Merlin Therapeutics Awarded Maximum Amount Under NIH STTR Grant to Develop Gene Therapy for Neurofibromatosis Type 2 (NF2)

King of Prussia, PA. August 19, 2024 – Merlin Therapeutics, an NF2-focused biotech startup, is pleased to announce that it has received a $699,559 grant from the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), to advance its leading gene therapy treatment for Neurofibromatosis Type 2 (NF2), which is currently optioned from the Research Institute at Nationwide Children’s Hospital.

NF2 is a serious genetic disorder characterized by the growth of encroaching tumors on nerves, particularly in the brain and spinal cord. These tumors can cause debilitating pain and lead to loss of hearing, vision, balance, along with muscle weakness and paralysis in nearly all NF2 patients while larger tumors can be life-threatening. Epidemiological studies show that NF2 shortens lifespan to an average of 36 years and severely diminishes patients’ quality of life. Currently, treatment options for NF2 are restricted to surgical resection of tumors and radiation, both of which come with significant risks including severe nerve damage, exorbitant costs, and unpredictable clinical outcome. There is an urgent need for more effective therapies and research into the complexities of this progressive disease, making this grant a significant step towards developing a treatment.

The Phase 1 grant, awarded through the NIH’s STTR program, will fund preclinical studies to demonstrate the safety and efficacy of Merlin Therapeutics’ leading gene therapy candidate in animal models. In collaboration with Nationwide Children’s Hospital and Massachusetts General Hospital, these studies are expected to pave the way for clinical trials and patient dosing.

“Our goal is to develop disease modifying therapies that offer effective and safe treatments for NF2 patients by addressing the underlying cause of the disease,” said Nicole Henwood M.D., CEO and founder of Merlin Therapeutics. “This funding represents a significant achievement, not only for our company but for a patient population that urgently needs more suitable treatment options.”

Merlin Therapeutics thanks the NINDS for their support, as well as its research collaborators and advocacy groups who have contributed to reaching this milestone.

Research reported in this release was supported by the National Institute of Neurological Disorders and Stroke of the National Institutes of Health under Award Number 1R41NS135883-01. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

For more information about Merlin Therapeutics, please visit https://merlintherapeutics.com/.

About Merlin Therapeutics

Merlin Therapeutics is developing breakthrough gene therapies that target the underlying etiology of NF2 and have the potential to reduce growth of existing and future tumors, thus addressing the urgent unmet medical need of patients suffering from NF-2 genetic defects.  Merlin partners with top gene therapy experts to successfully demonstrate the therapeutic viability of gene therapy for NF2.

Contact Info

Nicole Henwood MD

CEO

nhenwood@merlintherapeutics.com