Can the gene and cell therapy revolution still scale up today?

Gene cell therapies CRISPR pharma market

Over the last few years, gene and cell therapies are turning into a powerful engine of value creation for pharma companies. Their development and commercialization represent a huge potential for the future of the industry. These novel therapies contribute in fueling the global growth of the market, where total prescription sales are expected to be around $1.2 trillion within five years. This field raised more than $3.8 billion during the first few months of 2018, which represents a 135% increase from the same period of 2017. Thanks to these incredible stats, gene therapy has risen to second place in drug development, beating antibodies and positioning itself just behind traditional New Chemical Entities (NCEs).

Between many gene editing techniques, a particular DNA sequence called clustered regularly interspaced short palindromic repeats (CRISPRs) has created more enthusiasm than others. If you don’t know what this technique is, in a word, bacteria use CRISPR-associated system (Cas) proteins to fight viruses by cutting up their DNA.

CRISPR has very quickly been labelled “the biggest biotech discovery of the century” and “a seismic shift” in medicine. Although not a completely “new discovery”, actually “Crisper-Cas9” was elected scientific innovation of the Year 2015 by Science magazine. This genetic engineering tool has opened the door to a cheaper, simpler and faster DNA manipulation, that’s making it viewed as the top gene editing method.

Since 2017, specialists considering that gene therapies are the future of healthcare and the hottest innovation of our time. They are excepting to reinvent or revolutionize the health industry. Last year however, events have shown that our societies seem unprepared to confront the Transhumanist hopes that the manipulation of the genome carries in it.


Gene cell therapies CRISPR pharma market

Between ethical debate and huge milestone advances.

Speaking of reluctance, let’s make a quick point to what’s recently happened. Everyone in the industry heard about the terrific news that the Chinese scientist He Jiankui, who reportedly used the gene-editing technique on babies who were brought to term. This news has opened an ethical debate around the CRISPR technique involving safety question, some have given the possibility that gene-editing technology may cause cancer by off-target effects issues, and of course, some ethical consideration about the possible use of gene editing in biological attacks or even more simply on the “legitimacy” of editing human life.

The news was not readily approved by the majority of the experts in the field, unfortunately hit the share value and the reputation of all the biotechs involved. This could all be regarded as a waste knowing that at the same time, organizations in the development of legitimate therapeutic applications of the technology attained significant steps during 2018. Among them, Editas, which just lost its CEO and President Katrine Bosley, this only a few months after getting the all-clear to start its first CRISPR trial by the FDA. Secondy, it doesn’t help that biotech’s shares are facing the first breakdown due to the “babies-case” crisis in the world. Even with these unfortunate downturns, the company is still well positioned on the market thanks to this FDA’s green light for their IND application, a CRISPR-based treatment for LCA10, a rare form of blindness. The organization also signed a $25 million milestone payment from partner Allergan for hitting this early milestone.


“The team at Editas Medicine is making the future of medicine a reality. I’m very proud of them and all they have accomplished. I know they will keep driving forward to make unprecedented medicines to help people with serious diseases, and ones that may truly change patients’ lives. It has been a privilege to be part of Editas Medicine and to help pioneer this field, and I look forward to their continued success.”

Katrine Bosley, Editas Medecine’s CEO.


CRISPR a bright future in the industry


Apart from these incidents, CRISPR still has a bright future into the industry. We still consider it as a trend on the health market, especially since last year’s approval from the FDA for the first gene therapy for a disease caused by mutations in a specific gene. The treatment is called Luxturna, which was developed by Spark Therapeutics to fight Leber’s congenital amaurosis, a form of inherited blindness. It is the first therapy in which DNA-filled viruses are inserted directly in the body, and now it’s the first one to be commercialized.

“I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses. We’re at a turning point when it comes to this novel form of therapy.” Scott Gottlieb FDA Commissioner.

According to the business consulting firm Grand View Research, CRISPR market can reach a value of over $4bn by 2025.

Gene cell therapies CRISPR pharma market


“There is probably no large biotech or pharma company out there anymore, who have their own R&D, that are not using CRISPR. They are all using CRISPR in their labs.”

Dr Rodger Novak, CEO of start-up CRISPR Therapeutics


Naturally, with such a potential, major drugmakers have developed a big interest in gene and cell therapies. Here are some of the pharma giants which are involved in the field: Novartis, Pfizer, Gilead, Vertex, Celgene, Sanofi, Merck, Bayer & Roche. All these players know that this kind of treatment is a game changer, that it will carry with it profound changes for the sector in the near future, and of course, they wouldn’t miss the opportunity for anything. Some other names in the insudtry are starting to position themselves very strategically in the field, like CRISPR Therapeutics and Sangamo, two companies with the strongest presence in the medias, according to Commetric analyze.

A long patent battle currently taking place over the owndership rights to CRISPR, between biotechs to determine who will take the lead on “the next big thing”. Companies that are working on CRIPSR must use high precision tools to identify their competitors, strengths and weaknesses, like Orbit BioSequence, designed by Questel, a software which allows you to do sequence searching in a dedicated patent platform.

For now there seems to be no doudt, the gene and cell therapy revolution will still scale up! The market is in a state of continuous expansion, last year a handful of new startups emerged with new tools extracted from the extraordinary diversity of naturally occurring Cas endonucleases in the microbial world. In this complex market landscape, it’s as usual and boils down to innovation. Heavyweight companies acquire or create partnerships with startups or small specialized companies. Real pioneers in the field are small-cap biotechs and thanks to their work, gene-editing tech will soon be everywhere. Lot of Wise investors are betting on CRISPR, and some companies like CRISPR Therapeutics are now strong on the stock market ($2.4 billion). In order to get your Innovation project right on track for this race, it’s a high priority to quickly identify key players in this game, by using a strategy using network and competency mapping, technology scouting and publication analyses. At Innovation Intelligence we recommend the use of the Expernova platform to carry out this strategy.





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