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Gene editing: beyond the hype

pharmaphorum

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. That would just slow the whole field down.

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ElevateBio and Affini-T collaborate to develop T cell therapies

Pharmaceutical Technology

ElevateBio has entered a partnership with Affini-T Therapeutics to progress the latter’s engineered TCR-T therapies focused on Kirsten rat sarcoma viral oncogene homolog (KRAS), a dominant oncogenic driver mutation in solid tumours. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.

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Asklepios partners with ReCode on gene-editing platform

Pharmaceutical Technology

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

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Researchers develop CRISPR-based drug candidate for microbiome targeting

Pharmaceutical Technology

The Technical University of Denmark (DTU) has announced that an international scientific team has developed a CRISPR-based drug candidate that targets E coli directly and leaves the microbiome intact. The team then engineered the phages through gene editing to improve their targeting ability.

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ElevateBio raises funds to advance cell and gene therapies

Pharmaceutical Technology

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

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Editas grabs orphan drug status for sickle cell disease CRISPR therapy

Pharmaceutical Technology

EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. This increases the expression of fetal haemoglobin.