Gene Therapy: Commercial Opportunities and Challenges for Pharma

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September 2018
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As gene therapies for major diseases accelerate from the development pipeline, how can pharma exploit near-term opportunities and protect its business going forward?

As gene therapies for major diseases accelerate from the development pipeline, how can pharma exploit near-term opportunities and protect its business going forward?

As investor money pours into GT development it's time to take stock and Gene Therapy: Commercial Opportunities and Challenges for Pharma reveals the insights and vision of gene therapy experts who identify the real opportunities, challenges and strategies that pharma and biotech must focus on.

Experts scan the gene therapy horizon

  • Significant opportunities exist from the treatment of rare genetic disorders to cancer, CNS conditions and diabetes. What will the landscape look like 5-10 years from now?
  • Beyond haemophilia, what are the implications for the field of immuno-oncology for the treatment of solid tumours with chimaeric antigen receptor (CAR-T) cell therapies?
  • What genomic tools in the pipeline could have a significant impact on gene therapy development in the near future?
  • What innovative commercial models need to be put in place to ensure products are sustainable and accessible even in conditions with large patient populations?
  • What evidence do companies need to present to payers for the successful commercialisation of their gene therapeutics?
  • What role in GT will be played by China, South Korea and Japan?


What GT experts say…

"For the initial commercial products, it was a bit of a challenge trying to address specific medical needs and where stakeholders felt comfortable with the risk benefit. Like any skill, not everything works – that's why you have to work harder to improve. The business model has changed; there are a lot more offerings in CRISPR and TALEN that enable one to deal with the manufacturing aspects. The approval of GSK's Strimvelis and preliminary in vitro results in Duchenne's disease show that we have expanded our technical capabilities, the clinical results are reassuring and products are delivering value. We're at the dawn of gene therapy."
EU Industry Expert

"If you do a pay-for-performance annuity type model, there will be probably a first payment in the order of $400,000 or $500,000, which is not unusual when you think about rare diseases, and then they will command an annual payment. But unlike a chronic disease, where you basically treat a patient for life, in gene therapy, every time you treat a patient, you're depleting your pool of patients; you're living off the new incidents per year, which is typically less than 10 percent of the original pool. And that basically forces you to go into indications like sickle cell, where there's huge numbers of new patients every year. The issue then of course is where do these patients live with sickle cell? Most do not live in the Western developed world so I cannot see how they will gain access to a gene therapy unless it's at a special price."
EU Industry Expert

"We haven't seen anything yet. Venture capital investors have been jumping on the ship and institutional investors are getting in on it now. The valuation of gene therapy companies is already pretty significant, but I think it's still amazing to me to see how slow pharma are at getting on board, just like they were with monoclonal antibodies many years ago… I think the inevitable consequence is going to be that therapeutic areas will shift from one pharma to another just like we saw with the monoclonals when big players like AstraZeneca disappeared after the introduction of Remicade – this may happen again as gene therapies emerge."
EU Industry Expert

What to expect

  • A detailed report exploring through expert interviews and augmented with case studies the future opportunities and challenges gene therapy presents for pharma and the practical strategic considerations that need to be addressed
  • An examination of nine key issues that pharma and GT developers need to understand and respond to
  • 30 targeted questions put to GT experts
  • Their perceptive responses that yielded 37 current insights supported by 93 directly quoted comments

Case Studies included

  • uniQure's Glybera
  • GSK's Strimvelis
  • Spark's Luxturna
  • Voyager Therapeutics' VY-AADC

Expert contributors

This report, based on interviews with four industry experts, will look at the current and future opportunities gene therapies present to the pharma industry. Experts interviewed:

  • Joachim Fruebis, Senior Vice President of Development, Bioverativ, USA. In previous roles he held leadership positions within the R&D organisations at Bayer and Pfizer, where he established project portfolios and led cross-functional global teams advancing programmes from invention to clinical development and commercialisation.
  • Miguel Forte, COO, Zelluna Immunotherapy, and Chair of the Commercialization Committee, International Society of Cellular Therapy (ICST), Norway. Forte has extensive expertise in the regenerative medicine and cell therapy industries, as well as medical and regulatory affairs, ranging from leading early- and late-stage clinical trials to market authorisation and the launch of new biologic products for various indications.
  • Raymond Bartus, consultant, USA whose translational R&D experience extends 30+ years, gained during tenures at major multinational pharmaceutical companies as well as smaller development-stage biotech companies.
  • Sander van Deventer, is the Chief Scientific Officer of uniQure and has had a critical role in the development of the first commercial monoclonal antibody (Remicade; infliximab) and the first gene therapy to be granted market authorisation in the Western world (Glybera).

Why choose FirstWord FutureViews reports?

FirstWord's FutureViews reports analyse in detail significant emerging technology and market trends that pharma executives need to understand if they are to manage the opportunities and challenges that lay ahead. These concise and highly focused reports:

  • Are based on primary research with experts whose knowledge and current experience is proven
  • Present clear expert insights free from secondary source information and spurious observations
  • Include only latest research and content–we don't reuse or recycle content