pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. That would just slow the whole field down.

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Gene Editing Gene Gene Therapy In-Vivo 145

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

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Gene Editing Gene Gene Therapy Genetics 130

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The post Moderna partners with Life Edit for mRNA gene editing therapies appeared first on Pharmaceutical Technology.

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Gene Editing Gene In-Vivo Gene Therapy 147

STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

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Gene Editing Gene Genetic Disease Research 126

Bio Pharma Dive

FEBRUARY 28, 2024

The acquisition of Proof Diagnostics, which was formed to develop a COVID-19 test, gives Ginkgo a library of novel enzymes that can be used to make genetic medicines.

Gene Editing 173

Gene Editing Gene Genetics Medicine 173

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). The company will receive a combined upfront payment and equity investment of $60m.

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Gene Editing Gene Gene Therapy In-Vivo 130

BioSpace

MARCH 24, 2024

Ubiquitous potential, possible safety advantages and the recent growth of cell and gene therapy are driving investment in a different type of genetic editing.

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Gene Editing Gene Gene Therapy Genetics 140

XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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Gene Editing DNA Gene Gene Sequencing 98

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

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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Gene Editing DNA Gene Gene Therapy 235

Worldwide Clinical Trials

NOVEMBER 12, 2024

CGTs address rare and complex diseases at the root cause with increasing use in cancer, genetic disorders, and autoimmune diseases. Cell therapy, specifically, is changing our narrative around chronic and lethal conditions.

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Gene Therapy Clinical Supply Trials Gene 182

Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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Gene Therapy Gene Gene Editing Development 243

pharmaphorum

FEBRUARY 15, 2024

Gene-editing biotech Prime Medicine has priced its IPO, hoping to raise $140m to advance its pipeline of one-shot therapies for severe genetic disorders.

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Gene Editing Gene Medicine Genetics 64

STAT News

MARCH 1, 2023

Tessera Therapeutics chief scientist Michael Holmes had just finished presenting a much-anticipated peek at a technology the company had previously said could “ revolutionize genetic medicine ” and “cure nearly any genetic disease.”

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Gene Editing Genome Genomics Genetic Disease 119

Medical Xpress

MARCH 17, 2023

The study, to be published March 17 in the Journal of Experimental Medicine, uses a new, highly versatile form of CRISPR-based genome editing with the potential to correct a wide variety of disease-causing genetic mutations.

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Gene Editing Gene Genome Genomics 98

BioSpace

MARCH 21, 2024

Tegoprubart, Eledon Pharmaceuticals’ experimental anti-CD40L antibody, was part of the immunosuppressive treatment regimen used following the first-ever transplant of a kidney from a genetically modified pig to a human.

Gene Editing 98

Gene Editing Antibody Gene Genetics 98

pharmaphorum

MARCH 27, 2025

In a xenotransplant milestone, scientists have carried out the first transplant of a genetically modified pig liver into a brain-dead human patient

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Gene Editing Gene Genetics Scientist 74

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

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

Pharmaceutical Technology

AUGUST 24, 2022

A lead product candidate of the company, GTP-506 is a single-dose gene editing therapy. The ARCUS nuclease vector (GTP-506A) targets gene editing in the PCSK9 gene locus that is well characterised, while the therapeutic donor vector (GTP-506D) introduces the OTC gene to provide the required genetic correction.

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Gene Editing Gene Therapy Gene Genetics 130

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. The post Biogen teams up with gene-editing startup Scribe on ALS programme appeared first on. Jennifer Doudna.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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Gene Editing Gene Genomics Genome 64

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.

Gene Editing 105

Gene Editing DNA Genetics Scientist 105

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.

DNA 142

DNA Gene Editing In-Vivo Genetics 142

pharmaphorum

FEBRUARY 15, 2024

Gene-editing biotech Prime Medicine has priced an initial public offering, hoping to raise $140 million to advance its pipeline of one-shot therapies for severe genetic disorders. The Cambridge, Massachusetts-based company is offering 19.2 million shares at $6.25

Medicine 105

Medicine Gene Editing Genetics Gene 105

STAT News

MARCH 9, 2023

LONDON — Scientists at this year’s genome editing summit spent Tuesday showing the world just how far CRISPR -based medicines for treating human diseases have come in a decade.

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Gene Editing Genome Genomics DNA 98

BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

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Gene Editing Genetic Disease Gene In-Vivo 52

pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Scienmag

AUGUST 18, 2020

Credit: Rasgon Lab, Penn State Researchers who developed an improved method of gene editing for the study of arthropods will expand the technology for use in vertebrate species such as mice, fish and birds after receiving new funding from the National Science Foundation.

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Gene Editing Gene Development Research 61

STAT News

MARCH 3, 2023

Continue to STAT+ to read the full story…

Genome 111

Genome Genomics Gene Editing Genetic Disease 111

pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Novartis also recently inked an up-to $1.5

Gene Editing 52

Gene Editing Gene Gene Therapy Genetics 52

STAT News

NOVEMBER 29, 2022

He Jiankui, the Chinese biophysicist who created the first gene-edited children , had been quiet since completing a three-year prison sentence in April, leaving many to wonder whether he had plans to return to scientific research. Earlier this month, we got his answer.

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Gene Editing Scientist Gene Research 144

Scienmag

OCTOBER 21, 2020

–Genome editing of human embryos represents one of the most contentious potential scientific applications today. But what if geneticists could sidestep the controversy by editing sperm and eggs instead? Credit: Photo by L. Brian Stauffer CHAMPAIGN, Ill.

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Gene Editing Gene Genome Genomics 88

Scienmag

JANUARY 12, 2021

Martins Scientists have used gene-editing advances to achieve a tenfold increase in the production of super-bug targeting formicamycin antibiotics. Credit: Dr Dino J. The John Innes Centre researchers used the technology to create a new strain of Streptomyces formicae bacteria which over-produces the medically promising molecules.

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Gene Editing Gene Bacteria Scientist 53

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.

DNA 98

DNA Gene Editing Genome Genomics 98

Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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Research Gene Gene Therapy Genomics 264

BioSpace

JULY 12, 2021

The company's technology aims to precisely search out genetic mutations at their precise genome location, limiting concerns about toxicities or unwanted cellular changes.

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Gene Editing Genome Genomics Gene 72