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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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A supply chain to match the changing face of science 

Drug Discovery World

1 Emily was a seven-year-old girl who relapsed a second time following two years battling leukaemia, out of standard treatment options, and her life was saved by a CAR-T treatment in early clinical trial. This change in science demands a change in the supply chain to match. 15 years later .

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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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Overcoming inefficiencies to improve access to cell and gene therapy 

Drug Discovery World

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. Drug discovery researchers usually partner with a CDMO to provide support through the development, manufacturing, and clinical supply required to bring a drug to market.

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Africa: Unleashing the potential of the new superpower in clinical trials

Pharmaceutical Technology

Africa has considerable advantages and opportunities for trialling investigational medicinal products that, if pharma companies can correctly harness, will turn the continent into a superpower for clinical trials. Clinical trials data for Africa Globally, research has found that 62.2% being African American, 3.4%