Pharmaceutical Technology

MAY 24, 2023

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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Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. Cell and gene therapies are not your typical clinical supply chain.

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XTalks

APRIL 11, 2024

Concerns of Early Deaths The expanded approvals come after the FDA had flagged reports of early deaths in clinical trials evaluating the therapies in earlier treatment line settings. This prompted the convening of the ODAC advisory committee to weigh in on the concern.

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Drug Discovery World

MARCH 13, 2023

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.

Gene Therapy 52

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The Pharma Data

AUGUST 15, 2021

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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Drug Discovery World

NOVEMBER 3, 2022

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. References .

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Pharmaceutical Technology

MARCH 20, 2023

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%

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