects the spine and sacroiliac joints.
Facioscapulohumeral muscular dystrophy (FSHD) is a muscle wasting disease caused by aberrant expression of double homeobox protein 4 (DUX4). When DUX4 is activated in skeletal muscle, it triggers myocyte cell death after several transcriptional changes, thus genetic DUX4 silencing arises as a promising approach for treating FHSD.
Korea Research Institute of Chemical Technology has prepared and tested new serine/threonine-protein kinase LATS1 and LATS2 inhibitors reported to be useful for the treatment of sarcopenia, muscular dystrophy, inflammatory myopathy, congenital myopathy, myotonia, myofascial pain syndrome, motor neuron diseases and muscle injury.
With top-line pivotal data with gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) due in the next quarter, Regenxbio Inc. rolled out positive interim data from the phase I/II Affinity trial at the Muscular Dystrophy Association Clinical and Scientific Conference (MDA) in Orlando, Fla., where Bridgebio Pharma Inc., Capricor Therapeutics Inc., and Solid Biosciences Inc. also had clinical findings to talk about.
Qyuns Therapeutics Co. Ltd. has moved closer to its first commercial product after China’s National Medical Products Administration (NMPA) accepted its NDA for IL-17 antibody crusekitug (QX-002N) for treating ankylosing spondylitis (AS), a chronic inflammatory disease that affects the spine and sacroiliac joints.
The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal to consider PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation disease.
Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating Parkinson’s disease.
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