Biogen: A Fruitful Collaboration in Gene Therapy. A Small Gene Therapy Firm That You Should Know


Preparing for the Future, Biogen Signs a Lucrative Collaborative Agreements While Highlighting the Small Firm REGENXBIO

Pursuing gene therapy, Biogen (BIIB) announced collaborations with two gene therapy experts from the University of Pennsylvania as well as a gene therapy technology licensing deal with THE small gene therapy firm RegenxBio (RGNX).

The deal will allow Biogen to license RegenxBio proprietary NAV Technology Platform for the treatment of two rare genetic vision disorders. The collaboration agreement will allow Biogen to expand its pipeline of treatments for eye disease.

RegenxBio NAV Technology Platform is an adeno-associated virus (AAV) gene delivery platform with exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10.

Regarding the collaboration with the University of Pennsylvania experts, Biogen selected James Wilson and Jean Bennett to work with on the development of therapeutic approaches that target the eye, skeletal muscle and the central nervous system (CNS). Another aspect of the alliance will focus on validating next-generation gene transfer technology using AAV gene delivery vectors.

The collaboration will also explore the expanded use of genome editing. Biogen believes that the collaborative agreement with the two gene therapy experts will improve Biogen’s approaches for gene therapy treatments.

The agreement, which is valued at up to $2 billion will enable the university harness its gene therapy resources and expertise to develop therapeutic candidates and aid in the development of new manufacturing approaches needed to support commercialization of gene therapy products. Biogen on the other side, will use its drug development capabilities to advance the collaboration programs into the clinic and toward approval.

Biogen will make an upfront payment to Penn of $20 million with an additional $62.5 million committed to fund R&D costs extending over the next three to five years in seven distinct preclinical research and development programs. Each program may trigger milestones that range from $77.5 million to $137.5 million per product in addition to royalties on net sales of products.

Prohost Observations

The agreements with RenenxBio and the University of Pennsylvania is a great decision that demonstrates Biogen’s determination to be well prepared while going into the future. Benefiting from the information at a great academic Pennsylvania center is a wise step forward, which adds to the Biogen’s value. It also increases our faith in Biogen’s determination to become a prime contributor to the evolution of clinical capabilities.


Selecting RegenxBio (RGNX) highlights this Company’s technological capabilities and scientific strength. This firm’s focuses on the recombinant adeno-associated virus (AAV) gene therapy. its proprietary gene delivery NAV Technology Platform consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. RegenxBio develops and commercializes in vivo gene therapy products based on its NAV Technology Platform.

RegenxBio’s near-term strategy is to accomplish its clinical objectives through a combination of internal development efforts and NAV Technology Platform licensees. NAV Technology Platform is currently being applied in the development of 23 product candidates for a variety of diseases, including five internally developed candidates and 18 partnered candidates developed by its licensees.

With the current collaborative agreement, RegenxBio has attracted a top-tier biopharmaceutical ambitious firm. The agreement is great news for this small firm. The stock, which added 3% following the news is expected to give back some now. So, we will continue following up on its news.

RenenxBio has 5 products in clinical trials. Four are almost finishing the preclinical while one product is in Phase I/II clinical trials. All the current revenues are from licensing its NAV Technology Platform. In the current market situation, it is difficult for this stock to become fairly priced based on its technology and prospective future revenues and income.

A possibility of a take over, however, exists. Important to remember that Regenxbio’s gene therapy technology and programs have been selected for licensing by nine drug developers, including Biogen, Baxalta, Voyager, Annapurna Therapeutics, Audentes, lysogene and others.   


Symbol:                                 RGNX

Current Stock Price:         $12.08                        

Market Cap:                         $318.17 M

Total Cash:                           Around $100 M

Net Income applicable         (23,799)

to common shares:

The article about Portola and the VALUE of its Antidote product ANDEXXA for all Factor Xa inhibitors anticoagulants will be posted TONIGHT at 8 PM.  

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