iNFixion Bioscience is developing an antisense oligonucleotide (ASO) therapy designed to boost NF1 protein expression, aiming to address the underlying root cause driver of neurofibromatosis type 1 (NF1) symptoms, namely NF1 protein haploinsufficiency. Following promising in vitro data, demonstrating the potential for this approach across multiple human cell types, the company has launched a seed financing round to advance in vivo rodent studies. This funding will support crucial preclinical research to further validate the therapy’s safety and efficacy, and to move it one step closer to the clinic for NF1 patients.

Herb Sarnoff, CEO of iNFixion, along with several company investors joined forces in support of the Neurofibromatosis Network’s annual three-day advocacy trip to Washington, DC. Alongside over 100 NF advocates from across the country, Herb and his team met with Congressional leaders to push for continued federal funding of NF scientific research through both the CDMRP’s NF Research Program and at various institutes of the NIH. These efforts are critical in securing continued Federal funding to advance basic science and treatments for NF — not just for those living with NF, but with the potential for broader breakthroughs in cancer, genetic therapies, chronic pain, and more. Advocacy drives progress.

Learn more about the NF Network’s Advocacy Program here.

 

The Gilbert Family Foundation has awarded a three-year grant to iNFixion Bioscience as part of its Next Generation Neurofibromatosis Models Initiative to develop a more human-translatable rodent model for the cognitive and learning deficits experienced by individuals with NF1.  This project builds on research from Dr. Jared Young’s lab at UCSD, which will conduct all rodent testing. Equivalent tests in both kids and young adults with NF1 will be performed by Dr. Karin Walsh at Children’s National Hospital in Washington, DC.  iNFixion will evaluate the NF1 protein levels and distribution in various rodent tissues and coordinate the overall development of what is targeted to be a new rodent model for use in NF1 drug discovery programs targeting neurocognitive symptoms in people living with NF1.

Learn more about the Gilbert Family Foundation’s Next Generation Neurofibromatosis Models Initiative here.

iNFixion announced its third NIH SBIR Phase 1 grant from the National Institute of Neurological Disorders and Stroke (NINDS). iNFixion has received two previous NIH SBIR Phase 1 grants for NF1 drug discovery (2020 and 2021), and this third NIH grant will enable iNFixion to further advance efforts to correct for the lack of normal NF1 protein (called neurofibromin), a critical regulatory protein that is lacking in people living with NF1.

iNFixion’s three NIH grants all focus on developing gene-targeted therapeutics impacting the NF1 protein life-cycle, providing a multipronged drug discovery effort.  In parallel, iNFixion is advancing the development of scientific tools for NF1, such as a new monoclonal antibody to detect and measure NF1 protein, supporting further NF1 research community collaboration.  Herb Sarnoff, iNFixion’s CEO and NF1 ‘dadvocate’, noted: “We are very proud to have the continued support and confidence of the NIH to help iNFixion’s ongoing research to develop a revolutionary treatment for people living with NF1, and to support the NF1 research community wherever possible.”

The mission of iNFixion Bioscience is to discover and develop therapeutics that radically improve the lives of people living with Neurofibromatosis Type 1.

Read the full press release here.

iNFixion is working to discover a new therapeutic for Neurofibromatosis (NF1) by correcting for the lack of normal NF1 protein (neurofibromin), a problem that happens to people born with a mutation in the NF1 gene. One key tool in measuring NF1 protein is the monoclonal antibody (mAb), of which high quality versions simply are not available today.  After launching a project in late 2019 to pursue the design and development of a new and improved mAb for NF1 protein, iNFixion was proud to present its initial research at the annual NF Conference, sponsored by the Children’s Tumor Foundation (CTF), in Philadelphia, PA from June 17-21, 2022.

Mutations in the NF1 gene result in a lack of normal NF1 protein, a condition known as protein haploinsufficiency and which drives NF1 symptoms. As a start-up company, iNFixion is working to identify drugs that restore normal NF1 protein expression levels, thus treating and preventing a wide range of symptoms attributed to NF1. In order to do this, it is critical to have accurate measurement tools for neurofibromin that are both selective (recognize only NF1) and sensitive (can detect small amounts and changes).  In research presented by our Principal Scientist Dr. Michelle Mattson-Hoss (project PI), the new antibody was shown to provide highly selective and sensitive detection of neurofibromin, and to do so across a variety of important assay methods, including western blotting, ELISAs, and immunohistochemistry (IHC). The antibody is performing superior to current commercially available NF1 antibodies, and has now also gone through an exhaustive IHC qualification study executed by Dr. Michael Frost, a board certified pathologist and scientific advisor to iNFixion.

Partially funded by a grant from the Neurofibromatosis Therapeutics Acceleration Program (www.n-tap.org), iNFixion partnered on this project with Abterra Biosciences, a San Diego-based company using state-of-the-art antibody discovery technology, and pioneered the use of full length NF1 protein (as the antibody immunogen) which had been recently developed by the Frank McCormick Lab at UCSF in collaboration with Dr. Dominic Esposito of the Frederick National Lab.

This new antibody provides a significant advance in the ability of NF researchers to study NF1 protein expression. As noted by iNFixion’s CEO, Herb Sarnoff: “Once available, this new NF1 monoclonal antibody should prove valuable in a wide range of NF1 drug discovery efforts, including any therapy targeting the restoration of NF1 protein levels.” With further validation of antibody performance, including monitoring manufacturing variation, and assessing cross reactivity with NF1 protein in other species, iNFixion is now working to deliver this valuable new tool to NF1 researchers worldwide by early 2023, thus helping to drive continued progress toward treatments for NF1.