Question

A quote from the article: “Zolgensma is designed to address the genetic root cause of SMA [spinal muscular atrophy] by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion.”

2. A quote from the article: “AveXis has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Genethon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.”

Based on your knowledge of biotechnology patents, explain why AveXis (a Novartis company) has acquired exclusive licenses to use several different patented methods or materials for their gene therapy system.

Answer 1

In quote 2, two types of licensing is mentioned i.e. exclusive licensing and non exclusive licensing. In order to understand the information given above let's first discuss what does exclusive and non exclusive licensing respectively means.

EXCLUSIVE LICENSING:

In exclusive licensing, the patent holder can give the entire ownership and rights of the patented thing to a particular company. By getting a license of the patented thing , the licensee can manufacture more and more products and goods in the market and can have benefit in the economy of itself and the patent holder and also result in the welfare of mankind. In exclusive licensing, licensee does not have the right to tranfer the license to any other company. The name remains the same that given by the patent holder but whole soul responsibility belongs to licensee. There are very minimum chances of infringement.

For example: if any research institute has made any drug for a particular disease but does not have enough money for manufacturing and production, than they can give license to the pharmaceutical companies to manufacture the drug and sell in the market. The benefit is shared on the basis of terms and conditions they signed. Most of the patent holder give license for free without expecting any profit.

NON EXCLUSIVE LICENSING:

in non exclusive licensing, the patent holder gives the license to many companies ( more then one, unlike exclusive licensing). The licensed companies have the entire responsibility of the usage and manufacture. It means all the companies who got the license can exploit the product.

For example: a tyre making company makes a new tyre and gave the license to various car companies to use in their vehicles.

So in the above information, it shows that zolgensma( a medication drug used for the treatment of spinal mascular atrophy) was designed using copy of human SMN gene. It functions by stopping the SMN proteins formation by one time IV infusion. The Avexis company got exclusive licensing from hospital of providing all kind of AAV9 gene therapy related to SMA treatment. They also got exclusive licensing from the research centre REGENXBIO for their recombinant vectors which comes under their patent portfolio to be used in gene therapy and also got exclusive licensing from GENETHON for the I.V. delivery in central nervous system of humans for the treatment of SMA. here Avexis company got exclusive licensing so it is the only company to use the patents. Avexis company also got a non exclusive licensing from the research centre to use its DNA technology for treatment of SMA. Various other companies also got the non exclusive licensing from the research centre to use its DNA technology.

The patent licensing help in the large manufacture of goods, increase the economy, and serves the mankind. Infringement chances are reduced and many other benefits are gained.