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Gene therapy cures genetic hearing loss

Drug Discovery World

In the first case of its kind, gene therapy has restored hearing in a patient in the United States. AK-OTOF is a gene therapy being developed for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF). “These initial results show that it may restore hearing better than many thought possible.”

Therapies 260
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EU relaxes safety monitoring of CAR-T therapies

Drug Discovery World

The European Medicines Agency has approved a Type II variation to the Summary of Product Characteristics (SmPC) for Kite’s chimeric antigen receptor (CAR)-T cell therapies, Yescarta (axicabtagene ciloleucel) and Tecartus (brexucabtagene autoleucel).

Therapies 130
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How can long-read nanopore sequencing support gene therapy delivery?

Drug Discovery World

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. This webinar will detail how Oxford Nanopore evaluated long-read nanopore sequencing as a comprehensive and sensitive QC method for rAAVs which is supporting internal research activities at AstraZeneca.

Therapies 173
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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 post Stem cell therapy shows promise in advanced MS appeared first on Drug Discovery World (DDW). But this is an encouraging step towards a new way of treating some people with MS.”

Therapies 264
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Non-viral gene therapy shows early efficacy in lung cancer

Drug Discovery World

The data will be presented at the 2023 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston. Developed by gene therapy company Genprex, Reqorsa is a non-viral gene therapy that leads to expression of the TUSC2 tumour suppressor gene in cancers.

Therapies 277
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International consortium launches €12.8 million lung cancer project

Drug Discovery World

Over the next three years, the consortium will progress the development of a regenerative gene therapy for lung cancer. . The new treatment will use OmniSpirant’s OmniSom technology to develop an inhaled regenerative gene therapy. The post International consortium launches €12.8

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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. The post How ready are we for radioligand therapies? RA: What are the potentials of RLTs across cancer? JT: This is very important 6.

Therapies 147