Pipeline
In Vitro
In Vivo
Phase 1
Phase 2
Phase 3
Rett Syndrome
RTT-1
Obesity
NAFLD
GRN-1
Colorectal
Adenocarcinoma
CA-1
DLG4-1
DLG4
Pitt-Hopkins Syndrome (PTHS)
TCF4 PITT-1
GABA-A Variant
GABRA1
GABA-A Variant
GABRG2
RTT-1 (Eleanor)
Eleanor’s Story: The Founding Patient | Rtt-1
Eleanor Elnekaveh holds a historic place in Grann Pharmaceuticals’ story. She is the founding patient behind this therapeutic program, the first patient in the world to receive this treatment, and the first patient ever dosed by Grann Pharmaceuticals.
Eleanor has Rett Syndrome, but is relentless. She is a little girl who refused to be left behind.
Every day, Eleanor works incredibly hard, pushing her body to the point of exhaustion to regain the skills she has lost. Grann Pharmaceuticals gave her a catalyst to help her get there. From a child who had lost her ability to communicate and walk, Eleanor is now actively walking again, discovering a new world behind every step.
Eleanor’s therapy was made possible through her family’s relentless advocacy and personal commitment to funding and advancing a path forward.
What began as a deeply personal mission to help Eleanor has grown into something much larger.
At Grann Pharmaceuticals, this work represents the heart of why we exist: to move with urgency when children cannot wait. Behind Eleanor’s therapy was a team of young, relentless scientists who worked day and night to push through every obstacle.
Eleanor’s therapy was designed to address the root cause of her condition by delivering the missing or deficient protein her body needs. But the platform behind it has the potential to reach far beyond one child. By placing the correct protein into the right delivery vehicle, this approach may be adapted to support other children with similar genetic mutations and, over time, many rare neurological conditions that have been waiting for a path forward.
Eleanor’s treatment represents more than a first dose. It represents proof of possibility.
Her courage, her family’s relentless advocacy, and the determination of the Grann Pharmaceuticals team helped launch a new path in medicine: one built not around managing symptoms alone, but around addressing disease at its source.
At Grann Pharmaceuticals, Eleanor will always be remembered as the founding patient who helped make this work possible, and as the little girl who reminded us that the impossible only stays impossible until someone refuses to accept it.
Monica Joanna Elnekaveh is the founder of CURE GABA-A, a nonprofit organization dedicated to accelerating treatments and cures for children living with GABA-A receptor disorders.
Blood-Brain Barrier
Grann Pharmaceuticals Inc. has put forth continued research efforts into developing a lipid nanoparticle delivery system capable of crossing the Blood Brain Barrier (BBB). Current research indicates that any particle < 100 nm - 150 nm has the capability to cross the BBB (Nance et al., 2012).
Here at Grann Pharmaceuticals, we have tailored our lipid nanoparticles to an average size of 80 nm to successfully deliver mRNA and small molecules across the BBB. This has been observed in multiple animal trials spanning across several different species of mice.
Our goal at Grann Pharmaceuticals is to apply this lipid nanoparticle technology in therapeutics for CNS disorders and neurological diseases such as Obesity, Rett Syndrome, GABA-A Variants, Alzheimer’s and cancer.
