In our mission to accelerate a cure for neurofibromatosis (NF1) we are dedicated to creating an environment where the best and brightest minds can work together in collaboration. In August, we shared milestones and updates to our Vision Restoration Initiative, bringing together 28 leading researchers from intersecting medical fields. More recently, we hosted our first-ever in-person annual event for our Gene Therapy Initiative (GTI), where we brought together nine research teams who had been working with us since 2019, as well as the 12 new research teams that we began partnerships with this year. This research is critical, because current therapies only treat symptoms and do not solve for the underlying issue of genetic disease. 

During the event, our research teams presented exciting findings to advance development of curative therapies that address the inherent genetic abnormalities in NF patients.

Some of these findings include:

  1. Suppression of the nonsense NF1 mutations has been demonstrated to restore neurofibromin function in multiple tissue types.
  2. Sufficient and targeted delivery of NF1 gene therapies to specific tissues is a critical challenge.  We discovered that AAV, exosomes, and nanoparticles may provide alternate and complementary approaches.
  3. Standardization of in vitro and in vivo assays will be paramount to identifying the most promising gene targeting therapies for NF1 patients.

You can learn more about the event and our progress in the video, below. We are looking forward to uncovering more findings as our research teams reconvene to further delve into this important work.