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Stem cell therapy shows promise in advanced MS

Drug Discovery World

An international team of scientists has shown that a stem cell therapy for multiple sclerosis (MS) could prevent further damage to the brain. The stem cells are thought to reduce the inflammation that drives the disease. The post Stem cell therapy shows promise in advanced MS appeared first on Drug Discovery World (DDW).

Therapies 265
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Non-viral gene therapy shows early efficacy in lung cancer

Drug Discovery World

Positive Phase I trial data indicates that Reqorsa (quaratusugene ozeplasmid) has shown early efficacy for the treatment of non-small cell lung cancer (NSCLC). The data will be presented at the 2023 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Therapies 278
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Treatment potential for immunotherapy-induced cytokine release syndrome  

Drug Discovery World

Poolbeg Pharma, a biopharmaceutical company focused on the development and commercialisation of medicines targeting diseases with a high unmet medical need, has announced promising in vivo results for POLB 001 in addressing cancer immunotherapy-induced cytokine release syndrome (CRS). million cases of cancer by 2030 1,2.

Treatment 163
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Research reveals therapeutic potential of mRNA in rare diseases

Drug Discovery World

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

Disease 148
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How ready are we for radioligand therapies?  

Drug Discovery World

DDW Editor Reece Armstrong speaks to Jasminka Taleska , Director, RLT HCS Readiness and Policy at Novartis, about the opportunities radioligand therapies (RLTs) represent in treating cancer patients. Its unique mechanism of action aims to deliver treatment directly to cancer cells, minimising damage to nearby healthy cells 2,5.

Therapies 148
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Actigen moves closer to clinical trial for rare disease treatment  

Drug Discovery World

Biotech company Actigen has moved closer to clinical trials for rare disease treatment for Hunter syndrome, following a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA). ? . Official comments .

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First therapy for very rare blood clotting disorder gets US greenlight

Drug Discovery World

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). For prophylactic ERT, Adyznma is administered to help reduce the risk of disease symptoms.

Therapies 130