A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.
In a move aimed at incentivizing companies to go and stay public, the U.S. SEC proposed two rulemakings May 19 as a foundation to the agency’s Make IPOs Great Again agenda.
Regulatory snapshots for biopharma and med tech, including global submissions and approvals, and other regulatory decisions and designations: Accord Biopharma, Acist, Astrazeneca, Boehringer Ingelheim, Daiichi, Genentech, GSK, H. Lundbeck, Krystal, Natera, Vertanical, Viatris.
Biopharma and med-tech happenings, including deals and partnerships, and other news in brief: Amgen, Artivion, Bioatla, Context, Endospan, Ferring, Immedica, Immunitybio, Takeda.
New hires and promotions in the biopharma and med-tech industries, including: Byondis, Cytomx, Juniper, Kyverna, Novalgen, Novastra, Orca Bio, Sensorion, Triana, Tulyp, Turn, Vahaticor.
Eli Lilly and Co. lost its bid to have the U.S. Supreme Court strike down the whistleblower provisions in the False Claims Act (FCA) as unconstitutional.
It’s back to the drawing board for the U.S. CDC’s Advisory Committee on Immunization Practices (ACIP). After a year of Health and Human Services Secretary Robert Kennedy gutting the panel and restocking it mostly with people who share his views on vaccines, the CDC published a notice in the May 19 Federal Register saying it’s withdrawing the amended ACIP charter renewal issued April 6 and is instead “re-establishing” the committee.
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