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Editas, changing course again, looks to partner lead CRISPR therapy

Bio Pharma Dive

The gene editing company will focus on “in vivo” medicines, while seeking to license out or find a development partner for its clinical-stage treatment reni-cel.

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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

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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Ginkgo Bioworks partners with WARF for new GD2 CAR T-cell therapies

Pharmaceutical Technology

WARF is the patenting and licensing organisation for the University of Wisconsin–Madison in the US. All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

In-Vivo 264
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Editas grabs orphan drug status for sickle cell disease CRISPR therapy

Pharmaceutical Technology

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. On January 19, Editas announced that it entered into a definitive agreement with Shoreline Biosciences for the licensing of its natural killer cell program.

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FDA approves bluebird bio’s Skysona to treat cerebral adrenoleukodystrophy

Pharmaceutical Technology

The regulatory agency carried out the review of the Biologics License Application (BLA) for the therapy under the Priority Review approach. A one-time gene therapy, Skysona leverages ex-vivo transduction with the Lenti-D lentiviral vector for adding the ABCD1 gene’s functional copies into the hematopoietic stem cells of the patient.

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Xcell and aCGT Vector partner on cell and gene therapy development

Pharmaceutical Technology

Pharmaceutical companies Xcell Biosciences (Xcellbio) and aCGT Vector have partnered to expedite the development of cell and gene therapies. The two companies will aim to improve the manufacturing and analytic procedures used to develop personalised cell and gene therapies to treat cancer patients.

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JPM: Precision Bio continues offload of CAR-T rights

pharmaphorum

Precision BioSciences licenses non-cancer uses of its allogeneic CAR-T therapy azer-cel to TG Therapeutics as it pivots to in vivo applications of its gene-editing technology