When Negative Reactions to Biotechnology News Mislead. The Case of Regenxbio.

Regenxbio Misleading Selloff

A selloff of Regenxbio (RGNX) stock, following the FDA’s partial hold on the intrathecal administration of AVXS-101, is unwarranted and totally misleading.  


Because the selloff of RGNX gave the impression that the gene therapy Zolgensma, using the adeno-associated virus (AAV) vector , developed by Regenxbio, has failed, which is not true.

The partial hold was put only on the product administered intrathecally in a clinical trial for the treatment of spinal muscular atrophy Type 2 (SMA-2). The agency has not put a hold on the same gene therapy Zolgensma that is already approved and administered intravenously for the treatment of spinal muscular atrophy type 1 (SMA-1).

The Misleading Selloff

Zolgensma, which was called AVXS-101, is an adeno-associated virus vector-based gene therapy belonging to Novartis (NVS) with the vector sublicensed from Regenxbio. The stock selloff has given the impression that Regenxbio’s AAV vector has failed and that its royalty on the sales of Zolgensma could be eroded or erased; this is far from being true.  

Leave a Reply