Spotlight on gene therapy

Gene therapy has the potential to eliminate a number of diseases which are currently causing distress to patients and burden on the healthcare system. Gene therapies have been tested in vitro and were deployed in the clinic for the first time in 2017. The promise of a ‘cure’ has proved tempting for investors and developers alike.

In this spotlight, we will be discussing the current landscape of gene therapy and existing applications. We will review the unique manufacturing requirements of gene therapies and how developers and vendors are supporting this process. Finally, we will hear from leading experts in the field, who will review ethical and regulatory developments that could impact developers and patients.

Featured content

Panel discussion: gene therapy development and manufacture, present and future
Computational fluid dynamics could enable effective and efficient scale up of gene therapy
Addressing the challenges of cell and gene therapy manufacturing in process development: an interview with Behnam Ahmadian Baghbaderani
Process development for successful cell and gene therapy commercialization
GMP-compliant iPSCs show clinical potential

Spotlight highlights

Gene therapy in practice: Current and future uses
Working with gene therapy?
What is gene therapy?
Gene therapy: FAQ
Editor’s highlights: Opportunities and Challenges in Translating Cell and Gene Therapy Products

Expert interviews

On the brink of a treatment revolution: an interview with Steven Gray
Could gene and stem cell therapy meet unmet needs in cancer treatment?: an interview with John Rasko
Overcoming the obstacles to a commercially successful cell or gene therapy: an interview with Dr Nafees Malik
Curing the butterfly children: an interview with Michele De Luca
Performing a gene and cell therapy trial for heart attack patients: an interview with Duncan Stewart

Latest headlines

Cell therapy weekly: Collaboration to progress gene-based cures for HIV and sickle cell disease within a decade
Plant-based molecule found in red wine could improve the efficiency of gene therapy
Cell therapy weekly: Positive results from first liver disease cell therapy trial
Convergence of gene and cell therapy
Gene therapy prevents and reverses corneal blindness in canine model
Gene-edited cell lines could be compatible with 90% of the world
‘Standard’ production platforms can provide viral vector variance
CRISPR scientist claims to have made the world’s first gene edited babies
New treatment for genetic retinal disease is first gene therapy approved in the USA
Gene therapy: evidence, value and affordability in the US health care system
ISCT revises cell and gene therapy forecast following FDA approval of Novartis CAR-T therapy
Could gene therapy win the fight against sickle cell disease?
The commercialization of cell and gene therapies: regulatory challenges
Gene therapy may hold promise for preventing Alzheimer’s progression
Parkinson’s gene therapy trial initiates third cohort
The role of pharmacogenetics and advances in gene therapy in the treatment of diabetic retinopathy
UCLA receives funding for a new trial for gene therapy in X-linked chronic granulomatous disease