King of Prussia, PA – September 9, 2025 – Merlin Therapeutics, Inc., a biotechnology company specializing in genetic medicines for life threatening genetic disorders, today announced it has been awarded a $3 million Phase 2 Small Business Innovative Research (SBIR) grant from the National Institute of Neurological Disorders and Stroke (NINDS), a division of the National Institutes of Health (NIH). This grant will support the preclinical development of MRL-102, the company’s groundbreaking gene therapy, for the treatment of schwannomas.

Schwannomas are slow-growing, non-malignant tumors that develop along peripheral, spinal, and cranial nerves. Schwannomas can arise sporadically, or as a part of the debilitating genetic syndromes Neurofibromatosis Type 2-related schwannomatosis (NF2-SWN) and non-NF2-related schwannomatosis. The proximity of these tumors to the nervous system often leads to substantial disability and significantly diminished quality of life for patients, causing deafness, vision loss, balance disorders, severe and persistent pain, facial paralysis, substantial psychological comorbidities, and in many cases are life-threatening. The standard of care is surgical resection, which carries substantial risks and is not always feasible. There are currently no pharmacological treatments available that target the underlying genetic cause of these tumors. Merlin Therapeutics is addressing this major unmet medical need by developing a novel gene therapy, MRL-102, which delivers a pro-apoptotic inflammasome pathway gene as a targeted, life-changing treatment for schwannoma patients.

“We are incredibly pleased to have been awarded this significant grant from NINDS,” stated Nora Yang, PhD, CEO of Merlin Therapeutics and the Principal Investigator (PI) of this SBIR grant. “This funding will accelerate the preclinical and clinical testing of our innovative gene therapy, MRL-102, bringing us closer to delivering a transformative treatment to NF2-SWN patients and beyond. In addition to the substantial financial support, the rigorous NIH peer-review approval process itself is a powerful validation of MRL-102’s scientific and clinical merit.”

The grant underscores the scientific community’s recognition of MRL-102 as a promising therapeutic candidate for neurological disorders. The accelerated development pathway enabled by this funding is expected to expedite the availability of critical data, paving the way for regulatory submissions and ultimately, patient access.
This grant includes a sub-award to Dr. Gary Brenner, MD, PhD, the Massachusetts General Hospital award PI and Associate Professor at Harvard Medical School. Dr. Brenner’s dedicated work over several decades to find a treatment for NF2-SWN led to the discovery of MRL-102. Dr. Brenner expressed his enthusiasm for the NINDS SBIR award, stating, “I am excited to have received this NINDS SBIR subaward and looking forward to working with the Merlin Therapeutics team of translational science experts to bring this promising therapy to patients.” He also highlighted the innovative potential of MRL-102: “Once we achieve clinical proof of concept in schwannoma patients, we can readily switch the promoter sequence in MRL-102 to target other relevant tumor and cancer types, expanding the scope of this therapy to help more patients.”

Merlin Therapeutics extends its gratitude to the NINDS for this grant which will enable the company to complete the necessary work for an Investigational New Drug (IND) filing. This grant is supported by the National Institute of Neurological Disorders and Stroke of the National Institutes of Health under Award Number R44NS145814. The content of this public announcement is solely the responsibility of Merlin Therapeutics and does not necessarily represent the official views of the National Institutes of Health.