In this new company video, we share our vision of developing treatments that target the root cause of NF1. Thanks to Savanna for sharing her story, to our advisors and collaborators for their support, and to the team at WCB Films for making this video happen!
Watch to learn more:
iNFixion recently attended its first Gilbert Family Foundation (GFF) meeting where the team learned about many new NF1 preclinical models, including rodent models and organoid models, that are being developed with funding from the GFF.
iNFixion’s Medical Director, Dr. Mick Frost, presented our work on developing an improved animal model to evaluate the cognitive, learning and social deficits in NF1. Mick is a pathologist, and NF1 parent, who is 100% dedicated to NF1 research. Great job, Mick!
iNFixion is honored to be partnered with the Gilbert Foundation in fighting for people living with NF1.
Watch the GFF video about the event here: https://lnkd.in/gQre-ymu
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.
iNFixion CEO, Herb Sarnoff, was featured in the Translational Research Showcase of the Invest in Cures Forum where he shared the personal motivation behind founding iNFixion Bioscience and the company’s ongoing efforts develop a therapy to address the unmet needs of individuals, like his daughter, living with neurofibromatosis type 1 (NF1). Invest in Cures is a full-day forum focused on the promise of impact investing and innovative collaborations to revolutionize rare disease therapies. This unique annual event brings together researchers, clinicians and investment professionals from leading foundations to discuss cutting-edge approaches to supporting and funding breakthrough therapies in rare diseases.
Read more about the event here.
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.
Members of iNFixion’s executive team headed to Brussels for the 2024 Global NF Conference, the leading international meeting shaping the future of neurofibromatosis and schwannomatosis research and clinical care. Organized by the Children’s Tumor Foundation and the European NF Group, this event brought together over 1,000 experts, clinicians, researchers, and advocates. The team was honored to reconnect with the global NF community, share insights, and contribute to the progress driving new treatments forward. Several new collaboration opportunities were identified, including potential projects with the amazing NF1 research team from Murdoch Children’s Research Institute in Melbourne, Australia.
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 published a peer-reviewed paper in the Journal of Translational Genetics and Genomics highlighting the rationale for haploinsufficiency correction therapy (HCT) as a potential treatment for neurofibromatosis type 1 (NF1). Evidence supports that a reduced level of functional neurofibromin (NF1 protein) in cells harboring an NF1 mutation plays a direct role in driving NF1 manifestations. By addressing this protein haploinsufficiency, iNFixion’s NF1-HCT offers a promising therapeutic approach by targeting the root cause of NF1. By enhancing the expression of the NF1 protein from the one normal NF1 gene present in everyone with NF1, iNFixion’s NF1-HCT is predicted to be effective regardless of a person’s specific NF1 mutation. This is critical given that more than 4,000 unique NF1 mutations have already been identified.
Read the full scientific article 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.
A three-year grant through the DOD Neurofibromatosis Research Program (NFRP) has been awarded to Dr. James Walker’s lab at Massachusetts General Hospital (MGH), with iNFixion Bioscience as a sub-awardee, to develop and validate a lentiviral CRISPRa/Cas9 tool designed to enhance NF1 protein expression. The Walker Lab will design the CRISPRa/Cas9 compounds for testing, while iNFixion will evaluate their efficacy in both cellular and rodent models. This research will not only provide a tool to evaluate iNFixion’s NF1 haploinsufficiency correction therapy (NF1-HCT), but could also serve as the foundation for a potential NF1 therapeutic.