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Cell and Gene & Therapy Europe in Barcelona, 29-30 September 2015

October 02, 2015

by Nick Bellwood

Cell and Gene Therapy

What a delight! Hats off to Phacilitate and their advisory board for putting together a fascinating and such an energising event! From the chair’s opening remarks to the last session of the two day event, the passion and the mentality that we are “in this together” never waned.

Prof Chris Mason (Chair of Regenerative Medicine and UCL and all-round good guy) opened the conference, and this highlighted the quality of the delegates. His infectious enthusiasm for the field and all the hot topics set the tone. As a veteran in his field, the force of Prof Mason’s belief will have dispelled doubts from of the most hardened cynic that this space will deliver truly revolutionary medicines.

Prof Mason highlighted an ever-increasing interest from Big Pharma – and many players were in town to see what the latest noise was about. Jan Thirkettle (GSK – cell and gene therapy) and Joern-Peter Hallé (Merck Serono – Innovation) presented their respective approaches and demonstrated some of the diversity in how Big Pharma is embracing this field. GSK has invested heavily and built a distinct internal capability. Merck Serono are more hands-off, so their embrace doesn’t become a bear hug! Like all strategies, there’s never a perfect one – but success will be in the execution.

When it came to money, there was little, if any, moaning about the scarcity of available capital. Andrea Chiesi (Holostem & Chiesi Pharmaceutica) pointed out that if you had to pick a time to innovate, the macroeconomic conditions in Europe are hardly ideal at the moment (it’s not stopping him though). This sentiment was slightly askew to Kush Parmar’s (5AM Ventures) advice to “Go BIG with your strategy!” To sidestep the “supersize me” cliché, it is arguably easier to get more for less in the US.

To be fair to Kush, you have to get up early to catch him as the 5AM name suggests. They get in very early with their investments and are prepared to roll up their sleeves to minimise risk. Sara Nunez-Garcia (Sofinnova Partners – VC) heralded a new fund being raised by the Paris based VC firm. So their appetite for risk is unabated, despite murmurs of a bubble in healthcare investment – they are a life science VC after all!

First past the post, UniQure, with their gene therapy product Glybera™, got a pat on the back. They are blazing a trail and helping the whole segment learn what to do and what not to do. One delegate posed the question to a panel: “How do you price a therapy that can cure a patient with one shot?” Most shrugged their shoulders at this point, but in a later session, UniQure answered with a number with six zeros and no decimal point!

Leeza Osipenko (NICE UK) was understandably more cautious about pricing, citing that in the absence of any long term data and insight on potential adverse events, it would be difficult to justify some of the prices that biotech dream of. She also got the biggest laugh of the conference with her analogy that C&GT biotech are like men. Before you find one you like and fall in love with, you see men as all the same. At the moment though it’s like we have just fallen in love and got married; C&GT biotech is the newest, best, most wonderful thing and life couldn’t be better. Only after a few years of marriage you discover that all men are the same after all! Chuckle though we did, don’t be under any illusion that when it comes to pricing, NICE are going to put you through the same gamut of tests as for any other product!

Leeza though was just like everyone else; she wants this field to be successful. She wants to keep an eye on the public purse but she won’t obstruct innovation. If anything, her initiative to engage companies as early as possible is laudable, even if you do have to pay to talk to them. A few thousand upfront for a chat with NICE about your plans for your Cell or Gene therapy product could save you millions later.

Price was a key topic but the issue of cost was very much embedded in the subject of manufacturing. Many of the exhibitors (Terumo BCT, BioCision, Biosafe et al) were pedalling nifty kit to cater for centralised and decentralised options. It’s clear, though, that the likes of Novasep, Miltenyi, MasTHerCcell and Roslin Cells are banking on centralised processes. They intend to continue to serve a market destined to be the next big space for biopharma contract manufacturers.

What characterised the disparate state of manufacturing and logistics in C&GT was the case study presentation by Rachel Griffith (PCI UK). Her company oversaw the logistics, and in this case the QP sign off, for a European-wide clinical trial in cell therapy where the manufacturing took place in the US. An impressive achievement; the detail and complexity was almost mindboggling and everyone agreed there must be a simpler way to do this. No one has the answer just yet though!

All this industry/ tech stuff aside, Alistair Kent (Genetic Alliance UK) must be winner of the “keep it real” award! If there was any doubt why we were all at the conference, he jolted us back into line very nicely. I think Alistair looks rather cuddly (he looks a little like my Dad) but no mistaking he is a fierce and engaging figure. He has dedicated much of his career to the advocacy of patients and their families. His message was “speak to us, the patients and families, and learn from us what we really need”, with the intention of backing us (C&GT biotech). He gave an example of how, when faced with an inevitable loss of motor control, patients understandably want to keep the use of their hands as long as possible. So don’t make the clinical endpoint of a trial a walk-test when these patients would readily spend the rest of their lives in a wheelchair if they were still able to text friends and family on their smart phones…..

To counter Leeza Osipenko, I say not ALL men are the same. In fact some of us are bald too! Cell and Gene therapy may one day give a cure for baldness. But I’m happy to report that given the energy, enthusiasm and focus of all those at the CG&T conference, I will be polishing my head for a few more years to come, as the treatments that really matter are what they are working on.

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