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Gene editing in organoids: accounting for complexity in drug discovery

Drug Discovery World

The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. . What are organoids? .

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Innovation in immuno-oncology: Leading companies in in-vitro T-cell activation

Pharmaceutical Technology

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: In-vitro T-cell activation. Immatics is the leading patent filer in in-vitro T-cell activation.

In-Vitro 130
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Discover how iPSC derived cells can support drug and gene therapy development

Drug Discovery World

DDW recently hosted a webinar ‘Discover how iPSC derived cells can be used to support the development of drug and gene therapies’, supported by DefiniGEN. Chris explained that DefiniGEN is a liver cell company focussed on hepatocytes, particularly supporting the drug development industry in producing liver therapies.

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Could CRISPR cure HIV? US biotech Excision raises $60m to find out

pharmaphorum

Snipping out this viral code with powerful CRISPR gene editing technology, which last year won Drs Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize for chemistry, would in theory prevent the need for drugs to suppress the virus and the development of AIDS.

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The use of base editing in stem-cell based therapies

Drug Discovery World

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

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Nine new industry collaborations driving drug discovery forward

Drug Discovery World

With the integration of CRISPR/Cas9 gene editing technology we are increasing our capability to identify and validate potential drug targets, to generate more predictive pre-clinical models and to elucidate the mechanisms of action of novel therapeutics.” We see a great need for a vaccine against EBV.”

Drugs 52
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RNA interference method could treat muscular dystrophy

Drug Discovery World

One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. Co-author Hiroki Kurahashi added: “Patients with FCMD who carry the abnormal FKTN gene produce non-glycosylated α-DG, which makes them bedridden right from adolescence.

RNA 52