San Diego-Area Biotech Startup Achieves Major Breakthrough in the Fight Against Rare Neurodevelopmental Disorder

SAN MARCOS, Calif., Aug. 20, 2025 /PRNewswire/ -- Grann Pharmaceuticals today announced a historic milestone in the development of transformative therapies for a rare and debilitating genetic disorder known as Rett Syndrome. The company's very first patient, Eleanor Elnekaveh, has successfully completed the experimental treatment regimen of RTT-1 (Eleanor), an mRNA lipid nanoparticle (LNP) protein replacement therapy. Grann was able to achieve this accomplishment from proof of concept to treatment in only 18 months.

Rett Syndrome is a devastating, progressive neurodevelopmental disorder caused by mutations in the MECP2 gene. It primarily affects females and is characterized by the loss of previously acquired motor and communication skills, seizures, breathing irregularities, muscle stiffness, and severe cognitive impairment. There is currently no cure, and available treatments are limited to symptom management. RTT-1 (Eleanor) represents a potential paradigm shift in how this disease is treated by addressing the root cause of the disease.

Eleanor tolerated the therapy well— a critical first step toward making this innovative therapeutic available to more patients. Grann Pharmaceuticals plans to apply for long-term continuation of Eleanor's treatment and is preparing for discussions with the U.S. Food and Drug Administration (FDA) on advancing RTT-1 (Eleanor) through the regulatory process.

"This is a pivotal moment for our company, for the Rett community, and for the field of medical genetics," said Thomas Lyle Temple, CEO of Grann Pharmaceuticals. "We are committed to accelerating development so we can deliver innovative mRNA LNP therapies to Eleanor and children like her who face this catastrophic disease. For the first time, families affected by Rett Syndrome can have new hope where none previously existed."

Grann Pharmaceuticals is actively seeking partnerships to support the next phases of development and expand access to RTT-1 as quickly and safely as possible. The company extends its gratitude to its partners at California State University San Marcos' Infinity Lab, Salus ASC, Combined Brain, Mitchell Blair, Dr. Raj Batra, Monica Joanna Elnekaveh, and the Elnekaveh family (Founders of CURE GABA-A) for their instrumental roles in making this breakthrough possible.

About Grann Pharmaceuticals
Grann Pharmaceuticals is a biotechnology company pioneering mRNA lipid nanoparticle–based treatments for patients with diseases caused by the absence of a critical human protein. The company's mission is to improve the quality of life by reversing medical conditions caused by genetic abnormalities and mutations. Grann Pharmaceuticals was inspired by the resilience and legacy of Grannan, the grandmother of CEO Thomas Lyle Temple, who lost her battle with neuroblastoma cancer in 2018.

Contact Grann Pharmaceuticals

To learn more about our mission and pipeline, please visit: www.grannpharma.com
For all media inquiries or additional information, please contact:

Patrick Granahan
Chief Operating Officer
Grann Pharmaceuticals Inc.
[email protected]
www.grannpharma.com

River Danner
Business Development Manager
Grann Pharmaceuticals Inc.
[email protected]
www.grannpharma.com

SOURCE Grann Pharmaceuticals Inc.