CURE GABA-A (https://curegabaa.org/) is proud to announce a major new partnership with Grann Pharmaceuticals. CURE GABA-A is a nonprofit patient advocacy group founded by Monica Joanna Elnekaveh, mother to Eleanor Elnekaveh. Grann Pharmaceuticals has completed the initial safety regimen of RTT-1 (ELEANOR) in Eleanor as its very first patient.
Together with COMBINEDBrain, Agustina Fernandez, Sarah Poliquin, Amber Freed, and Roberto Gomez, CURE GABA-A is expanding into a truly global network connecting families, researchers, and industry partners to accelerate the development of life-changing therapies for rare neurological disorders caused by GABA-A gene receptor variants. CURE GABA-A would like to thank the Fernandez, Bornstein and Elnekaveh Families for their generous financial contribution, as well as COMBINEDBrain, led by Terry Jo Bichell, SLC6A1 Connect, led by Amber Freed, and the SYNGAP Research Fund (SRF), led by Mike Graglia, for their mentorship and support.
Monica Joanna Elnekaveh's advocacy began when Eleanor was initially diagnosed with two mosaic gaba receptor gene deletions (GABRA1 and GABRG2). Driven by the urgent need for treatments, Monica founded CURE GABA-A to unite families, clinicians, and researchers worldwide in the fight against GABAAR related disorders. Later, through whole exome sequencing, Eleanor received an additional diagnosis of Rett Syndrome, expanding Monica's mission to bridge GABAAR scientific research communities, patients and biotechs to accelerate therapeutic development.
This new collaboration between CURE GABA-A and Grann Pharmaceuticals builds on Grann's proven expertise in mRNA lipid nanoparticle (LNP) therapeutics and follows a major scientific milestone: the successful completion of the RTT-1 (ELEANOR) safety regimen in the very first patient, Eleanor herself. The therapy was well tolerated, marking a critical step forward in the development of mRNA LNP protein replacement therapies for Rett Syndrome and other rare neurodevelopmental disorders such as those caused by GABAAR variants.
Under this partnership, Grann will lead proof-of-concept and preclinical animal studies targeting GABAAR variants, applying the same innovative therapeutic platform and scientific rigor that made the RTT-1 (ELEANOR) achievement possible.
By combining Grann's technical capabilities with CURE GABA-A's deep patient insights and global network, we are advancing toward our shared goal: pushing the boundaries of innovation, accelerating the path to clinical trials, and delivering transformative therapies to children and families with rare genetic diseases.
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