Last week Bluebird bio unveiled to investors commercialisation plans for its portfolio of promising gene therapies to treat severe genetic diseases and cancer.
The company presented its initial country-by-country launch plans for ZYNTEGLO, the first gene therapy for transfusion-dependent β-thalassemia (TDT) which gained positive CHMP approval in EU earlier this year.
The issue is that it did not include many countries - and definitely not Australia.
The launch plans for TDT "will lay the foundation for future launches" said the company.
ZYNTEGLO is expected to launch in Germany in the second half of this year. Launches are planned for Italy, France and the UK in 2020, followed by wider expansion across the EU. ZYNTEGLO will be submitted to the FDA by the end of this year in advance of the planned US launch.
There are potentially three additional gene products planned for regulatory submissions in 2022.
Yet there are no current public plans to bring these potentially life-changing therapies to Australian patients.
Headquartered in Massachusetts, with a market capitalisation of over AUD$10 billion, Bluebird bio has emerged as one of the world's leading gene therapy companies.
The company is currently developing therapies for a range of rare diseases and cancers, including cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.
It has many more therapies in its research pipeline for diseases including a diffuse large B-cell lymphoma candidate. Their preclinical program for the DUAL B cell CAR builds on the knowledge from the current generation of CD19 specific CAR-T therapies such as KYMRIAH, YESCARTA and JCAR17.
The Bluebird next generation CAR targets two antigens instead of one. It is designed to enhance T cell activation and gene editing for potential potency and durability enhancements, all in a single therapy.
Dr Amanda Ruth