Patients and families are at the heart of Grann Pharmaceuticals. Their experiences and perspectives help guide our work to develop investigational mRNA therapies for rare genetic diseases.
Our commitment extends beyond Rett syndrome. We welcome conversations with patients, families, caregivers, and clinicians affected by other rare genetic disorders—including conditions not currently listed in our pipeline—to better understand their needs and explore potential research opportunities.
Patients
Our Investigational Programs
Our pipeline spans rare genetic disorders, oncology, and metabolic disease. RTT-1 for Rett syndrome is our lead program, alongside programs targeting additional conditions. Each program is at a different stage of development, and treatment availability depends on its research progress and applicable medical and regulatory requirements. If your condition is not currently listed in our pipeline, you are welcome to contact us to ask about potential research opportunities.
Development & Access
Grann is advancing multiple investigational programs through research and development. Patients, families, and treating physicians may contact our team about a specific program or another disease of interest. We review inquiries in the context of our scientific approach, available evidence, and development capabilities. Submitting an inquiry does not guarantee that Grann can develop a therapy, initiate a study, or provide treatment.
Two girls have received RTT-1 through single-patient expanded access. Grann is preparing for further clinical development, including a planned Phase 1 study. Families and treating physicians may contact our team to ask about the program’s current status and potential future opportunities. Submitting an inquiry does not guarantee eligibility, participation, or access to treatment.
About Expanded Access
Expanded access is a potential pathway for eligible patients with serious or life-threatening conditions to receive an investigational treatment outside a clinical trial when no comparable or satisfactory alternative therapy is available. Access requires medical review, company agreement, and applicable regulatory and ethics approvals.
Meet 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.
