Remove Clinical Supply Remove Genetics Remove Life Science Remove Regulation
article thumbnail

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. plan a, plan b, … charter flight standby option).

article thumbnail

A supply chain to match the changing face of science 

Drug Discovery World

The number of therapy classes in development has exploded as research has advanced methods for tackling a vast array of genetic conditions and diseases in both rare disease and common indications. This change in science demands a change in the supply chain to match. 15 years later . About the author.

article thumbnail

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.