Bringing the power of gene therapy to bear on neurological disorders

Neurological disorders continue to be some of the most difficult diseases to treat – Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis (ALS), and so many others. Fortunately, we live in a world where advances in medicine are having a profound impact on humanity.

Gene therapy is an example of the incredible progress being made in medicine and is certain to become an important tool to help solve some of the greatest challenges in healthcare. Targeting the genetic root cause of diseases, these treatments may have the potential to one day cure what is currently incurable. In only the last five years the number of gene therapy IND applications has jumped from 80 in 2015 to more than 200 last year. While there are more than 350 ongoing clinical trials of gene therapies around the world, they represent only a small opportunity to what could become a future of treatments.

I was privileged to present this week with Al Sandrock, head of R&D at Biogen at the 2020 Cell & Gene Meeting on the Mesa to share our vision and efforts in gene therapy. In the coming years we will work to build on the early successes in rare inherited disorders and turn to development for more common debilitating diseases.

Between the tremendous unmet need and worldwide impact, neurological disorders could become the next beneficiary of the remarkable science and engineering being done in gene therapy. Working in neurology has always been a challenge on its own, adding to it a new therapeutic technology increases the challenge. Our focus, however, always comes back to the patients we serve and our goal to bring forth future transformative medicines that have the potential to dramatically improve their quality of life.

I lead the Biogen Gene Therapy Accelerator Unit (GTxAU) which exists at the intersection of world-class neurology and immunology experts, along with extraordinary medical, manufacturing, and commercial organizations, that together allow us to quickly align to solve important problems in clinically impactful ways. Running with the agility of a start-up company but with the full force of a well-established biopharma, the GTxAU is driving innovation on both technology and drug discovery process fronts to accelerate gene therapies that may one day help transform the way we fight neurological diseases.

Our number one asset is our people – our vision is unapologetically bold and we are continuing to recruit some of the best minds in the world to make it come to life. Gene therapy is extremely complex and we can’t do it on our own. Thus, we are also continuously searching for the key collaborators and external innovators who can help us make what today is a dream – gene therapies for the most pervasive and cruel neurological diseases – into tomorrow’s reality. I hope we can count on you to join our mission.