RegenxBio Has Good News

RegenxBio License Agreement with Pfizer

RegenxBio (RGNX) announced a license agreement with Pfizer (PFE). Under the terms of the agreement RegenxBio has granted Pfizer a non-exclusive worldwide license, with rights to sublicense, to RegenxBio’s NAV AAV9 vector for the development and commercialization of gene therapies for Friedreich’s ataxia (FA). The most common hereditary ataxia (neurological lack of muscle coordination).

RegenxBio will receive an upfront payment and has the potential to receive ongoing fees, development and commercial milestone payments, and royalties on net sales of products incorporating the licensed intellectual property.

RegenxBio’s President and CEO 

Kenneth T. Mills, RegenxBio’s President and Chief Executive Officer said that the license agreement further validates the strength of the firm’s  intellectual property portfolio and the potential of systemic and central nervous system (CNS) manifestations of movement disorders. He hailed the relationship with Pfizer as it is expected to advance this program to develop a potential gene therapy treatment for Friedreich’s ataxia.

About Friedreich’s Ataxia

Friedreich’s ataxia  is the most common hereditary ataxia. FA patients have a genetic mutation in the FXN gene which limits the production of the protein frataxin; causing debilitating symptoms and complications including loss of coordination and balance, muscle weakness, impaired vision, hearing and speech, scoliosis, diabetes and cardiomyopathy.  

RegenxBio NAV Technology Platform

The NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. RegenxBio and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Prohost Observations

Instead of stock gains the good news seems not to reflect on the RegenxBio stock.  This is not hard to explain as the traders’ strategies are to sell stocks that have recently outperformed and buy those that have recently dived, without having a clue about the firms’ technologies, clinical trials, investigational products, approved products, the diseases they are specialized in treating and the size of their markets.

What attracted us to RegenxBio is its NAV Technology Platform which is described above. Both RegenxBio and its licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates for the treatment of various genetic-derived diseases.

It is important to note that many gene therapy trials have failed in the past few years as a result of using AAV vectors that were neither safe nor effective. Gene therapy passed the test in trials only when improved AAV vectors were used.

RegenxBio in 2009…

In 2009 RegenxBio secured exclusive rights to intellectual property covering novel recombinant adeno-associated viral vectors that were discovered at the University of Pennsylvania in the lab of James Wilson, M.D., Ph.D.  RegenxBio has sublicensed NAV adeno-associated virus 9 (AAV9) vector in 2014, to a firm called Avexis, for the use as gene delivery in this firm’s gene therapy for spinal muscular atrophy and other gene therapies. Novartis acquired Avexis and with it the NAV adeno-associated virus 9 (AAV9).

Recently, the FDA approved Novartis’ spinal muscular atrophy gene therapy product Zolgensma. RegenxBio is entitled to payment and royalty on the sales revenues of Zolgensma. RegenxBio has its own pipeline products and a parallel pipeline product belonging to its AAV licensees. Before adding Pfizer, RegenxBio had 20 licensees. Now, with Pfizer licensing the firm’s AAV vector, the number of licensees has become 21 large and small firms.     

RegenxBio is one of our favorite gene therapy biotech firms.

To read more about these firms please run a search from our website by clicking here.


  1. Janice Willett August 1, 2019
    • Prohost August 5, 2019

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