Rett Syndrome

Rare Disease·MeSH: Rett Syndrome

Prevalence

5.0 / 100K

15 drugs in development·8 companies·12 clinical trials

Drug Pipeline by Phase

Phase 3(12)

Drug	Company
ANAVEX®2-73	ANAVEX LIFE SCIENCES CORP. (AVXL)
bionetide	Q BioMed Inc. (QBIO)
epidiolex	GW PHARMACEUTICALS PLC (GWPH)
ngn-401	Neurogene Inc. (NGNE)
nti164	Innovation Pharmaceuticals Inc. (IPIX)
trofinetide	ACADIA PHARMACEUTICALS INC (ACAD)
TSHA-105	Taysha Gene Therapies, Inc. (TSHA)
TSHA-106	Taysha Gene Therapies, Inc. (TSHA)
TSHA-113	Taysha Gene Therapies, Inc. (TSHA)
TSHA-114	Taysha Gene Therapies, Inc. (TSHA)
TSHA-118	Taysha Gene Therapies, Inc. (TSHA)
TSHA-120	Taysha Gene Therapies, Inc. (TSHA)

Phase 2(1)

Drug	Company
ANAVEX® 2-73 (blarcamesine)	ANAVEX LIFE SCIENCES CORP. (AVXL)

Phase 1(1)

Drug	Company
TSHA-102	Taysha Gene Therapies, Inc. (TSHA)

Unknown(1)

Drug	Company
Rett syndrome program (out-licensed to Taysha)	ABEONA THERAPEUTICS INC. (ABEO)

BioSniper tracks 15 drugs in development for Rett Syndrome across 8 companies.

There are 12 clinical trials targeting Rett Syndrome tracked by BioSniper.

The estimated prevalence of Rett Syndrome is approximately 5.0 per 100,000 population.

Full trial data, competitive analysis, and AI-powered pipeline tracking.