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Since joining our team in 2022, Dr. Hackett has not only led but significantly propelled our gene therapy program forward, drawing upon his extensive experience in pre-clinical and translational gene therapy programs. Furthermore, under his visionary leadership, our foundation has assembled a multidisciplinary team of experts, greatly enhancing our potential to make a meaningful impact within the INAD community.

Currently holding the position of Professor of Research in Genetic Medicine at Weill-Cornell Medical College, Dr. Hackett boasts an impressive portfolio featuring 100+ scientific publications and numerous patents. His innovative contributions have led to the creation of two biotechnology companies. Additionally, he has played a pivotal role as a co-investigator in seven clinical trials, all focused on novel biological treatments for neurological disorders.

Thanks to his steadfast leadership and bolstered by the unwavering support of the INAD community, we are closer than ever to discovering an effective treatment for INAD. We are immensely grateful to have Neil Hackett on our team!

Neil Hackett, Ph.D., Professor of Research in Genetic Medicine Weill-Cornell Medical College

Neil Hackett, Ph.D.

Professor of Research in Genetic Medicine
Weill-Cornell Medical College

LET’S MEET: Dr. Hackett

Tell us a little bit about yourself.
I am located in New York City and work as an academic researcher and genetic medicine consultant for several foundations.

Why did you choose your career?
I started my scientific education just when recombinant DNA was beginning. The speed which we went from knowing very little to the human genome and now to gene editing has maintained my fascination about this subject.

When did you first decide you wanted to work with rare diseases? Please share what led to that decision.
Twenty five years ago I met a couple who wanted to develop gene therapy for their two children with a rare genetic disease. When you see the commitment of the parents to help their children, it is impossible not to be carried along on the journey and do everything you can to help.

Tell us a little about any current/previous work with other rare diseases. For example, have you ever been a part of a team that has worked on gene therapy/other drug development initiatives before, or has brought a potential treatment to a clinical trial? What has been one of your most rewarding experiences?
I have travelled the road from basic research to clinical trial several times. I also developed a gene therapy drug to treat advanced angina, now in late-stage clinical testing. To see actual clinical benefit in patients is the reward we work towards.

Why did you choose to work with the INADcure Foundation?
INADcure is a very focused and well-managed foundation who have defined their goals and moved decisively to achieve them. They are a pleasure to work with.

What recent breakthroughs have gotten you most excited for the future of rare disease?
Although I am not sure where it is going, the new somatic gene editing methods are showing great potential for treating some genetic diseases. I hope to participate in programs to make that a reality.

What is your favorite part of the work you do?
The foundations which I work for and our academic collaborators are a community fully committed to the goal of developing treatment for genetic diseases. It deeply satisfying to be a part of that effort and to contribute as best I can.

What do you do to relax (or have fun)?
The endless distractions of the big city.

If you could have a lifetime supply of anything, what would it be?

Last (or Best) book read?
Let the Great World Spin

If you could have one superpower, what would it be?
Never sleeping but not being tired!

Help us on our mission of finding a cure for INAD/PLAN.