A trial of Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD) has been halted after the death of a young male.
The company announced on 20 December that it had paused screening and dosing in the Phase I study and it...
Spotlight On High Doses Again After Pfizer Duchenne Gene Therapy Death
US Regulator May Call For Limits
Listen to this article
The death in Pfizer’s Phase I study could reflect wider safety issues for AAV-based gene therapy and could spur intervention from regulators in 2022.
Keep up with the top pharma business news of the week. On the go.
More from Business
- • By
The Swiss pharma company is rebalancing the portfolio, emphasizing best-in-class drugs and moving into new areas like cardiovascular and metabolic disease.
- • By
In this week's episode: Trump’s UK drug pricing win; Novo explains semaglutide Alzheimer's rationale; J&J and Novartis on this year’s biggest M&A deals; a look at the mixed performance of CAR-Ts; and Akeso’s goals beyond ivonescimab.
Pipelines in bioconjugates continue to develop, from ADCs to XDCs, with radionuclide drug conjugates (RDCs) and antibody-oligonucleotide conjugates (AOCs) leading the expansion.
![Spotlight On RDCs, AOCs As Leading Novel Bioconjugates[Updated]](https://web.insights.norstella-labs.com/resizer/v2/3M7ZAWZOBNK7NG4A4WDOIFG6DU.jpg?smart=true&auth=392a1fdb3cfacb29445720c412410e598dbcc5f90f14857652cd498bd0faa0fb&width=400&height=225)
More from Scrip
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.
