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

XTalks

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Where is the drug discovery expertise happening in the UK?

Drug Discovery World

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

Drugs 75
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Bioinformatics Jobs: How to Succeed in This Competitive Space

XTalks

Bioinformatics jobs are commonly found in the fields of computer information science, pharmaceuticals, biotechnology, medical technology, computational biology, proteomics and medical informatics. The Human Genome Project could not have succeeded without the use of bioinformatics. Bioinformatics Engineer. Job Description.

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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. Rosalind Franklin.

Genetics 119
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Unlocking disease treatment with large-scale research

pharmaphorum

The importance of health data to the life science industry is growing just as rapidly as the technology enabling its capture is advancing. A few decades ago, gathering genetic data on the scale of the 100,000 Genomes Project would have been unthinkable – it was only in 2003 that the entire human genome was mapped.

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The era of precision neuroscience

Drug Discovery World

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 64
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Why a recent advancement is a giant leap for human genomics

Drug Discovery World

The first complete, gapless sequence of a human genome was published 1 April 2022 in a special issue of the journal Science 1. While The Human Genome Project mapped about 92% of the human genome two decades ago, sequencing the last 8% of the genome proved highly challenging.

Genome 52