Bio Pharma Dive

JUNE 21, 2021

The next six months could feature clinical milestones for CRISPR gene editing, the treatment of COVID-19, microbiome drugs and gene-targeted cancer therapy.

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Gene Editing Clinical Trials Clinical Trials Gene 363

Bio Pharma Dive

MARCH 27, 2023

Vertex will license CRISPR technology to develop insulin-producing islet cells that are more resistant to immune rejection, adding to other efforts directed at Type 1 diabetes.

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Gene Editing Insulin Gene Licensing 290

Bio Pharma Dive

OCTOBER 19, 2020

The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.

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Bio Pharma Dive

JANUARY 12, 2021

Verve, a buzzy gene editing startup, unveiled its first drug candidate on Tuesday, while Eli Lilly's and Pfizer's CEO gave updates on Alzheimer's and dealmaking.

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Gene Editing Gene Drugs 189

Bio Pharma Dive

DECEMBER 3, 2020

A closely watched gene editing therapy, a slate of new multiple myeloma drugs and much more are set to highlight the American Society of Hematology conference this weekend.

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Gene Editing Gene Drugs 341

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.

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Gene Editing Gene Gene Therapy In-Vivo 189

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 270

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 281

Bio Pharma Dive

SEPTEMBER 16, 2022

Preliminary study results show the biotech’s gene editing treatment could reduce markers and symptoms of hereditary angioedema, though the data come from few patients and follow-up remains limited.

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Gene Editing Gene Drugs 302

Bio Pharma Dive

JANUARY 12, 2022

A restrictive Medicare decision on Alzheimer's drugs sent Lilly shares tumbling, while the CEO of Editas brushed off competitive threats to his company's gene editing technology.

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Gene Editing Gene Drugs 319

Bio Pharma Dive

JUNE 22, 2022

The pharma will pay $75 million to access Precision’s gene editing technology, adding another program to the lengthening list of drug development efforts targeting the inherited blood condition.

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Gene Editing Gene Development Drugs 304

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 274

Bio Pharma Dive

SEPTEMBER 12, 2022

Scientists at Pretzel believe fixing mutated mitochondrial DNA with a mix of small molecule therapies and gene editing could be key to solving a number of hard-to-treat diseases.

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Gene Editing DNA Gene Scientist 259

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

JUNE 10, 2022

Three of the six lymphoma patients who received Caribou’s gene editing treatment have relapsed in the latest sign that so-called allogeneic drugs could have trouble matching their CAR-T counterparts.

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Gene Editing Gene Drugs 271

Bio Pharma Dive

MAY 26, 2022

Bosley, who once led the CRISPR gene editing biotech, will become a venture partner at Advent Life Sciences, which backs a range of early-stage drug companies.

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Gene Editing Life Science Gene Drugs 260

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 121

Bio Pharma Dive

OCTOBER 31, 2023

Already a partner to Verve, the pharma is paying $200 million upfront and investing another $50 million in Beam to acquire option rights to several gene editing programs.

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Gene Editing Gene Drugs 213

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

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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Bio Pharma Dive

JANUARY 22, 2025

The biotech said it has dosed the first participant in a Phase 3 study of its in vivo gene editing treatment. Elsewhere, the FDA imposed a hold on Atara and lifted another on Amylyx.

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Gene Editing In-Vivo Gene Drugs 173

Bio Pharma Dive

JULY 31, 2024

staff, while gene editing specialist Intellia is now cleared to start a U.K. Elsewhere, Fibrogen plans to lay off three-quarters of its U.S. study focused on AAT deficiency.

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Bio Pharma Dive

DECEMBER 15, 2023

While a positive recommendation for the gene editing treatment Casgevy was expected, a clearance for Biogen’s Skyclaris, acquired through a recent acquisition, was no sure bet.

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Gene Editing Regulation Gene Drugs 173

STAT News

NOVEMBER 7, 2022

Verve Therapeutics said Monday that its experimental gene-editing treatment for a common form of heart disease was placed on clinical hold by the Food and Drug Administration, potentially delaying an ongoing, early-stage clinical trial. The Cambridge, Mass.-based

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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 164

Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022.

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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. Cell therapy, specifically, is changing our narrative around chronic and lethal conditions.

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pharmaphorum

FEBRUARY 1, 2024

A single dose of a gene-editing drug being developed by Intellia Therapeutics almost eliminated attacks in patients with hereditary angioedema (HAE) in a clinical trial, pointing to the potential of CRISPR-based drugs delivered in vivo to treat diseases.

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pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications.

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Gene Editing Gene Gene Therapy Drugs 59

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 post Vertex, CRISPR prep filing for gene-editing blood disorder therapy appeared first on.

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Gene Editing Gene In-Vivo Clinical Trials 59

BioSpace

AUGUST 23, 2021

With a handful of drugs for cystic fibrosis already approved, Vertex Pharmaceuticals has turned its attention to expanding its pipeline through collaborations and acquisitions the past few years.

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Gene Editing Gene Drugs 98

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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Pharmaceutical Technology

APRIL 4, 2023

Vertex Pharmaceuticals and CRISPR Therapeutics have completed the rolling submission of biologics licence applications (BLAs) to the US Food and Drug Administration (FDA) for exagamglogene autotemcel (exa-cel) to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

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Gene Therapy Gene Editing In-Vivo Gene 246

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . The post Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug appeared first on.

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In-Vivo Gene Editing Trials Drugs 105

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. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

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Gene Editing Trials Drugs In-Vivo 98

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. The move marks a continuation in its ongoing commitment to exploring gene-editing technology. Novartis also recently inked an up-to $1.5

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Gene Editing Gene Gene Therapy Genetics 52

pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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