Remove Genetics Remove Immune Response Remove In-Vivo Remove RNA
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LNP formulations produce strong immune responses, data shows 

Drug Discovery World

The etherna paradigm for cLNP design is ushering in the rapid generation of novel families of delivery systems for not only vaccination but also for in vivo tolerisation for a host of autoimmune diseases.” The post LNP formulations produce strong immune responses, data shows appeared first on Drug Discovery World (DDW).

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Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

WCG Clinical

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 52
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Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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Taking an ‘upside-down’ approach to mRNA delivery

pharmaphorum

Once its potential as a means of stimulating an immune response had been established, attention quickly turned to where else the technology could provide a therapeutic solution. The intracellular barriers include endosomal escape, RNA sensors, and endonucleases.

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The challenges and trends of cell & gene therapies 

Drug Discovery World

Speaking about emerging opportunities, Carlo Russo, CEO of Genenta, says that whilst “immuno-oncology treatment has been very successful in treating patients suffering from blood cancer,” its success has been limited “by the durability of the responses”. billion, compared to $19.9 million price point per dose.

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Delivering on the promise of gene editing

Drug Discovery World

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al.