Bio Pharma Dive

JUNE 21, 2023

20, roughly two weeks after a verdict is expected on a rival gene editing treatment from Vertex and CRISPR Therapeutics. The regulator will issue a decision on lovo-cel by Dec.

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Gene Editing Gene Gene Therapy Regulation 251

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

Fierce Pharma

DECEMBER 8, 2023

| Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. The transformational treatment is a potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black.

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Gene Therapy FDA Approval Gene Gene Editing 129

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 144

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 Gene Therapy 126

Fierce Pharma

DECEMBER 8, 2023

Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell | Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival (..)

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Gene Editing Gene Gene Therapy FDA Approval 137

Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We By focusing on the in-vivo delivery of genetic cargoes to the microbiome, Eligo’s hopes to go beyond traditional gene therapy and gene editing.

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Gene Editing Gene In-Vivo Genetics 52

Bio Pharma Dive

FEBRUARY 23, 2021

Guide Therapeutics, the company Beam acquired, is working on ways to more efficiently deliver gene editing therapies into the body.

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Gene Editing Gene Gene Therapy Development 283

BioSpace

NOVEMBER 21, 2023

CRISPR gene-editing has had its first ever approval in the UK. Will the FDA follow suit? What can patients expect the price tag to be?

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Gene Therapy Gene Editing Gene 125

Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT).

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Gene Therapy Gene Gene Editing Clinical Trials 111

BioSpace

SEPTEMBER 27, 2023

The New England Journal of Medicine study suggests that the 2022 death of a Duchenne muscular dystrophy patient was due to an adverse effect of recombinant AAV used to deliver the gene therapy.

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Gene Editing Gene Gene Therapy Medicine 126

BioSpace

JUNE 9, 2023

Data show the potential of Editas’ sickle cell disease and transfusion-dependent beta thalassemia gene therapy candidate, but it might not be enough to overtake Vertex and CRISPR Therapeutics.

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Gene Editing Gene Gene Therapy 110

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

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.

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Gene Therapy FDA Approval Gene In-Vivo 98

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.

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Gene Editing Gene In-Vivo Clinical Trials 59

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. DOWNLOAD THE FULL ARTICLE HERE.

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Gene Editing Gene Gene Therapy Genetics 52

BioSpace

SEPTEMBER 28, 2021

?Gene therapy research delivers breakthroughs this week with a CRISPR-edited droplet that could lead to a cure for heart disease and the possibility of turning glial brain cells into neurons.

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Gene Editing Gene Gene Therapy Research 109

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 Genome Genomics 64

Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

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Gene Therapy Gene Gene Editing Manufacturing 52

pharmaphorum

FEBRUARY 23, 2022

As investment in gene therapies continues to grow rapidly, more effectively engaging patients throughout development has become a priority for any company serious about building safer and more meaningful gene therapy programs. The post 2nd Annual Gene Therapy Patient Engagement Summit appeared first on.

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Gene Therapy Gene Gene Editing Clinical Trials 52

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Gene Therapy FDA Approval Gene Gene Editing 52

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Gene Therapy Gene Gene Editing In-Vivo 59

BioTech 365

JANUARY 10, 2022

Bayer and Mammoth Biosciences to Collaborate on Novel Gene Editing Technology Bayer and Mammoth Biosciences to Collaborate on Novel Gene Editing Technology Agreement strengthens Bayer’s new cell and gene therapy platform further Supports Mammoth’s vision of unlocking the full potential … Continue reading →

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Gene Editing Gene Gene Therapy 52

Fierce Pharma

DECEMBER 21, 2023

As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their CRISPR-based gene-editing therapy Casgevy, one doctor who treats sickle cell disease patients is already | As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their world-first CRISPR-based gene therapy Casgevy, one doctor (..)

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Doctors Doctor Gene Editing Gene Therapy 116

Medical Xpress

DECEMBER 21, 2022

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

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Gene Therapy Gene Editing Gene Research 98

pharmaphorum

OCTOBER 21, 2020

million) to develop its gene therapy for a rare ocular disease. SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one oxidative stress reducing enzyme which, acting together, aim to slow or stop the degeneration of photoreceptors. million euros ($52.7

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Gene Therapy Gene Gene Editing Genetics 96

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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Gene Therapy Gene Gene Editing In-Vivo 40

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

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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Gene Therapy Gene Gene Sequencing Gene Editing 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.

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Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA.

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Gene Editing DNA Gene Gene Therapy 235

Drug Discovery World

MAY 23, 2023

DDW’s Megan Thomas caught up with George White , General Manager, Product Management, Cell & Gene Therapy at Cytiva, to learn more about the company’s insight into the CGT market. Key challenges for cell and gene therapies In order for cell and gene (CGT) therapies to succeed, George White identifies several challenges to overcome.

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Gene Therapy Gene Gene Editing Production 52

BioSpace

OCTOBER 26, 2021

With the complete acquisition of Life Edit, ElevateBio will be able to integrate that company’s genome editing capabilities with its cell and gene therapies.

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Gene Editing Gene Gene Therapy Genome 95

Drug Discovery World

OCTOBER 12, 2023

Samulski’s graduate work demonstrated the first substrate for adeno-associated viruses (AAV) as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field.

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Gene Therapy Gene FDA Approval Clinical Trials 52

STAT News

MARCH 22, 2023

DALLAS — It took just three years, after the tool’s invention, for researchers to devise ways of using CRISPR-Cas9 gene editing to treat mice with Duchenne muscular dystrophy. Yet, with one exception, no gene-editing treatments for the rare muscle-wasting disease have entered the clinic and none appear particularly close.

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Gene Editing Gene Therapy Research Gene 98

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Gene Therapy Gene Production Clinical Trials 52

Pharmaceutical Technology

NOVEMBER 16, 2022

A custom-made viral vector and cell therapy centre of excellence, ElevateBio BaseCamp has an end-to-end process development and existing Good Manufacturing Practice (cGMP) production expertise for research, clinical and commercial cell and gene therapies and regenerative treatments.

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Development Gene Therapy Engineer Gene Editing 246