Pharmaceutical Technology

SEPTEMBER 30, 2022

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. This approach remains logistically complex, which builds the desire for allogeneic treatments that can be manufactured from unrelated donors on a larger scale. But there is that significant challenge.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

Drug Discovery World

JUNE 14, 2023

We have now our lead product in a Phase I/II study, which is a HER-2 directed tag T cell product for patients with HER-2 positive solid tumours. I hope that the communities will give us that chance. On top of that, specifically for cell therapy, the allogeneic piece remains a topic of debate, and that is because there are pros and cons.

Research 52

Research Engineer In-Vivo Production 52

XTalks

SEPTEMBER 27, 2022

Treatment options prior to Skysona were limited to allogeneic hematopoietic stem cell transplant (allo-HSCT), which has a high risk of serious complications including death, particularly in patients without a human leukocyte antigen (HLA)-matched donor. Related: Bluebird’s $2.8M

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

Drug Discovery World

NOVEMBER 9, 2023

It is moderated by chairpeople Ulf Grawunder and Astero Klampatsa, and will cover in vivo CAR T cells, solid tumour targeting, myriad constructs and atypical CARs, non-antibody designs, scalability and engineering advances, and allogeneic CAR T approaches.

Engineer 52

Engineer Antibody Protein In-Vivo 52