PRODUCTVARIOUS (Zolgensma, RGX-121, RGX-111, RP-A501, UX701, DTX301)

The royalty bond is backed by a number of products, both approved and not-yet-approved including Zolgensma, RGX-121, RGX-111, RP-A501, DTX301 and UX701.  A brief description of some of the referenced products is set forth below.

Zolgensma using intravenous delivery is a one-time treatment providing a functional copy of the SMN gene. The product was first approved for the treatment of SMA by FDA in 2019 and has since been approved in more than 50 countries. In January 2025, Novartis announced the Phase III study of Zolgensma using intrathecal delivery had met its primary endpoint.

RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with MPS II. RGX-121 expressed protein is structurally identical to normal I2S. Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. In May 2025, FDA accepted the BLA for review and set a PDUFA date in November. Commercial rights to RGX-121 and RGX-111 were licensed to Nippon Shinyaku.

RP-A501 is a gene therapy being developed by Rocket Pharma for the treatment of Danon Disease and is currently in a Phase 2b pivotal trial. Ultragenyx is developing DTX301, a gene therapy for OTC-deficiency, and UX701, a gene therapy for Wilson disease. DTX301 is in a pivotal Phase 3 program and UX701 is in a multi-phase trial with clinical proof of concept established.

Background

RGNX issued to HCRx, a royalty bond backstopped by the license agreement economics including royalties and most milestone payments for a portfolio of assets including RGX-121, Zolgensma, RP-A501, RGX-111, DTX301 and UX701. REGENX sought to raise non-dilutive capital to extend their runway through a variety of value enhancing milestones and to support the development of their clinical pipeline.