Industry keeps faith in finding solutions to bring stem cell and gene therapies to the masses

Experts from the International Society for Cell and Gene Therapy (ISCT) told The Pharma Letter last month that the last 12 months had been the most important yet for advances in their field.

In reality, a lay onlooker might have guessed as much, as they have been in the limelight as never before since Kymriah (tisagenlecleucel) became the first therapy based on gene transfer technology to win approval from the US Food and Drug Administration (FDA) in August 2017.

That approval in B-cell precursor acute lymphoblastic leukemia (ALL) was swiftly followed by Gilead Sciences’ (Nasdaq: GILD) Yescarta (axicabtagene ciloleucel), another chimeric antigen receptor T (CAR-T) therapy, receiving the nod in certain types of large B-cell lymphoma, an indication where it now faces direct competition from Kymriah after the Novartis (NOVN: VX) drug won a second approval last month.