相关临床试验
2
1 进行中
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0
批准总数
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0
监管机构数
成立时间
1877
尚未招募
1
50.0%
招募中
1
50.0%
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- Japan has approved a new indication for Buphenyl (sodium phenylbutyrate) to treat progressive familial intrahepatic cholestasis types 1 and 2, rare genetic liver disorders. - The drug increases bile salt export pump proteins at the liver cell surface, helping move bile acids into bile and reducing toxic buildup in liver cells. - An investigator-initiated trial in six children with PFIC type 2 showed the treatment could slow liver damage caused by the disease. - Researchers established the CIRCLe registry and biorepository to address the lack of disease information that had hindered pharmaceutical company investment in PFIC drug development.
- Andelyn Biosciences has partnered with Amplo Biotechnology to provide scalable manufacturing of clinical-grade AAV material using Andelyn's suspension AAV Curator® platform for neuromuscular junction disorders. - The collaboration aims to advance Amplo's AAV gene therapy programs toward clinical evaluation, with a focus on safety and efficacy for upcoming trials targeting genetic NMJ diseases. - Neuromuscular junction disorders typically result in muscle weakness and can be life-threatening, with genetic forms usually diagnosed in early childhood but potentially manifesting in adolescence or adulthood. - Amplo's lead program, AMP-101, is advancing toward a first-in-human trial in Dok7 congenital myasthenic syndrome, built on research from the University of Tokyo and Oxford.
- University of Tokyo researchers have developed a novel nasal COVID vaccine that demonstrated high immunity in animal tests, offering broader lung coverage compared to mRNA vaccines. - The vaccine uses engineered viruses with removed viral assembly genes, creating immunity proteins without multiplication risk while preventing virus growth in nasal cavities and lungs. - In hamster studies, the nasal vaccine prevented delta and omicron XBB variant propagation in lungs, with half showing no nasal viral growth by day three post-infection. - The research team claims this approach could prevent infection spread by inhibiting viral growth in the nose, providing immunity similar to natural infection unlike current mRNA vaccines.
