Gene Therapies for Hemophilia. Possibly Curative, Likely Ultraexpensive

September 08, 2022

Three gene therapies in late-stage development are designed to supply working copies of genes that people with hemophilia are missing. They may be one-time, curative treatments, but the seven-figure price tags will be an issue.

Zynteglo May Herald “New Dawn” of Many Seven-Figure Gene Therapies

August 05, 2022

An OptumRx drug pipeline report highlights Zynteglo, a curative gene therapy treatment for beta-thalassemia, and Skysona, a gene therapy for a cerebral adrenoleukodystrophy. FDA approval decision are imminent for both. Price tags of $2 million are expected.

Fourth Shot is Recommended for the Immunocompromised

March 10, 2022

Getting boosted is now the rule, not the exception, when it comes to COVID-19 vaccination recommendations. The CDC now recommends that everyone, ages 12 and older, get a dose of an mRNA COVID-19 vaccine, either Pfizer’s or Moderna’s. For people who have gotten the two-shot Pfizer or Moderna series, the booster is their third jab. For those who got the single-dose Johnson & Johnson vaccine, it is the second.

The Pipeline is Full and Brimming with Promise. Hat Tip to Science.

January 06, 2022

2021 was a year in which investigators, government officials and drug companies overcame immense challenges to develop successful vaccines against COVID-19. That spirit of innovation is expected to carry over into this year.