BioSpace

NOVEMBER 21, 2023

CRISPR gene-editing has had its first ever approval in the UK. What can patients expect the price tag to be? Will the FDA follow suit?

Gene Therapy 122

Gene Therapy Gene Editing Gene 122

Drug Discovery World

JANUARY 9, 2023

“Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumour cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” . CRISPR-Cas9 gene editing .

Scientist 52

Scientist Gene Editing Gene Engineer 52

Drug Discovery World

OCTOBER 31, 2022

The last month has seen huge strides forward in our understanding of cancers, particularly in how they develop resistance to therapies and how we can ‘outsmart’ them using gene editing or different therapeutic pathways, but also how we can better target drugs to individuals and accurately predict treatment outcomes.

Drugs 52

Drugs Research Gene Editing Protein 52

XTalks

DECEMBER 13, 2023

Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. The process leading up to injection includes blood transfusions over three to four months, extraction of stem cells and chemotherapy to destroy the bone marrow to ensure there are no remaining defective stem cells.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

The Pharma Data

MARCH 7, 2022

Cas9 is the protein used in the gene editing process called CRISPR. Cas9 binds and uses RNA as an address-tag. The system allows scientists, by synthesizing a specific “address-tag” RNA, to bring Cas9 to a precise location in genome and therefore cut and splice genes at specific sites.

Protein 52

Protein Gene Genome Genomics 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

XTalks

FEBRUARY 11, 2021

After several such Nobel Prize snubs for female scientists, 2020 saw the Nobel Prize in Chemistry go to a female scientist duo for the first time for their revolutionary discovery of the CRISPR-Cas9 gene editing system. Their pioneering work has led to the “CRISPR revolution,” spurring a new era in genomics research.

Genetics 119

Genetics DNA Genome Genomics 119