TSHA-102
Rett Syndrome
Key Facts
About Taysha Gene Therapies
Taysha Gene Therapies is dedicated to eradicating monogenic CNS diseases through intrathecal delivery of gene therapies. The company has built a robust pipeline leveraging academic partnerships, most notably with UT Southwestern's Gene Therapy Program, and has advanced multiple candidates into clinical development. Following a strategic refocusing in 2023, Taysha is prioritizing its most promising assets, TSHA-102 for Rett syndrome and TSHA-120 for GAN, while seeking partnerships for earlier-stage programs. Its mission centers on addressing the high unmet need in rare neurological diseases with single-administration curative potential.
View full company profileAbout Abeona Therapeutics
Abeona Therapeutics is dedicated to delivering transformative cell and gene therapies for patients with severe rare diseases. The company has achieved a significant milestone with the commercial launch of ZEVASKYN® and maintains a diverse pipeline spanning preclinical to Phase 1/2 development, primarily targeting inherited retinal diseases and neurological conditions. Its strategy is supported by in-house manufacturing capabilities at The Elisa Linton Center for Rare Disease Therapies and key partnerships with Ultragenyx and Taysha Gene Therapies.
View full company profileTherapeutic Areas
Other Rett Syndrome Drugs
| Drug | Company | Phase |
|---|---|---|
| DAYBUE® (trofinetide) | Acadia Pharmaceuticals | Approved/Commercial |
| Trofinetide (NNZ-2566) / DAYBUE™ | Neuren Pharmaceuticals | Approved |
| ANAVEX®2-73 (blarcamesine) | Anavex Life Sciences | Phase 2/3 |
| NGN-401 | Neurogene | Phase 1/2 |