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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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J&J’s Carvykti and BMS’ Abecma Win FDA Approvals for Earlier Use in Multiple Myeloma

XTalks

Moreover, rather than producing antibodies, the cancer cells produce abnormal proteins that can cause complications. The companies have also expanded a contract manufacturing agreement with Novartis that will help produce Carvykti for both commercial and clinical supply at the New Jersey plant through the end of 2029.

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Overcoming inefficiencies to improve access to cell and gene therapy 

Drug Discovery World

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. Genetic Engineering & Biotechnology News. August 2020. link] “Going Digital Can Help CDMOs Upgrade Their Value Propositions” Gareth John Macdonald.