The Pharma Data

MARCH 7, 2022

The approach will allow researchers to understand the role individual genes play in normal cell growth and development, in aging, and in such diseases as cancer, said Shiri Levy, a postdoctoral fellow in UW Institute for Stem Cell and Regenerative Medicine (ISCRM) and the lead author of the paper. Cas9 binds and uses RNA as an address-tag.

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Protein Gene Genome Genomics 52

XTalks

NOVEMBER 30, 2023

Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. In 2021, the FDA granted approval to San Francisco-based Excision BioTherapeutics to start trials assessing CRISPR gene editing as a potential treatment for HIV.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article: Where are the hottest drug discovery hubs in the US?

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Gene Therapy Gene Antibody FDA Approval 59

Delveinsight

JANUARY 14, 2021

The study’s primary result will be the proportion of patients attaining a functional treatment, defined as an off-therapy loss of hepatitis B surface antigen (HBsAg) and HBV DNA from the serum. Verve Therapeutics has an aim to bring one-and-done gene editing treatments to heart disease.

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Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52

Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

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DNA In-Vivo Genetics Medicine 52

Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. This is reflected by our recent share price performance outperforming the index in the past year.”

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Drugs Life Science Gene Therapy Genome 75

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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Gene Editing Gene DNA Engineer 96

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.

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Gene Editing DNA Gene Genome 98

Drug Discovery World

MAY 2, 2023

Steve Wowk , VP, Business Unit and General Management of Integrated DNA Technologies, shared with DDW the value of CRISPR-Cas9 in drug discovery. The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system.

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Genome Genomics Gene DNA 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g.,

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DNA Genome Genomics RNA 98

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR. pyogenes dCas9.

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pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. Other CRISPR-based therapies generate a DSB at a precise location, after which cellular processes carry out the editing function.

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Gene Editing Trials Drugs Gene 98

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.

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Gene Editing Gene Engineer DNA 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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DNA Gene Gene Silencing Genome 52

The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. Photo courtesy of Science Advances.

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Gene Editing DNA Genome Genomics 52

Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. One of the most exciting recent developments is the use of gene editing tools to create cell and gene therapies.

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Research Drugs Protein RNA 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize. The illness was later named coronavirus disease 2019 (COVID-19).

Life Science 111

Life Science Vaccination Vaccine Gene 111

Pharmaceutical Technology

FEBRUARY 20, 2023

He says these issues must be urgently addressed over the next few years as advancing medical knowledge and the role of genomics in treating disease ushers in a new era of precision medicine and new potential to address diseases that affect certain populations.

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