More commercial approvals would certainly help. And yes, initially a limited patient population, but another indication for Sickle Cell Disease could greatly increase the patient pool. I’m not even sure if Cryoport is actually being used for this treatment, but I do recall some slides of the Northstar 2 trial from BLUE that showed cryopreservation was part of the manufacturing process for Lentiglobin.
From Motley Fool:
Zynteglo: Positive opinion
Until recently, this experimental therapy has been known as LentiGlobin because it uses a lentivirus to insert functional hemoglobin genes into stem cells that produce new blood cells. This treatment could be used for patients with transfusion-dependent thalassemia (TDT) and sickle cell disease (SCD). Both of these conditions are caused by dysfunctional genes for hemoglobin, the protein in red blood cells that carries oxygen to tissues that need it.
Recently, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency recommended conditional approval of Zynteglo for TDT patients who can't find a matched stem cell donor. If the European Commission follows CHMP's opinion, as it usually does, this will be the first available treatment for TDT besides frequent blood transfusions, which are painful, inconvenient, and expensive.
If approved in the EU in the first half of 2019, and the U.S. in 2021, annual Zynteglo sales could reach $1.12 billion in 2023. Partly because it looked extremely effective in the Northstar-2 study, where 10 out of 11 treated patients had stopped receiving blood transfusions and had hemoglobin levels up near the normal range after three months.
Around 60,000 people inherit some form of beta-thalassemia, and a large portion of this population could be eligible for treatment with Zynteglo. That's probably enough to drive blockbuster sales on its own, and approval for SCD down the line could push this gene therapy even further. In the U.S. alone, there are about 100,000 people living with SCD, and perhaps a million worldwide that could use a treatment option. Misformed hemoglobin causes red blood cells to flatten out in a way that isn't helpful. Sickle-shaped blood cells get stuck in blood vessels, effectively strangling the organs they service and causing a great deal of pain along the way.
