Summary
- As a leading gene therapy innovator, Regenxbio is undergoing many strong fundamental developments.
- Contrary to market pessimism, my forecast for Zolgensma approval came true. Two patient deaths in STRIVE are unrelated to the drug. Zolgensma essentially cures spinal muscular atrophy Type 1.
- The aforesaid approval validated the efficacy and safety of Regenxbio platform technology, NAV.
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In the world of securities, courage becomes the supreme virtue after adequate knowledge and a tested judgment are at hand. - Benjamin Graham (The Father of Value Investing)
Though there are different approaches to a successful investment, I leverage on one that is tailored specifically for the bioscience sector. In my experience, the combination of value and growth works optimally for young bioscience innovators. Using this framework, I stumbled upon an investment gem known as Regenxbio (RGNX). As a leader in the lucrative gene therapy niche, Regenxbio is enjoying the first fruit of success. The gene therapy Zolgensma that employs Regenxbio platform technology NAV is recently FDA approved. Now that Zolgensma got the nod, there is more evidence that gene therapy is certainly here to stay. The Luxturna approval, as I elucidated on in the research on Sparks Therapeutics (ONCE), was simply icing on the cake. It is interesting that Sparks was later acquired by Roche (OTCQX:RHHBY). Perhaps, Regenxbio will also be bought out. Be that as it may, I'm not betting the barn on an acquisition. More importantly, the aforesaid approval substantially deleveraged the investment risks for all pipeline assets.
