New York, NY and Cambridge, MA: The Children’s Tumor Foundation (CTF) has provided funding to NF2 Therapeutics, Inc. (NF2RX) to develop a first-in-class gene therapy for NF2-related schwannomatosis (NF2), a devastating rare genetic disorder characterized by the growth of tumors along nerves. This funding follows an initial $100,000 round of funding previously provided by CTF. Both rounds were structured as venture philanthropy funding. The initial funding enabled experiments in the lab of Dr. Helen Morrison, Leibniz Institute on Aging – Fritz Lipmann Institute (FLI) in Jena, Germany, which demonstrated that biologic therapies can successfully rescue the function of NF2 in a cell culture model. Dr. Morrison is a key opinion leader who has published extensively on the function of human merlin, the protein that is mutated in NF2, and has helped elucidate its role in tumorigenesis.
The Children’s Tumor Foundation and NF2 Therapeutics Inc. are optimistic that an NF2 gene therapy will provide a disease-modifying treatment for patients. NF2RX is working with key opinion leaders and leading technology partners to develop vectors tailored for delivery to Schwann cells where schwannomas occur and arachnoid cells where meningiomas occur. Michael Wootton, CEO of NF2 Therapeutics, said, “We are pleased to receive a second round of funding from the Children’s Tumor Foundation, and look forward to developing a gene therapy that we hope will be a disease modifying treatment for NF2.”
Annette Bakker, PhD, President of the Children’s Tumor Foundation said, “NF2 leads to tumors that include vestibular schwannomas, which lead to deafness and problems with balance, and meningiomas, which lead to significant morbidity and mortality. We are excited to work with NF2RX and provide funding to develop a gene therapy that could greatly benefit the quality of life of those affected by NF2.”
About NF2
NF2-related schwannomatosis, which is caused by mutations in the NF2 gene, occurs in one in every 25,000 people worldwide. It is a rare genetic disease characterized by the growth of tumors along nerves, which leads to tumors of the brain, spinal cord, and peripheral nervous system. In NF2, vestibular schwannomas lead to deafness and dysfunction of the vestibular system. Meningiomas, which are tumors on the lining of the brain and spinal cord, cause significant morbidity and mortality for patients. NF2 is worldwide in distribution, affects all populations equally and has no particular gender, racial, geographic or ethnic distribution. The current standard of care for these tumors is similar to that of many cancers, including surgery, radiation, and chemotherapy. While these therapies can help to slow the progression of these diseases, they can have devastating consequences in the lives of patients. Currently there are no FDA-approved drugs for either condition.
About NF2 Therapeutics, Inc.
NF2 Therapeutics, Inc., is a Cambridge, Massachusetts-based biopharmaceutical company founded in 2016 to focus on the development of novel therapies for Neurofibromatosis II (NF2). The company was founded by experienced biotechnology executives and entrepreneurs Mark de Souza, PhD, Michael Wootton, and Scott Plotkin, MD, PhD, a key opinion leader in neurofibromatosis.
About Leibniz Institute on Aging
The Leibniz Institute on Aging – Fritz Lipmann Institute (FLI) – upon its inauguration in 2004 – was the first German research organization dedicated to research on the process of aging. More than 330 employees from over 30 nations explore the molecular mechanisms underlying aging processes and age-associated diseases. For more information, please visit http://www.leibniz-fli.de.
About the Children’s Tumor Foundation
The Children’s Tumor Foundation is a 501(c)(3) not-for-profit organization dedicated to funding and driving innovative research that will result in effective treatments for the millions of people worldwide living with neurofibromatosis (NF), a term for three distinct disorders: NF1, NF2, and schwannomatosis. NF causes tumors to grow on nerves throughout the body and may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, and cancer. NF affects 1 in every 3,000 births across all populations equally. There is no cure yet – but the Children’s Tumor Foundation mission of driving research, expanding knowledge, and advancing care for the NF community fosters our vision of one day ending NF. For more information, please visit www.ctf.org.
