Gene therapy
An early stumble for a Doudna-founded startup
Aurora Therapeutics, a startup focused on building personalized gene-editing drugs, has scrapped its lead program and slashed staff, just seven months after it launched, my colleagues exclusively report.
Co-founded by Nobel Prize-winning chemist Jennifer Doudna, the company initially focused on the liver disease phenylketonuria (PKU). But Aurora appears to have been undercut by Beam Therapeutics, which is already advancing its own set of custom gene editors for the exact same disease.
Aurora was one of several groups that cropped up in the past year to develop custom drugs for individual mutations, influenced by the success of Baby KJ's personalized gene-editing drug.
Read more.
pricing
Orphan drugs should not be exempt from price controls, two analyses say
The drug industry has long argued that orphan drugs for rare diseases should not be subject to pricing policies, so as to not further disincentivize the development of such treatments. But two recent studies both argue that's a bad idea.
Orphan drugs are currently exempt from the Medicare negotiation program established by the Inflation Reduction Act. One study found that the drugs that qualify for exemption actually had lower trial costs and comparable revenues as drugs that don't qualify. Those rare disease drugs also recovered costs more quickly. Read more.
Separately, the industry is lobbying the Trump administration to exclude orphan drugs from most-favored nation pilot programs that aim to lower drug prices in the U.S. to levels charged in other rich countries.
The second study finds that excluding rare disease treatments from the pilots would wipe out much of the savings that could be achieved with retail drugs. Read more.
infectious disease
Moderna launches early trial of Ebola mRNA vaccine
From STAT's Helen Branswell: With no sign that the Ebola outbreak in the Democratic Republic of the Congo is abating, the need for vaccines to protect against the Bundibugyo strain is growing increasingly urgent.
To that end, Moderna announced yesterday that it has started a Phase 1 trial of its experimental Bundibugyo vaccine in Canada. A messenger RNA vaccine — should it prove to be effective — could have advantages over the other vaccines in development. As seen in the Covid-19 pandemic, production of this type of vaccine can be scaled up more quickly than that of other types of vaccines.
Preclinical testing and the Phase 1 trial is being supported by the Coalition for Epidemic Preparedness Innovations, or CEPI, which has committed $50 million to this phase of the project. The Phase 1 trial is expected to enroll about 80 participants.
This is the second largest Ebola outbreak on record, with more than 3,800 confirmed cases to date, approximately 1,700 of them fatal.