Each month we profile an innovative, growing company from across The Golden Triangle of Cambridge, London and Oxford. This month we speak to Mark Rothera, CEO and President of Orchard Therapeutics.

Tell us about Orchard Therapeutics?

Very simply, we transform the lives of patients with rare diseases with innovative gene therapy. We’re a biotech company that was formed in 2015 and which entered into foundational partnerships with leading research centres in gene therapy in the UK and the US.

In Europe, rare disease affects around 35m people. Our programmes use ex-vivo stem cell gene therapy to restore normal gene function in serious and life-threatening inherited disorders. We are most well-known for our work for children born without a functioning immune system in a condition called ADA-SCID, also known as bubble babies. ADA-SCID is a life-threatening disease. Of the 53 patients we have treated so far 100% have survived, with the longest follow-up at five and a half years. I worked in industry for 28 years and I have launched many therapies, but I have never seen the outcomes that gene therapy can deliver.

You were formed off the back of partnerships with leading research centres, how did this happen?

We have well-established collaborations with academic institutions pioneering gene therapy research, including at University College London and Great Ormond Street Hospital for Children NHS Foundation Trust and The University of Manchester in the UK and Boston Children’s Hospital and the University of California Los Angeles in the US. Years of research, together with an initial investment from F-Prime Capital, meant that we could accelerate our programmes to bring life-changing treatments for patients.

How does the merging of academia and industry help to push forward your innovations?

The research centres have strong expertise in ex-vivo gene therapy, but have come up against problems when trying to translate their research into registrational clinical trials and through to commercialisation. It works for us to join together as we each bring a different set of capabilities which is fundamental for getting the fruits of science to patients as approved therapies.

We aspire to set a standard for how well academia and industry can work together. Children are dying in their early life because of these diseases, so time really matters. We work hard to manage that timeline. One of the challenges is to start early, whilst being aware of standards and regulations required.

You have just closed a Series B funding round for a large amount, led by a consortium of UK, US and Asian investors – why do you think you have been so successful?

We have just completed a very significant Series B fundraise of $110m. The reason for this is that investors are very aware of the transformative nature of our treatments. Investors are keen on disruptive technology and we are at the forefront of this wave of gene therapy.

We are a UK-based and established company and we are leading significant programmes, demonstrating that you can translate medicine coming out of academia. My advice is always to have a bold vision and to seek investors and management with a similar vision to help realise that potential.

Does this demonstrate that the UK is still open for business?

I honestly don’t think the Brexit conversation even featured. Our investors are from across Asia, USA and Europe and everyone is excited about the merging of technology and healthcare and our treatments. We are a global, fully-integrated biotech company specialising in rare diseases. The vision is global and is not linked to political issues. Of note, we currently have many positions open for recruitment at our London office.

What is next on the cards for Orchard Therapeutics?

Our development programmes focus on restoring normal gene function in primary immune deficiencies and inherited metabolic diseases. Our lead program in ADA-SCID (OTL-101) is in late stage development and anticipated to progress to a file with the FDA in 2018. We have just announced a collaboration with Genethon for a clinical-stage program in a second immune deficiency disorder, X-CGD (X-linked chronic granulomatous disease). Our lead program in the inherited metabolic franchise is in Sanfilippo syndrome type IIIA. The next steps for this program are to complete the ongoing pre-clinical development and to open a first-in-man clinical study. We have also recently announced a new collaboration with the University of Manchester for a preclinical program in Sanfilippo syndrome type IIIB. By this time next year, we will have one filed lead program and have a further three clinical stage programs, including in X-CGD, Sanfilippo syndrome type IIIA and in one further indication (yet undisclosed).

You mentioned your work around ADA-SCID, and you have received a rare paediatric disease designation for OTL-101, your gene therapy treatment. What is different about this, compared to GSK’s Strimvelis?

To start with, I really want to acknowledge and honour what GSK and its Italian partners, Fondazione Telethon and Ospedale San Raffaele have accomplished in bringing to market the first gene therapy for ADA-SCID in Europe. They are pioneers who have done an outstanding job in creating a platform and a route to getting approval for gene therapy: they deserve tremendous kudos. We have taken a different approach by using a different vector (a lentiviral vector) and by developing a cryopreserved formulation, which consists of freezing the patient stem cells after they have been genetically-corrected. With this approach, the patients can be treated at a transplant centre in their home country.

And finally, tell us a bit about you?

I’m a natural science graduate and I have a business qualification. The most exciting period of my career has been the last 20 years as I have worked with emerging biotechs, launched seven treatments for rare diseases including Metabolic Disorders, launched the world’s first antibiotic for cystic fibrosis and, also launched the first treatment for Duchenne muscular dystrophy. I love working bringing the fruits of great science to patients and making a huge difference for patients who would otherwise have no options.

Find out more about them here: http://www.orchard-tx.com/