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Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective 

Pharmaceutical Technology

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Overcoming inefficiencies to improve access to cell and gene therapy 

Drug Discovery World

The cell and gene therapy (CGT) landscape has grown significantly in the past year. Between the 24 therapies already approved by the FDA 1 and a marked increase in clinical trials, widespread accessibility to precision medicine feels within reach.

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VTX-801 RECEIVES U.S. FDA FAST TRACK DESIGNATION FOR THE TREATMENT OF WILSON DISEASE

The Pharma Data

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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A supply chain to match the changing face of science 

Drug Discovery World

Ten years ago, cell and gene therapy (CGT) was really finding its feet as success stories such as that of Emily Whitehead began to hit the news. In early clinical trials, a broad indication therapy may have sufficient patients and supply chain partners locally, but this changes on scale up to commercial approval.