Introducing Dr. Hila Ben-Moshe, Rafa’s Moonshot Scientific Director

When we embarked on this journey, we knew that we must think big for such a challenging mission. In the early days of Raphael’s diagnosis, Ella and I prepared our strategic plan and understood two important things: 

  1. Finding a cure for STXBP1 is a complex project that requires proper management and hands-on involvement.
  2. We are not researchers, and we need someone to fill that role full-time.

This led us to search for a scientific director to join and complete our team.

We are proud to have reached this milestone and to have Dr. Hila Ben-Moshe on board with us in this challenging journey. Let’s get to know her better! 

An exclusive interview with Hila

Rafa’s Moonshot: Could you tell us about your background and journey in neuroscience?

Dr. Hila Ben-Moshe: Science has always been a part of my life, and finding cures for illnesses was my dream, leading me to major in biology. Neuroscience was a natural choice for my university focus due to my fascination with the brain.

My passion for neuroscience grew during my undergraduate studies, leading to my BSc cum laude. I then pursued an MSc, specializing in addiction mechanisms, using rat models, collaborating with labs, and securing a patent for addiction treatment.

For my PhD, I specialized in Alzheimer’s disease and neurodegeneration, creating a novel mouse model, employing various techniques, and securing another patent for Alzheimer’s treatment.

After my postdoc, I searched for a place where I would be able to move the needle and make a difference. At Rafa’s Moonshot, I’m harnessing my skills, studies, passion for neuroscience, and the chance to develop a real cure. 

The early days of my PhD studies at Tel Aviv University.

Rafa’s Moonshot: Why did you decide to join Rafa’s Moonshot?

Dr. Hila Ben-Moshe: When I first heard of Rafa’s Moonshot, I deeply connected with its mission to find a cure for Raphael and all STXBP1 patients. The goal to change the world for Raphael and many other kids who are suffering from rare genetic disorders touched me and inspired me to be a part of Rafa’s Moonshot’s journey and success.

Rafa’s Moonshot: What excites you most about working with Rafa’s Moonshot?

Dr. Hila Ben-Moshe: What excites me most is combining my passion for neuroscience with a noble cause. Rafa’s Moonshot approach, combining traditional biology and cutting-edge technology, is thrilling and unique. We are working with the top researchers in the world and scouting for the newest therapeutics approaches and technologies. This allows me to be at the forefront of science and tech.

Rafa’s Moonshot: What key milestones or goals do you aim to achieve with Rafa’s Moonshot?

Dr. Hila Ben-Moshe: Rafa’s Moonshot aims to achieve, on the one hand, a deep understanding of Raphael’s single-point mutation, which is a hotspot mutation in the STXBP1 gene, and on the other hand, advance the broader STXBP1 landscape through key milestones:

  1. Understanding the R292C mutation mechanism of action by establishing and examining novel in vivo and in vitro models.
  2. Identifying and validating FDA-approved compounds for STXBP1. We will consolidate our efforts with the STXBP1 Foundation and other leading researchers in the world.
  3. Evaluating and investing in new technologies and therapeutic approaches.
  4. Donating grants to leading researchers that are aligned with our strategy.
  5. Better Together. We are committed to establishing and maintaining collaborations within the STXBP1 research community and sharing our knowledge and data.
  6. Adding more therapeutic approaches to the STXBP1 pipeline, including our oligos (ASO) and Small Molecule (Drug Repurposing) approaches.

Rafa’s Moonshot: What would you like to tell families and individuals dealing with STXBP1 and other rare genetic diseases who are looking for hope?

Dr. Hila Ben-Moshe: The world is changing, and there are big promises in the near future and, certainly, hope. At Rafa’s Moonshot, we are dedicated to finding answers and solutions. We put every effort into our work, conducting each experiment with utmost seriousness and using every available technology to advance treatments and find a cure. Sagi and Ella managed to find the first therapeutic for Raphael in six months from diagnosis, so we are confident in our strategic plan to find a cure eventually.

Rafa’s Moonshot: Lastly, how do you envision your role at Rafa’s Moonshot contributing to the broader landscape of rare disease research and treatment?

Dr. Hila Ben-Moshe: I aim to impact beyond a single disorder through interdisciplinary collaboration and unwavering dedication. By leading efforts to understand disease mechanisms, establish animal models, and explore novel therapies, I hope to generate insights applicable to other rare genetic diseases. We see many similarities with other NDDs and Single-Gene disorders. The findings and methods of Rafa’s Moonshot can also apply to other researchers and families.

Another important piece of the puzzle is collaboration between researchers. Bringing them together can lead to faster and more efficient advances.

Welcome to the Team

Thank you, Hila, and we are so pleased to welcome you to Rafa’s Moonshot team. Hila’s extensive experience and innovative thinking are vital as we continue our efforts to find a cure and breakthrough for STXBP1.

Follow our journey on this blog and LinkedIn to stay updated about our latest research developments, team achievements, and collaborations in the fight against rare genetic diseases.