Grann Pharmaceuticals has announced a milestone in the development of transformative therapies for a rare and debilitating genetic disorder known as Rett syndrome.
The company’s 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 characterised 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.
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