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World’s first engineered B cell therapy enters human trials

Drug Discovery World

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.

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Breast cancer researchers to be recognised with SABCS award  

Drug Discovery World

The San Antonio Breast Cancer Symposium (SABCS) and the American Association for Cancer Research (AACR), an SABCS cosponsor, will award two researchers for their significant contributions to breast cancer research at SABCS 2023, 5-9 December, at the Henry B González Convention Center in San Antonio, Texas.

Research 147
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Potential disease-modifying therapy for Parkinson’s disease

Drug Discovery World

An experimental drug has shown potential as a disease-modifying therapy for Parkinson’s disease, according to a new study published in Nature Communications. This paper strongly supports further study of USP30 inhibition as a potential disease-modifying therapy for PD.”

Disease 130
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Chinese biotech company consolidates gene therapy portfolio

Drug Discovery World

CANbridge Pharmaceuticals has exercised its option to secure the exclusive global rights to develop, manufacture and commercialise a novel gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School. . The benchmark therapy uses a vector similar to that used in the only gene therapy approved for SMA.

Therapies 130
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Concussion drug therapies on the horizon

Drug Discovery World

Valera, an Associate Professor of Psychiatry at Harvard University and a leading researcher on traumatic brain injuries among survivors of domestic violence, estimated that while hundreds of concussions occur in the NFL each year, there could be nearly 1.6 There are a number of companies currently developing concussion drug therapies.

Therapies 147
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Ancient viruses may hold key to gene therapy treatments

Drug Discovery World

The research focuses on a group of small, ubiquitous viruses called ‘parvoviruses’ (from the Latin word ‘ parvus’ meaning ‘small’, ‘puny’ or ‘unimportant’). It was led by scientists at the MRC-University of Glasgow Centre for Virus Research (CVR) and University of Massachusetts Chan Medical School. .

Virus 130
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FDA-approved therapy targets ESR1 mutations in breast cancer

Drug Discovery World

This therapy targets the ESR1 mutations in metastatic breast cancer and provides patients with a convenient oral once-daily dose.” The post FDA-approved therapy targets ESR1 mutations in breast cancer appeared first on Drug Discovery World (DDW).