fbpx
Connect with us

Health + Fitness

Eric Leire – Guide to Clinical Trials for Gene Therapies

Dr. Eric Leire, MD, MBA is the CEO and Founder of GenFlow Biosciences. He has an impressive background in biotechnology and gene therapy sector. Furthermore, Eric has a plethora of impressive experience both in the pharmaceutical industry, having held posts at Pfizer, Schering Plough and Pharmacia. He also has found success in academic research, having held research position at Harvard University.

In addition, his career has soared throughout the biotechnology industry, being the CEO of several private and public biotech companies. He is also the inventor of several patents and serves on the board of several biotechnology companies, such as Pherecydes (ALPHE.PA), Immunethep, Inhatarget and BSIM.  Academically, Eric holds an MD from Grenoble University and an MBA from HEC, Paris and Kellogg, Northwestern University.

In recent years, biotechnology startups have sprouted alongside computer technology companies in Silicon Valley. The aim of most is to use biotechnology processes to create breakthrough drugs. According to some experts’ projections, by 2026, medical biotechnology alone will hit $500 billion. Given the potential value and return on investment, research and clinical trials are extremely important.

We chatted to Eric about the key features of Clinical Trials for Gene therapies.

To summarise, Gene therapy is the delivery of working copies of a desirable gene to the cells that need it. However, our cells have evolved mechanisms to prevent such intrusion of foreign molecules. To overcome this barrier we use vectors to carry the new gene into the cell, whilst shielding it from the mechanisms designed to prevent this happening.

The number of clinical trials for gene therapies is increasing dramatically year on year, with the UK being somewhat of a hotspot. Almost 1,000 cell and gene therapy clinical trials are on-going today, most of them in early phase.  The clinical trials conducted with gene therapies produce their own idiosyncrasies with very different challenges than the ones met in usual clinical trials. In general, clinical trials conducted to assess gene therapies are more difficult to justify, to design, to implement and to interpret.

First, because animals have similar but somewhat different sets of genes than humans, the animal models may not be fully predictive. Therefore, the investigators may face a more difficult task to document and justify the rationale for the human clinical trials.

The gene therapy clinical trials are also more difficult to design.  The trial design has, for example, to take into account new issues such as the usually smaller size of the cohort of patients which forces the investigators to use different statistical methods to interpret the data.  With the necessity of longer follow up for gene therapies, defining the duration of the trial may also become an issue.  Old rules for clinical trials design no longer apply.  For example, comparative, randomized double blind trials are now considered the gold standard.

But several factors render difficult the use of this gold standard in clinical trial for gene therapies.  With gene therapies, comparators are often not available or not ethically justifiable.  The use of a placebo as a comparator may be hard to implement in a gene therapy trial.  Also, the blinding of patients and investigators may be unfeasible with gene therapies.  In addition, the design of the dose finding part of the trial has to be more sophisticated with often more limited pharmacokinetic and pharmacodynamic data available due to the lack of trial in healthy volunteers.

Also, the investigators must pay extra attention to logistics feasibility and processes. Logistic issues may influence the gene product and therefore significantly impact the trial results.

Finally, Clinical trials for gene therapies are also more complex to interpret.  In ‘classical’ clinical trials, investigators used to assess the relationship between doses and effects, but for gene therapies the investigators now have to evaluate the more complex correlation between gene expression, pharmacodynamic data, bio-distribution and functional parameters.

In spite of all these challenges, gene therapies hold promise as treatments for previously untreatable and high-burden diseases. And with such high expectations, the health authorities have offered high-quality guidance documents for the conduct of gene therapy clinical trials.  In general, the regulators are open to discussion on new gene therapies as long as the approach has scientific merit, and does not improperly increase the risk to the patient.

Therefore, early and repeated engagements with regulatory authorities are key success factors for a gene therapy company such as Genflow Biosciences.

 

Lewis Schenk is an Australia-based Entrepreneur, Writer & PR Consultant. His work has been seen in Entrepreneur Magazine, International Business Times, Addicted2Success & The Good Men Project. Lewis is passionate about sharing empowering stories from people around the world, particularly those in entrepreneurship & business. He has been privileged to work with over 600 businesses and entrepreneurs, helping them amplify their message and build their brand & online presence.

Advertisement defi

Join Disrupt Magazine

defi
Become A Disrupt Contributor

Most Disruptive

Social Media5 days ago

Khaby Lame Becomes The Most Followed TikToker in The World

Khabane Lame was a machine operator but now trending and just became a star from an account he created on...

Tiger_Shroff Tiger_Shroff
Entertainment5 days ago

Meet Bollywood Star Tiger Shroff

Tiger Shroff has a long history in the film industry, starting before his birth. With prominent parents in the industry...

Amanda Bernardo Amanda Bernardo
Women Who Disrupt6 days ago

Meet “The Pizza Princess” Amanda Bernardo

If you live in or near New Haven, Connecticut, you may be familiar with the hidden gem that is “New...

Shirley_Setia Shirley_Setia
Women Who Disrupt6 days ago

Meet Upcoming Bollywood Star Shirley Setia

Shirley Setia is the little Kiwi that could. Coming out of seemingly nowhere with a hit cover on YouTube, she...

two people shaking hands two people shaking hands
Diversity in Tech1 week ago

How the Best Leaders Create and Build Trust in a Modern Workplace

Being a leader in any setting can be challenging but within professional settings, you really are juggling a lot at...

Jay Majors Jay Majors
Executive Voice3 weeks ago

How Jay Majors Is Disrupting The Barber Industry

When we see a successful figure in any industry, learning their story, where they came from, and how they’ve struggled...

Daymond JOhn Daymond JOhn
Executive Voice3 weeks ago

Daymond John Can’t be Stopped

Perhaps you know Daymond John from the wildly popular show Shark Tank. Or maybe you’re familiar with his revolutionary clothing...

Finance3 weeks ago

Ryan Carroll Establishes Wealth Assistants to Serve as a Launching Pad for Hopefuls Worldwide

Countless highly accomplished personalities and established authorities have proven that the pursuit of one’s passion can be a way to...

Business4 weeks ago

Social Media Star, TheBlondeJon’s Journey from Homelessness to Multimillionaire Status

A strong self-belief in oneself is a powerful enabler no matter the obstacles or challenges one faces on a journey....

Entrepreneurship4 weeks ago

Skylar Domine on His Bold Moves in the Entrepreneurial Realm and Overarching Mission to Help People and Communities

Many of today’s powerhouses managed to gain a solid foothold in the world of business and stand out in an...

Trending

Copyright © 2020 Disrupt ™ Magazine - Disrupt is a Minority Owned Privately Held Company

Disrupt ™ is the voice of Latino entrepreneurs around the world. We are part of a global movement to increase diversity in the technology industry and we are focused on using entrepreneurship to grow new economies in underserved communities around the world. We enable millennials to become what they want to become in life by learning new skills and leveraging the power of the digital economy. We are living proof that all you need to succeed in this new economy is a landing page and a dream. Disrupt tells the stories of the world top entrepreneurs, developers, creators, and digital marketers and help empower them to teach others the skills they used to grow their careers, chase their passions and create financial freedom for themselves, their families, and their lives, all while living out their true purpose. We recognize the fact that most young people are opting to skip college in exchange for entrepreneurship and real-life experience. This Podcast was designed to give them a taste of that.