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Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Related: Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug The advisors appeared convinced that exa-cel can offer significant benefits to people with sickle cell.

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Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

coli bacteria, as well as lambda bacteriophage, can adapt to the alterations in the composition of their nutrient medium. Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Figure below presents an overview of the mechanism of action of gene switch.

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Global Roundup: EU Approves ViiV’s HIV-1 Drug and More

The Pharma Data

Additionally, Avacta announced it submitted a Clinical Trial Application in the UK for a Phase 1, first-in-human, open label, dose-escalation and expansion study of its lead pre|CISION prodrug, AVA6000, in patients with locally advanced or metastatic selected solid tumors. Elsewhere around the world: Avacta Group – U.K.

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BioSpace Global Roundup, Oct. 15

The Pharma Data

VBL has recently demonstrated ex-vivo activity of anti-MOSPD2 antibodies in patients with relapsing-remitting and progressive multiple sclerosis (MS), as well as in animal models of rheumatoid arthritis (RA), nonalcoholic steatohepatitis (NASH) and inflammatory bowel disease (IBD). 1. -- -->. -- [if lte IE 8]--> .