On the European CGT landscape, zebras and the future of medicine

Alongside industry professionals interested in the cell and gene therapy landscape in Europe, DDW’s Multimedia Editor Megan Thomas was welcomed to Advanced Therapies Europe 2022 by Phacilitate’s Vice President, Becky Johnson-Kent.

Johnson-Kent introduced the notion of the zebra company and compared its two-fold purpose of profitability and societal advancement to the cell and gene therapy industry. This was echoed across this two-day event in London, UK.

What are zebra companies?

The drug discovery industry is familiar with unicorn companies, but what about zebra companies? Johnson-Kent explained: “Zebra companies aim for sustainable growth and place value on coexistence with other players in the field. A zebra is both black and white and in this way a zebra company is both profitable and works to improve society. There is also a play on words in that zebras are known to move in groups, with zebra companies being characterised as mutualistic, tending to band together to support and preserve each other. Rather than butting heads and having a purely competitive relationship, zebra companies cooperate so that their individual inputs come together to produce a greater synergistic product. It struck me that these characteristics are similar to those values that I personally see within the CGT community, a collective drive, unity and ambition in order to advance the next pillar of medicine.”

CAR-T breakthrough

The first plenary panel discussion was a noteworthy example of the therapy’s real-world potential. After seven-year-old Emily Whitehead was diagnosed with acute lymphoblastic leukaemia 10 years ago, she became resistant to all treatment options available at the time and was subsequently the first paediatric patient to receive CAR-T. May 2022 marked Emily’s tenth year cancer- free, and so the discussion between Tom Whitehead (Emily’s father and the co-founder of the Emily Whitehead Foundation), Dr Stephan Grupp from the Children’s Hospital of Philadelphia who helped save Emily’s life, Novartis’ Amir Hefni and Oxford Biomedica’s James Miskin, was nothing short of inspirational.

Individuals and organisations who have played key roles in shaping the CAR-T space provided insight into the challenges and opportunities within the current UK and European CGT landscape; the shifting regulatory pathways to develop a stronger CGT growth strategy; the opportunities to drive efficiency and reproducibility when manufacturing cellular therapies at scale; the current challenges facing therapy manufacturing automation, and more.

On the exhibition floor and during presentations by company representatives, attendees could learn about and admire the work of companies such as BoneTherapeutics, a cell therapy company in the field of orthopaedics and bone diseases; Rocket Pharma, who is advancing an integrated pipeline of genetic therapies that correct the root cause of complex and rare life-threatening childhood disorders; and Minoryx, a clinical stage biotech developing therapies, namely for orphan diseases of the central nervous system. These are just a few examples of companies embracing both profitability and the betterment of society in the cell and gene therapy sector.

Panellists discussed what they were hoping to achieve a decade ago, what they anticipate for the future, and the inflection points along the way. This included the University of Pennsylvania and Novartis joining forces in 2012, a year after a team of physicians and scientists from Penn’s Abramson Cancer Center published breakthrough results in several chronic lymphocytic leukaemia patients treated with personalised versions of their own immune cells, as well as the genesis of the Oncologic Drugs Advisory Committee in 2017.

Ultimately, one thread was consistent in every discussion: collaboration is a primary driver of exponential growth when it comes to cell and gene therapy. Competition is healthy; it is the reason that we are still investing in innovations and continuously learning over 20 years since the first experimental cell and gene therapy was performed. But ‘we can’t keep reinventing the wheel’, explained more than one speaker. 10 years ago, ATMPs and CAR-T were being called the future of medicine and this will remain the case if, like the zebras who survive in groups, we continue to build on the wealth of research, trials and expertise that have come before.

Volume 23 – Issue 4, Fall 2022

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