Frank Gentile, PhD

Frank Gentile, PhD, is one of our original board members and has become an invaluable asset to Usher 1F Collaborative. We took the opportunity to learn more about Frank and to share why Usher 1F Collaborative has become such an important cause to him.

Frank is a chemical engineer. He received his BE in chemical engineering from the Cooper Union in New York, NY, followed by a PhD in chemical engineering from the Massachusetts Institute of Technology (MIT). Subsequently, he completed a post-doctoral fellowship at the Swiss Federal Institute of Technology in Zurich, Switzerland.

Frank’s first job in biotechnology was as a scientist at CytoTherapeutics, eventually managing all clinical programs including the company’s ALS and chronic pain phase I and II clinical trials. Jobs at Reprogenesis and Millenium Pharmaceuticals followed, and then he left biotech for capital management, investing in both private and public biotech and medtech companies. His experience culminated in becoming Venture Partner at Third Rock Ventures in Boston, where he helped start companies such as Decibel, Maze, and Casma. He left Third Rock to become first full-time COO and then CEO at Casma Therapeutics, where he remains. Casma develops therapies for neurological conditions. 

With his background, it is no wonder that Frank has been an invaluable member of our board, serving not only as an expert scientist who helps evaluate our research grant proposals but also as a bridge between the academic and commercial biotech arenas, helping us in our work to advance our research from the lab to the clinic.

Frank has been married for 38 years to his wife Erin, and they have three children, Stefan, a post-doc in molecular biology and bioengineering at the Broad Institute of MIT and Harvard, Ben, a film and documentary director, and Sam, an occupational therapist. 

We asked Frank questions about his involvement with and dedication to Usher 1F Collaborative.

1.         What is your connection to Usher 1F Collaborative and what brought you onto the board as one of its first members?

I was asked to be on the board by Elliot Chaikof, one of the founders of the Usher 1F Collaborative. Elliot and I were classmates at MIT where we were both in the PhD program in chemical engineering doing research in polymer science and engineering. Elliot and his co-founder, Melissa Chaikof, wanted a board member with biotechnology industry experience, and given my background in approved product development, private investing, and starting biotech companies, they believed I could be useful in helping to make targeted investments in scientific and clinical programs for the Collaborative.

2.        Why is finding a cure for the vision loss of Usher 1F important to you and what motivates you to continue to work with us?

As a child and young person, I was very close to my paternal grandfather. He twice immigrated to the US for employment opportunities, once when he was seventeen and again in his 30s during the Great Depression. In 1944, he was blinded in a farm accident in southern Italy. He died in 1992 at the age of ninety. While he was quite functional, I saw how devasting a permanent loss of vision was for him and his entire extended family. I always wanted to work in the area of vision loss, and while I was an investor, one of my best public company investments was in Regeneron Pharmaceutics, a company that developed a drug called Eylea, a novel approach to wet age-related macular degeneration, a common cause of blindness in older adults. During my time at Third Rock Ventures, I worked on starting Decibel Therapeutics, a company focused on hearing loss, which is also a hallmark of Usher 1F. (Coincidentally, Decibel was purchased by Regeneron). Again, I came to realize how painful hearing loss is, especially in young children and how underserved this area was by biotech and pharmaceutical companies. Thus, these personal and professional experiences make me very motivated to do everything I can to help the Usher 1F Collaborative in its mission to find therapies and even a cure for vision loss in these very young people. 

3.        What makes Usher 1F Collaborative unique and enhances its chances for success?

There are three things that make the Usher 1F Collaborative unique. First, while there are many such groups focused on raising funds and awareness for specific rare genetic diseases of children, few have the benefit of having a world class physician-scientist as one of their founders. Not only can such a founder help evaluate various research projects for scientific rigor and feasibility, but he or she can also attract other world class researchers to put their talents to work in finding therapies and a cure. Elliot Chaikof is such a founder, and as just one example of how the Collaborative has benefited from his leadership position at Harvard Medical School (HMS), the Collaborative attracted David Corey, a professor at HMS who developed a novel approach to gene therapy for large gene constructs, which is required with existing technology to treat Usher 1F.  I do not believe other groups have those kinds of networks. 

Second, Elliot and Melissa Chaikof have created a Board of Trustees for the Collaborative that is a combination of other parents of children with Usher 1F, an adult with Usher 1F, folks that have experience in similar charities, and people who can help evaluate scientific proposals. In this manner, a high percentage of every dollar raised by the Collaborative goes to great researchers with a singular focus of being able to translate scientific discoveries into therapies. The return on investment we have made has led the Collaborative, in a brief period of time, to be on the cusp of a first clinical trial for a therapy for Usher 1F. 

Third, the Usher 1F Collaborative invested in a natural history study of the disease. This allows us to determine the normal time course of the disease and thus will allow the appropriate evaluation of success for potential therapies. As an example, it was not until a natural history study was conducted in Duchenne Muscular Dystrophy that potent therapies were developed. To have a foundation take on this challenge is nothing short of extraordinary. We have also been able, due to the highly professional operation we have, to encourage the Foundation Fighting Blindness to co-sponsor this Usher 1F natural history study.