Novartis Gene Therapies
NEWS
New data shows Zolgensma, Novartis’ gene therapy for spinal muscular atrophy, has the potential to be used presymptomatically in juveniles.
Molecular Partners announced yesterday it has filed for a $100 million initial public offering with the Securities and Exchange Commission in the U.S., funding which will go toward supporting the company’s work in the development of protein-based treatments for COVID-19 and various cancers.
The COVID-19 battle dominates the news, but it doesn’t constitute all the news. Here’s what several Southern California Biotech Beach companies are doing that you might have missed.
It was a busy week for clinical trial announcements. Here’s a look.
In a 56-page response to the investigation from the U.S. Food and Drug Administration, Novartis said the Kaspar brothers personally manipulated or instructed others to alter some of the preclinical data that ultimately led to the approval of the $2.1 million priced SMA therapy.
Last week was an unusually busy week for clinical trial news, with numerous companies presenting results at conferences. Here’s a look at the top stories.
While the approval of AveXis’ gene therapy for spinal muscular atrophy has been tainted by the revelation of early data manipulation, the Novartis subsidiary presented some long-term data at a conference that backs up the treatment’s efficacy.
AveXis presented new interim data from the Phase III SPR1NT trial of Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1.
The Midwestern region of the United States is often referred to as “flyover country” by residents of both coasts as a way to diminish or dismiss the importance of the states that make up this part of the nation. But the Midwest can be a great place to live and raise a family.
JOBS
IN THE PRESS