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

XTalks

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.

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Why genomic healthcare data matters in the development of new therapies 

Drug Discovery World

One might argue that this all started getting exciting with the launch of the Human Genome Project, which the National Human Genome Research Institute consider to be one of the greatest scientific feats in history 1. DDW’s Megan Thomas observes developments of accessibility in this sector and the potential impact. .

Genome 98
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Where is the drug discovery expertise happening in the UK?

Drug Discovery World

The country’s pioneering genomics research, crucial for future drug discovery, is demonstrated by institutes like the Wellcome Sanger Institute and projects like the 100,000 Genomes Project. Cambridge is a focal point for many pharmaceutical companies working in this therapeutic area.

Drugs 75
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CRISPR therapies targeting the next breakthrough in oncology

pharmaphorum

The potential to develop treatments with CRISPR gene editing is hinged on its ability to add, delete or replace elements within a target DNA sequence. The potential for CRISPR gene editing to offer a therapeutic option to patients is clear, based on its mechanism, but there are already gene editing therapeutics on the market.

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Women in Science Who Have Paved the Way Forward in Genetics

XTalks

The Human Genome Project recently marked 20 years since the publication of the first full sets of human genomic sequences, an endeavor that spanned well over a decade. Today, new next-generation sequencing technologies allow for the sequencing of complex genomes within just a day or two.

Genetics 119