STAT News

SEPTEMBER 21, 2022

The short history of CRISPR gene editing in humans has, with rare exception, been a history of triumphant progress: A patient apparently cured of sickle cell in 2019, six patients with toxic DNA knocked out of their liver last year, another six patients with a different strand of toxic liver DNA knocked out last week.  

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Gene Editing Gene DNA Research 128

Bio Pharma Dive

NOVEMBER 14, 2022

The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.

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Gene Editing RNA DNA Gene 167

STAT News

NOVEMBER 14, 2022

Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA.    On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.

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Gene Editing RNA Gene Drugs 98

Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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STAT News

SEPTEMBER 16, 2022

Intellia Therapeutics said Friday the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells.

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DNA Gene Editing In-Vivo Genetics 142

Scienmag

MARCH 21, 2022

Wilmington, DE, (MARCH 21, 2022) – A new study from scientists at ChristianaCare’s Gene Editing Institute is advancing the safety and efficacy of using CRISPR gene editing in patient treatments by demonstrating how to identify and evaluate the broad-based biological impact of gene editing on targeted tissues, where the edits are designed to fully disable (..)

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Gene Editing Gene Scientist DNA 80

Scienmag

MAY 2, 2022

WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

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Gene Editing DNA Gene Development 58

Worldwide Clinical Trials

NOVEMBER 12, 2024

For example, once considered incurable and terminal, patients with sickle cell disease may reach new summits in their lives with gene editing technologies such as CRISPR to repair affected DNA and, in some cases, functionally cure the condition.

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Gene Therapy Clinical Supply Trials Gene 182

Scienmag

OCTOBER 20, 2021

The ability to edit the genome by altering the DNA sequence inside a living cell is powerful for research and holds enormous promise for the treatment of diseases. However, existing genome editing technologies frequently result in unwanted mutations or can fail to introduce any changes at all.

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DNA Gene Editing Genome Genomics 75

Camargo

JULY 27, 2021

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Additionally, gene editing allows us either to remove or to modify harmful genes.

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Gene Therapy Gene Gene Editing Genetics 238

STAT News

SEPTEMBER 20, 2022

A new CRISPR startup — backed by some big names in venture capital — is planning to develop gene-editing treatments that can insert a genetic sequence of any length, at any location in the DNA strand, according to industry insiders and documents.

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DNA Gene Editing Genomics Genome 98

STAT News

MARCH 16, 2023

CRISPR, by contrast, lets you cut almost any stretch of DNA with a simple chemical code. Many of these once beloved tools were tossed aside when CRISPR came along, having helped few actual patients but driven plenty of graduate students to exhaustion. They were stubborn, inflexible enzymes, requiring endless engineering.

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Gene Editing Gene DNA Genomics 98

BioSpace

APRIL 29, 2024

The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.

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Gene Editing Gene Medicine In-Vivo 88

Scienmag

JULY 18, 2022

HOUSTON – (July 18, 2022) – Rice University researchers have demonstrated that CRISPR-Cas9, increasingly famous as a gene-editing tool, can be employed in powerful additional ways in human cells.

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Gene Editing DNA Gene Research 84

BioPharma Reporter

OCTOBER 22, 2020

The use of CRISPR, the genetic scissors that allow scientists to edit the instruction manual of life, DNA, has drawn massive global attention over the last several years.

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Gene Editing DNA Genetics Scientist 105

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 Genomics 98

BioPharma Reporter

JANUARY 9, 2023

The collaboration will combine ReCodeâs delivery technology with AskBioâs gene editing and DNA cargoes to develop gene correction therapies for liver and lung diseases.

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Gene Editing Gene DNA Development 52

The Pharma Data

SEPTEMBER 3, 2020

Current scientific techniques are not yet safe or effective enough to be used to create gene-edited babies, an international committee says. The world’s first gene-edited babies were born in China in November 2018. Why is gene-editing babies controversial? The committee was set up in response.

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Gene Editing Gene Genomics Genome 52

BioTech 365

JANUARY 28, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: TALEN gene editing tool more efficient than CRISPR-Cas9 in compact DNA: study.TALEN gene editing tool more efficient than CRISPR-Cas9 in compact DNA: study aliu Thu, 01/28/2021 – … Continue reading →

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Gene Editing DNA Gene Research 40

STAT News

MARCH 9, 2023

On the third and final day of the Third International Summit on Human Genome Editing, the leading world experts in germline editing — that is, the practice of altering the DNA of sperm, eggs, and embryos — took center stage at the Francis Crick Institute in London to share their latest work.

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Gene Editing Genomics Genome DNA 98

STAT News

MARCH 3, 2023

Next week, hundreds of scientists from around the world will convene in London for an international summit on genome editing. That technology, which enables scientists to easily excise, alter, or replace specific sections of DNA, was awarded the 2020 Nobel Prize for Chemistry. Continue to STAT+ to read the full story…

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Genome Genomics Gene Editing Genetic Disease 111

Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

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DNA Gene Gene Editing Scientist 56

XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

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Gene Editing Life Science Gene Gene Therapy 52

Pharmaceutical Technology

JUNE 8, 2023

The ttLNP platform can deliver a range of high-impact genomic payloads, including gene editing, siRNA, mRNA, DNA and gene-writing apparatus, to tissues, cells and organs. The development will be advanced through both internal pipeline programmes and external collaborations.

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Genomics Genome Medicine Development 130

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 98

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 In-Vivo 98

BioTech 365

DECEMBER 4, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Advancing gene editing with new CRISPR/Cas9 variant.Using a new variant to repair DNA will improve both safety and effectiveness of the much-touted CRISPR-Cas9 tool in genetic research, … Continue reading →

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Gene Editing Gene DNA Genetics 40

pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. The technique introduces a break in a specific place within DNA that triggers a self-repair mechanism.

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Gene Editing Bacteria DNA Gene 105

STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy.

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Gene Editing Gene Gene Therapy Scientist 105

BioTech 365

OCTOBER 20, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: New tool offers ways to improve CRISPR gene-editing method.The ability to edit the genome by altering the DNA sequence inside a living cell is powerful for research … Continue reading →

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Gene Editing Gene DNA Genomics 40

Scienmag

JULY 2, 2021

The ongoing effort to explore […].

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Gene Silencing Gene Editing Gene DNA 75

pharmaphorum

FEBRUARY 19, 2021

Once someone is infected with HIV it stays with them for life thanks to the retrovirus’s ability to inject its DNA code into the host. Khalili is a named inventor on patents that cover the viral gene editing technology and also holds equity in Excision, where he will act as chief scientific consultant.

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Gene Editing In-Vivo DNA In-Vitro 105

pharmaphorum

APRIL 23, 2021

CRISPR Therapeutics is to receive a hefty $900m payment from Vertex after the companies amended a collaboration to develop, manufacture and market a gene editing therapy for sickle cell disease and beta thalassemia.

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Gene Editing Gene DNA Bacteria 64

Pharmaceutical Technology

FEBRUARY 15, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. The company is largely involved in genome editing using CRISPR/Cas9 and CRISPR/Cas12a systems.

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In-Vivo Genomics Genome Gene Editing 162

BioTech 365

OCTOBER 20, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: CRISPR meets Pac-Man: New DNA cut-and-paste tool enables bigger gene edits.Gene editing for the development of new treatments, and for studying disease as well as normal function … Continue reading →

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DNA Gene Editing Gene Development 40

BioTech 365

APRIL 9, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: New, reversible CRISPR method can control gene expression while leaving underlying DNA sequence unchanged.Over the past decade, the CRISPR-Cas9 gene editing system has revolutionized genetic engineering, allowing … Continue (..)

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Gene Expression DNA Gene Editing Gene 40

Pharmaceutical Technology

MARCH 1, 2023

The drug candidate targets DNA-dependent protein kinase (DNA-PK). Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems. The drug candidate is administered through the oral route in the form of capsule and tablet.

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Gene Editing DNA Cosmetics Life Science 100