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RTT1

Rett Syndrome

Pre-clinicalActive

Key Facts

Indication

Rett Syndrome

Phase

Pre-clinical

Status

Active

Company

Grann Pharmaceuticals

About Grann Pharmaceuticals

Grann Pharmaceuticals is a private, pre-clinical stage biotech leveraging mRNA-LNP technology to address diseases caused by genetic mutations and protein deficiencies. Its pipeline includes programs for obesity/NAFLD, GABA-A variants, Rett syndrome, and colorectal adenocarcinoma, with a core technological focus on engineering LNPs capable of crossing the blood-brain barrier for CNS applications. Founded and led by CEO Thomas Lyle Temple, the company is motivated by a personal legacy and operates as a pre-revenue entity advancing its research toward human trials.

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Therapeutic Areas

Oncology Metabolic Disease Rare Genetic Disease Neurology

Other Rett Syndrome Drugs

Drug	Company	Phase	Status
NTI164	Neurotech International	Phase 2	Active
NEX-1 / NEX-2	Nexien BioPharma	Pre-clinical / Phase 1	Active
KIT-13	NeuroCores	Pre-clinical	Active
DPM-1003	DepYmed	Phase 1	Active
ExoEdit® for Rett Syndrome	Evox Therapeutics	Pre-clinical	Active
RVL-001 (vorinostat)	Unravel Biosciences	Clinical Trial Initiated	Active
Rett Syndrome Program	Herophilus	Discovery	Active
Glutamate Modulator for Rett Syndrome	Numedicus	Licensed/Orphan Designation	Active
TTI-0102	Thiogenesis Therapeutics	Phase 1	Planned
DAYBUE® (trofinetide)	Neuren Pharmaceuticals	Approved	Commercial
TSHA-102	Taysha Gene Therapies	Phase 1/2	Active (Pivotal-Enabling)
Sarizotan	Newron Pharmaceuticals	Phase III	Discontinued