Bio Pharma Dive

JULY 12, 2022

A patient in New Zealand became the first person to receive the biotech’s medicine, which uses base editing to turn off a specific gene in the liver and thereby lower cholesterol.

Gene Editing 278

Gene Editing Gene Medicine 278

Bio Pharma Dive

JUNE 15, 2023

Lilly will pay the genetic medicine specialist $60 million upfront to work together on a treatment targeting a risk factor known as Lp(a).

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Gene Editing Gene Genetics Medicine 316

Bio Pharma Dive

APRIL 3, 2023

for a CRISPR-based medicine and puts the partners ahead of a rival therapy from Bluebird bio. The approval application is the first in the U.S.

Gene Editing 306

Gene Editing Gene Medicine Drugs 306

Bio Pharma Dive

NOVEMBER 17, 2022

While the biotech will not develop the gene editing therapy further on its own, CEO Gilmore O’Neill claimed the data are still a meaningful demonstration of what CRISPR can accomplish.

Gene Editing 306

Gene Editing Medicine Gene Development 306

Bio Pharma Dive

FEBRUARY 28, 2022

The findings build on early evidence that gene editing inside the body could safely and effectively treat disease, and suggest the effects of Intellia's medicine might last.

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Gene Editing Gene Medicine Drugs 344

Bio Pharma Dive

APRIL 24, 2023

Submission of the application comes weeks after rivals Vertex and CRISPR filed their gene editing medicine for the disease with the agency.

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Gene Editing Gene Gene Therapy FDA Approval 258

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

Bio Pharma Dive

OCTOBER 31, 2023

Expert advisers are reviewing the companies’ case for approval of what could be the first medicine based on the gene editing technology. Follow their discussion here.

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Gene Editing Gene Medicine 328

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.

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Gene Editing Gene Gene Therapy In-Vivo 145

Bio Pharma Dive

SEPTEMBER 26, 2022

The startup, which launched last year with $315 million in funding and plans to advance a new form of gene editing, is one of the first big venture-backed biotechs in several months to seek an IPO.

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Gene Editing Medicine Gene 289

Bio Pharma Dive

OCTOBER 3, 2023

The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.

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Gene Editing In-Vivo Gene Genetics 258

Bio Pharma Dive

JUNE 15, 2021

A deal with biotech startup Capsida, which recently raised $140 million, gives CRISPR access to a Caltech technology that aims to improve the delivery of genetic medicines.

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Gene Editing Gene Genetics Development 281

Bio Pharma Dive

OCTOBER 22, 2024

The gene editing company will focus on “in vivo” medicines, while seeking to license out or find a development partner for its clinical-stage treatment reni-cel.

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Gene Editing In-Vivo Licensing Licensing 286

Bio Pharma Dive

JUNE 26, 2024

The findings provide early proof that multiple doses of a gene editing medicine can be safely administered with additive effects, overcoming a key constraint of the complex therapies.

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Gene Editing Gene Medicine Drugs 278

Rethinking Clinical Trials

JULY 31, 2024

Adrian Hernandez In this Friday’s PCT Grand Rounds, Adrian Hernandez of Duke University will present “Precision Health to Population Health: Opportunities and Challenges for Gene Editing Therapies.” ” The Grand Rounds session will be held on Friday, August 2, 2024, at 1:00 pm eastern. Join the online meeting.

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Gene Editing Gene Clinical Trials Clinical Trials 144

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. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Bio Pharma Dive

FEBRUARY 28, 2023

An alliance with the startup brings Vertex an RNA-based medicine for the disease, adding to a portfolio that already includes a gene editing treatment.

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Gene Editing RNA Gene Research 269

Bio Pharma Dive

SEPTEMBER 9, 2021

Formed as a diagnostics developer, Mammoth Biosciences, which Doudna started with two of her former students at the University of California, has accelerated plans to make gene editing medicines as well.

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Gene Editing Development Gene Medicine 271

Bio Pharma Dive

AUGUST 18, 2022

The company plans to soon seek approval of a once-monthly preventive drug for hereditary angioedema, a disease other drugmakers are targeting with gene editing and RNA medicines.

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Gene Editing RNA Drugs Gene 268

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

Biology is opening up new frontiers in medicine. We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics.

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Genetic Disease Genetics Gene Editing Medicine 189

Bio Pharma Dive

FEBRUARY 8, 2024

The company is one of three preclinical biotechs to successfully go public since last summer, but it pulled in significantly less than others who’ve debuted with more advanced medicines.

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Gene Editing Gene Medicine 200

Bio Pharma Dive

AUGUST 16, 2023

The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on gene editing medicines.

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Gene Editing Gene Research Medicine 246

Bio Pharma Dive

FEBRUARY 15, 2022

By partnering with ONK Therapeutics, Intellia joins CRISPR Therapeutics and Editas Medicine in striking deals that marry gene editing with a fast-emerging form of cancer immunotherapy.

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Gene Editing Gene Medicine 246

Medical Xpress

MAY 1, 2023

Gene-editing therapy aimed at two targets—HIV-1, the virus that causes AIDS, and CCR5, the co-receptor that helps the virus get into cells—can effectively eliminate HIV infection, new research from the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) shows.

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Gene Editing Gene Medicine Drugs 122

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

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).

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Gene Editing Gene Gene Therapy In-Vivo 130

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 161

Bio Pharma Dive

OCTOBER 7, 2024

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

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Gene Editing In-Vivo Gene Medicine 183

BioSpace

JULY 6, 2021

This appears to be a second example of CRISPR gene editing inside the body, as opposed to in cell cultures.

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Gene Editing In-Vivo Gene Medicine 96

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.

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Gene Editing Gene Genetics Medicine 169

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

BioSpace

JUNE 12, 2022

Shape Therapeutics is evolving programmable RNA medicines that push the limits of current gene editing capabilities. BioSpace spoke with CSO David Huss.

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Gene Editing RNA Gene Medicine 71

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

Bio Pharma Dive

DECEMBER 5, 2023

The gene editing biotech is shelving two of its most advanced cancer drugs, and joining a growing group of companies exploring cell-based medicines for inflammatory diseases like lupus.

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Gene Editing Gene Medicine Drugs 161

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

pharmaphorum

MAY 29, 2024

In a new episode of the pharmaphorum podcast, Rahul Kakkar, CEO of gene editing company Tome, speaks with web editor Nicole Raleigh about programmable genomic integration (PGI) technology.

Genomics 118

Genomics Genome Gene Editing Medicine 118