Summary
- Regenxbio is a leading company in the gene therapy space.
- The premier technology platform NAV is validated through Zolgensma's successful commercialization.
- The crown jewel of the pipeline recently generated robust clinical data for the lucrative wet AMD market.
- I do much more than just articles at Integrated BioSci Investing: Members get access to model portfolios, regular updates, a chat room, and more. Get started today »
The successful investor is usually an individual who is inherently interested in business problems. - Phillip Fisher (Warren Buffett's mentor)
One of the hottest emerging areas in all of biotech is the gene therapy space. Using various techniques, companies developing gene therapies are either attempting to enhance or correct a defect in cells. To date, this has been achieved most successfully in the space of chimeric antigen receptor T-cells (CAR T-cells) in cancer therapy, where they've made a huge impact on the standard of care for certain kinds of leukemia.
What most people think of with gene therapy, however, is the delivery of "corrected" genes into the body, hoping to change the course of a disease. Hemophilia is a key recent example, with companies like Spark Therapeutics demonstrating a highly encouraging ability to get the liver to make correct clotting factors. It's been a powerful proof of concept. Now, gene therapies are spidering out into all kinds of different disease areas, and Regenxbio (NASDAQ:RGNX) is looking to be a pioneer. Today, we want to provide an update as to how the company is doing. When last we provided a full report, COVID-19 was not in full effect as a pandemic, and the company had not yet reported quarterly financials in Q1.
Figure 1: Regenxbio chart (Source: StockCharts)
About The Company
As usual, we'll present a brief corporate overview for new investors. If you are familiar with the firm, we suggest that you skip to the subsequent section. Operating out of Rockville, Maryland, Regenxbio is engaged in the innovation and launch of gene-therapy. In doing so, it is poised to serve the unmet needs in retinal, metabolic, and neurodegenerative diseases. We noted in the prior research:
Harnessing the power of its stellar NAV technology platform, Regenxbio delivers a single gene therapy injection to alter the disease course and thereby improves its outcome. As you know, Regenxbio's NAV technology platform is validated after the Zolgensma approval. Hence, it's not surprising that the company attracted over 20 partnerships.
Figure 2: Therapeutic pipeline (Source: Regenxbio)
