JimName: James (Jim) Rusche

Where do you work? I am retired from directing Research and Development for a Biotech company that was active in FA drug development, Repligen Corporation. They are no longer active in therapeutic development projects.

How long have you been working on FA and who was the first fellow FA researcher you met? I started working on FA in 2007 when I was made aware of the research findings of Dr. Joel Gottesfeld at Scripps Research Institute. We started a collaborative research and development program to advance an approach Dr. Gottesfeld had identified, which involved increasing frataxin expression.

What got you interested in FA research? As a leader in a commercial company, I was charged with finding opportunities to advance new therapies for rare diseases in which there was no current treatment. Rare diseases with recently identified single gene defects like FA were important opportunities to develop new treatments and help patients.

What question or challenge were you setting out to address when you started this work? Can we restore the frataxin protein and directly address the cause of the disease? When we started this quest, gene therapy was not yet an option, but chemically inducing the gene to express more protein was a viable option.

What research topics or questions are you currently focused on? I am assisting FARA as a Board member for strategic initiatives as well as contributing to the Scientific Review Committee to help steer FARA resources to make the greatest contribution possible to developing new treatments for FA.

Jim 2What do you hope to achieve or what excites you in FA research? There is no single disease treatment opportunity in my career as exciting as the potential to help FA patients through developing new therapies. I fully expect to help the community of scientists, drug developers, and patients find a life-changing treatment for FA. I believe the most promising approach today is gene therapy.

If you have met someone living with FA, please tell us about that interaction. Did it have an impact on your work? Like many scientists and pharmaceutical executives, I have had the pleasure of meeting Kyle Bryant and through him many other people with FA. It is their collective energy, enthusiasm, and life skills that stimulate me to find a path forward. I have met many FA patients that have elevated their game as human beings, perhaps because of this challenge of FA. My major focus after retirement with my 30 years of scientific skills is dedicated to this endeavor because of the people I have met in this community.

You serve voluntarily on FARA's Scientific Advisory Board. Please tell us what you see as FARA's key role in the research process. FARA, to me, has, through its leadership, instilled a caring, positive, yet pragmatic approach to try and cure this disease. FARA is an advocate and a facilitator of progress for developing patient treatments. Sometimes that requires a clinical treatment network, sometimes a patient registry, sometimes funded research or research centers of excellence, and sometimes advocacy before the government (FDA) or companies. With this focus, it has attracted top key scientists and institutions to make this disease a priority.

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