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Cell and gene therapy will be top industry trend for pharma in 2024

BioPharma Reporter

Healthcare industry professionals scored cell and gene therapy (CGT) as the industry trend to have the greatest impact on the pharmaceutical industry in 2024, in a recent survey launched by data and analytics firm GlobalData.

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Cell and gene therapies: why advanced medicines call for specialised logistics

Pharmaceutical Technology

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research. Securing the supply chain.

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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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Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Pharmaceutical Technology

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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QN-023a by Hangzhou Qihan Biotechnology for Relapsed Acute Myeloid Leukemia: Likelihood of Approval

Pharmaceutical Technology

According to GlobalData, Phase I drugs for Relapsed Acute Myeloid Leukemia have a 71% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how QN-023a’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks.

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UniQure sells part of Hemgenix royalty rights in deal worth up to $400 million

Pharmaceutical Technology

UniQure is selling a part of its royalty rights to the haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) to HealthCare Royalty and Sagard Healthcare for a gross purchase price of up to $400 million dollars. HealthCare Royalty is a royalty acquisition company. The gene therapy was given a list price of $3.5

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QN-019a by Hangzhou Qihan Biotechnology for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia): Likelihood of Approval

Pharmaceutical Technology

According to GlobalData, Phase I drugs for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia) have a 90% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. Hangzhou Qihan Biotechnology overview Hangzhou Qihan Biotechnology is a healthcare company. Buy the report here.