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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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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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. g/dL and 45.4%

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pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

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In-Vivo Gene Editing Gene Therapy Gene 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8

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Drugs Gene Clinical Trials Clinical Trials 59