pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

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Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

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Drugs Life Science Gene Therapy Genome 75

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. Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys.

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Gene Editing Gene Gene Therapy Clinical Trials 105

Drug Discovery World

MARCH 9, 2023

This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures. The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

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In-Vivo Gene Editing Gene Trials 52

Drug Discovery World

NOVEMBER 17, 2023

The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field.

Gene Therapy 52

Gene Therapy Gene Drugs Gene Editing 52

Drug Discovery World

AUGUST 2, 2022

CureHeart is using gene therapy technologies to edit or silence the faulty genes that cause inherited heart muscle diseases. . It’s estimated that one in 250 people worldwide are affected by genetic cardiomyopathies, with a 50:50 risk they will pass their faulty genes on to each of their children. .

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Gene Editing Gene Research Genetics 52

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. These cells present a highly scalable platform, capable of manufacturing advanced cellular therapeutics.

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Gene Editing Gene Engineer DNA 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. These methods offer safer CAR T-based therapies, laying the foundation for clinical trials and the rapid development of a new generation of CAR T cells for both autologous and allogeneic use.

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DNA Genome Genomics RNA 98

XTalks

AUGUST 2, 2023

Johnson & Johnson’s Top 5 Best-Selling Drugs of 2022: 1) Stelara (ustekinumab) Stelara is an immunosuppressant biologic therapy that blocks the IL-12 and IL-23 proteins that play a role in plaque psoriasis and Crohn’s disease. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A.

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Sales Manufacturing FDA Approval Life Science 98

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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Life Science Vaccination Vaccine Gene 111

Drug Discovery World

FEBRUARY 22, 2024

For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. years it takes to complete an entire cancer clinical trial. The organisation also notes that only 1% of the people that can take part in dementia clinical trials actually do so. This compares to the 2.3

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Drugs Gene Clinical Trials Clinical Trials 59