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.

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Gene Editing Gene Gene Therapy In-Vivo 189

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. There were many impactful insights shared from the several sessions and amazing conversations at the #WeekInRare, but the top themes I took away are: 1.

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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

XTalks

FEBRUARY 27, 2024

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. Both therapies were awarded their approvals on the same day in December 2023.

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Research Drugs Gene Therapy Clinical Trials 59

XTalks

SEPTEMBER 20, 2024

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. So that’s really exciting,” Dr. Siciliano explained.

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Gene Editing Manufacturing Gene In-Vivo 52

WCG Clinical

OCTOBER 21, 2024

In a recent episode of WCG Talks Trials, WCG experts explored topics like the importance of community engagement, the latest developments in gene and cell therapies, and the challenges of clinical research in rare and chronic diseases like sickle cell disease and melanoma.

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Clinical Trials Clinical Trials Trials Clinical Research 52

Worldwide Clinical Trials

MAY 30, 2024

This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications. For a more in-depth discussion on challenges and solutions to ALS research, read our blog.

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Gene Clinical Trials Clinical Trials Gene Therapy 204