What difference does a day make?

Who gets to have gene therapy? And are biotech startups OK? We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast.

Our colleague Jason Mast joins us to explain how the approval of a landmark gene therapy for Duchenne muscular dystrophy set in motion a frantic race to get children treated before their 6th birthdays. Then, HSBC Managing Director Jonathan Norris calls in to discuss why so many biotech startups are facing financial bridges to nowhere.

Listen here.

stem cells

What's the meaning of 'embryo'?

Scientists can gestate human stem cells remarkably well in lab settings, creating what some have dubbed "embryo models" — or more controversially, "synthetic embryos." These new technological advances are blurring the lines between what's considered a viable, maturity-bound cluster of cells, and what's a lab-bound cipher.

The definition of "human embryo" is evolving along with the science, one stembryologist — that is, synthetic embryology expert — told STAT. It "becomes more and more important to think about the embryo not in terms of how it was formed but about the potential it has to generate something."

He and a team of like-minded developmental biologists wrote in Cell that an embryo can earn its title if it has the potential to survive the equivalent of eight weeks into a pregnancy. This contradicts a statement earlier this year from the International Society for Stem Cell Research, which emphasized that lab-forged embryos lacked the developmental potential to grow into humans.

global health

South African court mandates Covid-19 contract transparency

The South African government now has to reveal the details of its procurement contracts for Covid-19 vaccines. Advocates have claimed that authorities overpaid for the vaccines, allowing biopharma companies to make a windfall at the public's expense. The Health Justice Initiative, a South African advocacy group, won a lawsuit against the government — and the resulting ruling found that authorities are "constitutionally obliged to act in an accountable and transparent manner.… It is, in my view, self-evident that there is a public interest in the disclosure of the records."

When the pandemic began, wealthy nations raced to develop, manufacture, and then disseminate vaccines for Covid-19. But low- and middle-income countries were left with insufficient access to the inoculations. This led to a number of deals between governments and pharma companies to secure access to much-needed Covid-19 supplies — but until now, it has not been clear at what cost.

"Pharmaceutical companies should never be allowed to operate without public scrutiny, particularly in a pandemic," one advocate said. "But in South Africa and many other countries, governments were forced to sign up to strict secrecy clauses for their populations to access lifesaving vaccines and medicines. This landmark decision shows that the public can take on powerful pharmaceutical companies and win."

middlemen

U.S. court strikes down Oklahoma PBM law

Oklahoma designed a law to try and rein in pharmaceutical benefit managers, but a U.S. appeals court shot down key portions of it. A PBM trade group sued to block the 2019 law, saying the state's efforts to regulate these pharmaceutical middlemen impeded insurers' ability to provide more affordable choices for patients.

"Oklahoma's network restrictions do more than increase costs" for PBMs, the court wrote, saying they target pharmacy networks, and prevented basic things like preferred pharmacies, mail-order pharmacies, and specialty pharmacies. This is a real win for PBMs, which have been under increasing scrutiny for allegedly driving up consumer costs.