The Future of Gene Therapy

Sponsored by: GlobalData

Focused on:

  • Gene Therapy
  • Orphan Diseases

Date: 12 April


Time: 4PM London/11AM New York

Understanding the potential vs caveats

This expert presentation is exclusive only for GlobalData's clients. The webinar recording and presentation will be available on the Intelligence Center. For any requests or questions about this webinar please contact kim.roz@globaldata.com.

Every few years, the pharma industry narrows in on a new buzz therapy area, and this year, it is without a doubt Gene Therapy.

The complex, sophisticated and somewhat “super” therapeutic not only brings precision-based medicine to a more established clinical reality but also edges the industry closer to more targeted benefits for the most difficult-to-treat diseases. Gene therapies have arguably taken the innovation spotlight from other novel therapies, with numerous companies attempting to develop a potential cure for genetic orphan diseases.

On the back of the first FDA approval, in December 2017, of a gene therapy – Spark Therapeutics’ Luxturna for a rare inherited eye disease – GlobalData’s webinar will dive into the complexities of these therapies, what this landmark approval means for future drugs in the pipeline and how gene therapies could potentially shake up current patient access and payer structures.

The global gene therapy market is touted by various industry commentators as a multibillion-dollar market.
Other notable gene therapy developers include Sangamo Therapeutics, BioMarin, Shire, Audentes and bluebird bio.

In particular, gene therapies have been focused on nonmalignant hematology and orphan diseases, and these are expected to generate the most near-term revenue for developers. While niche and under-treated indications offer the greatest blockbuster prospects for developers, these drugs still have gruelling clinical benchmarks to demonstrate significant efficacy that balances with a clean safety profile. The mechanism of using viral vectors -- such as adenoviral vector (AVV) or lentivirus vectors -- to carry and manipulate genetic material, is a risk-ridden approach and developers will continue to face hurdles in generating clinical data worthy of crossing that approval barrier.

However, the biggest stickling point for the future of these therapies is patient access, and how payers will handle these potentially one-time and definitely high-cost therapies, particularly with current insurance structures not equipped to process such unorthodox drugs.

In this webinar, GlobalData’s journalist team dives into the debate among clinical trial investigators, Key Opinion Leaders (KOLs) and other industry experts on development and commercialization hurdles that gene therapies face.

Senior reporter Alaric DeArment and reporter Shuan Sim will discuss what they have been hearing from sources and how experts view recent achievements and future development, commercial and pricing challenges for these specialty therapies. North America editor Surani Fernando will moderate the presentation, followed by a Q&A session.

Presented by

Surani Fernando,

Editor North America

Surani Fernando manages the North America editorial desk. Surani first joined BioPharm Insight, which GlobalData acquired, as a drug development reporter in the London newsroom in 2010 and has generated a number of market-moving scoops and in-depth analyses on key therapeutic areas. She has also reported on drug reimbursement, regulatory affairs, patent litigation, M&A and business financing. Surani has covered academic and industry meetings in both Europe and the US, where she has interviewed key opinion leaders and C-level executives and has also made TV appearances on CNBC and Sky News as an industry commentator. In November 2014, Surani became the Editor of BPI’s European new desk and moved to New York in September 2016 to lead the US team. Prior to her career in journalism she worked in clinical operations at CRO PAREXEL. She has a Bachelor of Medical Science and Master of Health Communication, both from Sydney University, Australia.

Alaric DeArment,

Senior Reporter, North America

Alaric DeArment covers cancer drug development. He served as associate editor of Drug Store News from 2008 to 2014, covering branded and generic drugs from development to distribution, retail and specialty pharmacy and regulatory affairs. In 2011-2012, he edited the book Contestation and Adaption: The Politics of National Identity in China. A native of Seattle, he graduated with honors with a Bachelor’s degree in journalism from Ball State University and also lived in China from 2001-2004.

Shuan Sim,

Reporter, North America

Shuan Sim has a Bachelors degree in linguistics and journalism from New York University. He had previously worked in various trade publications covering technology, precious metals and diamond trade and more. Shuan has worked as a breaking news reporter covering Asia and an international reporter in the Czech Republic. He’s also fluent in Mandarin and proficient in Japanese.

Key Learning Objectives

  • Learn about the major pipeline drugs: imminent trial catalysts and approvals
  • Understand which indications are best suited to gene therapies and why?
  • Insights on the real risk profile
  • Pricing and access hurdles with a one-time high-cost therapy


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