CGTP Symposium Oral

With the significant progress in the development of individualized medicines, several issues with respect to their routine quality testing have emerged…

Potency testing is a critical part of the assessment and release of cell therapy products. These functional assays provide quantitative assessments of the biological activity of a cell or gene therapy product that are associated with that product’s in vivo mechanism(s) of action (MOA)…

Many cell and gene therapy products use special delivery devices. Generally, there are two approaches for development and commercialization. 1: specify a device brand in the label to be used with the cell or gene therapy products…

Gene editing holds the promise to revolutionize medicine due to its potential to correct or disrupt target DNA sequences with a putative long-lasting effect…

The further advance of techniques for genetic modification also necessitates development of novel and more comprehensive genetic characterization tools to identify intended and unintended genetic changes in (subpopulations of) genetically modified cells…

Decentralized manufacturing involves the use of manufacturing units that can be deployed to multiple locations and in cases, to sites close to the patients…

Cell and Gene Therapy programs are progressing from early academic settings to commercial manufacturing at increasingly faster pace and in greater numbers. These therapies are complex, difficult to manufacture in large quantities, and involve many bespoke technologies to make, test and release…

With the recent approval of several Adeno-Associated Virus (AAV)-based gene therapies, the application of AAV gene therapy approaches to the treatment of human disease is expanding…

Bringing treatment to those afflicted with an ultra-rare disease is complex. These products are encumbered with challenges ranging from high development costs with a limited ROI, to issues with executing and interpreting clinical trials with unusually small patient populations…

Allogeneic cell therapies have a potential to revolutionize life-saving Cell and Gene therapy (CGT) field by removing the bottlenecks created by long scheduling delays, lengthy manufacturing times and potential product failures of autologous cell therapies…

Several cell and gene therapy products have received marketing approval from health authorities and, with that, a more complete picture is now emerging about the regulatory CMC requirements to commercialize these products…

Raw material selection decisions early in the development of ATMPs are crucial and can impact the final product quality and ultimately patient safety…

Gene Therapy Drug Development for Ultra-Rare Disease: Challenges & Opportunities

Stem cell-derived medicines have seen somewhat of a renaissance over the last year, following a difficult period in terms of regulatory approval…

Join us for an engaging fireside chat delving into the revolutionary landscape of cell and gene therapy…

The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) brings together regulatory authorities and representatives from the pharmaceutical industry from across the globe to discuss a range of technical and scientific considerations for medicinal…

The field of Cell and Gene Therapy has witnessed expanding numbers of regulatory approvals in recent years…

Genome editing is a tool that enables a new generation of medicines by directly targeting with high precision the genetic cause of a disease. It also allows for immune evasion and prevents microenvironment responses: concepts vital to more potent, off the shelf, cell, and gene therapies…

In addition to the now well-known CAR T (chimeric antigen receptor T cells) and HSC (CD34+ hematopoietic stem cells) cell-based gene therapies, there are several other modes of gene modification as well as cell types already approved and on the horizon…

Measurement of the biological activity is crucial for lot release, stability and any comparability exercise of all biological medicinal products…

Our ability to treat diseases previously untreatable through the delivery of adeno-associated virus (AAV)-based gene therapies has been a tremendous advancement in pharmaceutical development…