Interview: Bringing Biotech Spirit to a Non-Profit Environment

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As part of a series of interviews conducted with leading industry experts in the run up to the World Orphan Drug Congress in Geneva, Total Orphan Drugs was delighted to sit down with Frédéric Revah, Ph.D, CEO of Généthon Total Orphan Drugs would like to take this opportunity to thank Frédéric Revah for speaking to us.

Bringing Biotech Spirit to a Non-Profit Environment: Interview with Frédéric Revah, Ph.D, CEO of Généthon

Patient input informs innovation in developing gene therapies for rare diseases according to Dr. Frédéric Revah, Ph.D, Généthon CEO

In 1990, L’Association française contre les myopathies (AFM), France’s largest patient organization funding muscular dystrophy research, made a political decision; rather than only funding existing research, it would build a R&D powerhouse of its own. AFM spun off Généthon, a non-profit biotech with the mandate of developing therapies for rare diseases with methodologies that could be generically applied to other disorders. Généthon receives over 80% of its funding from AFM’s very successful annual telethon, which last year raised over 88 million Euros.

“We are looking at very severe, very life threatening diseases where we think that the proof of concept is doable from the technology point of view.” said Dr. Frédéric Revah, Ph.D., Généthon CEO. A starting point is whether a technology will enable able to delivery of the right gene in the right organ. Whether a clinical trial can be conducted with clear outcome measures to demonstrate efficacy in a clear-cut way, given that cohorts are very small.

“Orphan drugs are specific, it is not only the numbers of patients, but the technologies are specific.” said Revah. “If you want to be curative, if you want to have important benefits for the patient, you really have go to technologies at the cutting edge of innovation. So you have to think about gene therapy, cell therapy, things such as exon-skipping and stop-codon readthrough. You really have to get deep into innovation.”

Généthon embarked on progressively building an infrastructure of knowhow, creating strategic networks of appropriate academic experts, clinicians and industrial partners. An early player in mapping the human genome (most rare diseases are genetic in origin), Généthon moved on to genomics, and then, in 2000, started designing and developing drugs including vectors for gene therapy. Généthon created the first production center in 2005, and in 2009 launched the first clinical trial. 2014 promises five different clinical trials and a dozen clinical and preclinical projects. Despite AFM being devoted to muscular dystrophy, neuromuscular research makes up only a third of Généthon’s portfolio. The rest includes genetic immune deficiencies, ocular disorders, central nervous system, liver, blood disorders, as well as two current multicenter international trials for immune disorders.

The strategy is to build a sustainable platform. “We are not going to cover seven thousand different pathologies, obviously.” said Revah. “We are addressing the most severe pathologies, and trying to prove the concept to the market that the approach works and is a relevant and sustainable approach for rare diseases.”

The next step is to adapt methodologies for more prevalent disorders. “Whenever you look at gene therapy for rare disorders or disorders of the retina, you think how can I apply this to age-related macular degeneration?” said Revah. “Whenever you go for gene therapy for Huntington’s disease, for instance, the question is how can I apply this therapy to Parkinson’s disease. Whenever you go for gene therapy for rare immune diseases then the next question is how can I apply this to cancer?”

While AFM, the parent organization, is not requiring specific return on investment, it does expect workable drugs. Généthon works closely with timelines. “We are bringing biotech spirit to a non-profit environment.” said Revah. “But more than a business plan, we have a mission.”

1. Inform innovation with feedback from the people who will use it

Généthon research priorities are set by its parent organization, however: “We would not have gotten to where we are now with a full portfolio of products that are in development if we would not have had the patients from the patient organization.” Revah said.

2. Communication must be two-way

To inform setting priorities and objectives for clinical treatment, it is important to know what are the types of risk benefit ratios that patients expect.

3. Build layered networks of expertise

To build an organization of professionals, Généthon has strategically fostered partnerships with pharma and biotech and seeks commitment from industry.

Organization profile

Created in 1990 and funded almost exclusively by the AFM-Telethon, Généthon is a major player in gene therapy for rare diseases. Généthon’s mission is to provide patients with breakthrough treatments for gene therapy. Having played a pioneering role in decoding the human genome (publication of the 1st human genome maps between 1992 and 1996), Généthon is today, with more than 200 scientists, physicians, engineers, regulatory affairs specialists, one of the major international centres for research and preclinical and clinical development of gene therapy for rare diseases.

image_thumb4_thumbSpecial thanks go to Wendy Wolfson who conducted the interview on behalf of Total Orphan Drugs.

Wendy Wolfson covers innovations at the intersection of medicine, science and healthcare as a columnist for Chemistry & Biology, a publication of Cell Press. She has contributed to magazines including Science, Nature Biotechnology, the Lancet, and Red Herring. Her work can be found at wendywolfsondotcom.wordpress.com and she can be contacted at wendywolfson@nasw.org

Read more interviews in this orphan drug series
Partnering from the pharma perspective
How to deliver value to patients
Perspective from patient advocates in Sweden
Interview: Surmounting barriers to patient access
Unique opportunities to develop cures for orphan diseases in the Middle East shouldn’t be overlooked
Where do the current and future challenges lie in bringing new orphan drugs to market?
Bringing Biotech Spirit to a Non-Profit Environment
Paving the way for a whole new medical paradigm
Tailoring your reimbursement strategy, step by step, country by country
An Interview With Pfizer on the Orphan Drug Industry: Part I
An Interview With Pfizer on the Orphan Drug Industry: Part II
An Interview with the Chair of the IRDiRC
An Interview with an Orphan Drug Payer

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