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Personalised medicines face unique patenting challenges say pharma innovators

With multiple pieces and parts, protecting CAR-T therapies is more like patenting a mobile phone than a single-molecule drug, according to counsel at Celgene and other drug innovators

New CAR-T therapies offer evermore promising ways to fight cancer, but they also present several patenting conundrums, according to in-house counsel.

Industry sources at Celgene and elsewhere say patenting CAR-T products is not like patenting a typical molecule that is wrapped up in an easily swallowed tablet. Personalised therapies, such as CAR-T comprise multiple-steps that involve harnessing a patient’s own cells, modifying them using gene therapy, and then transfecting the cells back into the patient.
Resembling more of an assembly line of petri dishes and lab coats, these new therapies have little in common with drugs past and cannot really be considered to be ‘products’ in the conventional sense.
As such, drug companies are unsure where in the process they should file a patent and how they might determine whether or not the product meets eligibility criteria in places such as the US.
“CAR-T therapy patents are more similar to a cell phone patent portfolio than a typical drug because each part has its own portfolio,” says Peter Cicala, chief patent counsel at Celgene in the US. “We in the pharma and biotech industries are used to thinking of a drug as a unitary entity; that is the thing that has the physiological effect we all want.”
He explains that CAR-T cells’ mode of action is to kill foreign bodies. They are genetically engineered to selectively bind to tumour tissue that they would not naturally bind to without genetic intervention.
The therapy separates out a patient’s white blood cells. These cells are then reprogrammed by introducing a genetic sequence so that the t-cells produce new surface receptors.
“These cells’ magic is to kill foreign bodies. We are giving these t-cells a pair of eyes and feelers that will pop up on the surface of the t-cell so they have special powers to fight the foreign tumour tissue that they were blind to prior to infection,” explains Cicala.
The magic of the treatment is its high success rate. Gilead’s Yescarta had an 80% response rate in clinical trials, with patients experiencing complete or partial remission.
Kymriah by Novartis had similar success rates, with 80% of children using the product going into remission. The clinical trials involved 20 years of research and the reconversion of a US factory.
“We have a mission control,” says the head of IP at a CAR-T therapy innovator, “For every patient we know when they will be coming in and the treatment is scheduled on our production line. The cells are put inside a churn and flown across the globe to be delivered to our facility.”

Innovative patents

In the midst of all the development in the CAR-T space, pharma innovators are finding new places to protect their IP.
“There are plenty of opportunities for IP, but the bigger challenge is the sheer complexity of running a process of this nature. It is not the average thing a person can do.
“You can patent aspects of the production process, aspects of the treatment and how the product is administered to the patient and what dose is administered, and what side-effects you might mitigate,” says the head of IP.
Cicala at Celgene explains: “In the event a patent cannot be obtained on a particular biologic product, there is still a possibility of patenting the method of using such product.”
The competitive battlefield for personalised medicines is very different from single molecule and biologic drugs. The future competitors are other start-ups and innovators who are racing to come up with new ways to perfect the technique.
The IP head explains: “This is not an area where your typical generic or biosimilar is looking to compete; so the conventional mind set of ‘I want protection for as long as possible’ doesn’t hold because this is such a new technology, and the end scenario is not clear.
“What you do with your patent protection and how that ensures your exclusivity is not so clear because it is such a competitive field.”
Because the pace of progress in the CAR-T space is moving so quickly, many of the patents filed today might also be worthless in 10 years. Cicala tells Patent Strategy that this rapid progress of science is one of the big IP challenges for these products.
As one technology replaces another, older patents will not protect the most modern inventions making five-year-old patent portfolios obsolete.
What innovators do care about is finding the golden patent in the CAR-T race that removes the ‘personal’ from personalised medicine.
At the moment, each individual cancer patient receives a therapy using his or her specific blood cells. But innovators are racing to find a technique that can deliver the therapy to a wider audience that bypasses the time it takes to grow the individual t-cells.
“The holy grail would be allogenic CAR-T cells which can be given to any patient,” says Cicala.

101 problems

For those innovator companies with a presence in the US, Section 101 of the US Code poses a huge roadblock in the development of personalised medicines. Because CAR-T involves genetic therapy, which could be considered by examiners and judges to be a law of nature or natural phenomenon, there remains a question about what parts of the procedure are eligible for a patent.
“More clarity around Section 101 would create more certainty around patenting biologic products including CAR-T cells,” says Cicala at Celgene. “There is draft legislation circulating around that attempts to clarify what is patentable subject matter and what is naturally occurring, and what is manmade.”
Innovators can patent a synthetically made gene sequence; but because this technology is new and untested, nobody is quite certain how the patents will hold up in court.
The real concern, according to Cicala, is that pharma innovators could eventually find themselves sued for using different parts of the procedure.
And even if the patents are held up in court, another problem will be the enforcement of their patents. As the science becomes more common, IP holders could find they are have to look in multiple places at once when it comes to enforcing their patents due to the long supply chain it takes to produce the therapy. If the patent is infringed, innovators will be left wondering who they sue along the production process. Doctors and manufacturers may be outside the scope of enforcement.
With patent eligibility laws under review, and the pace of technology advancing so quickly, it is anybody’s guess how the IP protection for personalised medicine will unfold. The only hope is that cancer patients will soon have access to better treatment. 

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