Rett syndrome - Page 1

Bring it back home: Taysha regains gene therapy as option expires

Oct. 17, 2025

The full rights to Taysha Gene Therapies Inc.’s lead gene therapy candidate are coming back to the company. In 2022, Astellas Pharma Inc. invested $50 million in Taysha for 15% of the company and the exclusive option to license TSHA-102 for treating the rare disease Rett syndrome. That option has expired, according to Taysha.

Rett bet set includes Neurogene, Taysha gene therapies

July 24, 2025

By Randy Osborne

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Neurogene Inc.’s disclosure June 30 of its registrational trial design in Rett syndrome (RS) added spice to the space, where Taysha Gene Therapies Inc. is another closely watched player. The New York-based firm said Embolden will test NGN-401, a gene therapy for the treatment of females age 3 and older with RS. U.S. regulators have signed off regarding key aspects of Embolden’s structure.

Neurology/psychiatric

Unravel seeks Colombian approval to study RVL-001 for Rett and Pitt-Hopkins syndromes

April 30, 2025

Unravel Biosciences Inc. has submitted clinical study applications in Colombia seeking to begin proof-of-concept clinical trials for RVL-001 for Rett syndrome and Pitt-Hopkins syndrome.

Neurogene slides on Rett syndrome gene therapy safety concern

Nov. 18, 2024

By Karen Carey

Neurogene Inc.’s stock sank 43% on news that its phase I/II gene therapy, NGN-401, resulted in a serious adverse event in a pediatric Rett syndrome patient receiving the highest dose.

Illustration of Toxoplasma gondii tachyzoites

Drug design, drug delivery & technologies

Parasite could deliver therapeutic proteins to the brain

Aug. 13, 2024

By Mar de Miguel

Infection or cure? Scientists from Tel Aviv University and the University of Glasgow genetically modified the Toxoplasma gondii to bring a protein inside neurons. The novelty of using a protozoan that can travel from the gut to parasitize the CNS contrasts with the possibility of causing a disease. The scientists are already working on how to avoid it.

Drug design, drug delivery & technologies

Parasite could deliver therapeutic proteins to the brain

Aug. 8, 2024

By Mar de Miguel

Infection or cure? Scientists from Tel Aviv University and the University of Glasgow genetically modified the Toxoplasma gondii to bring a protein inside neurons. The novelty of using a protozoan that can travel from the gut to parasitize the CNS contrasts with the possibility of causing a disease. The scientists are already working on how to avoid it.

Neurotech’s NTI-164 meets endpoints in pediatric autism trial

April 17, 2024

By Tamra Sami

Neurotech International Ltd.’s cannabinoid NTI-164 met the primary and secondary endpoints in a phase II/III trial in children with autism spectrum disorder. The company also reported positive top-line data for Rett syndrome in a phase I/II trial.

Neurology/Psychiatric

Depymed’s DPM-1003 receives IND clearance for Rett syndrome evaluation

March 20, 2024

Depymed Inc. has received FDA clearance to initiate a phase I trial for DPM-1003 for the treatment of Rett syndrome.

Step Rett up: Acadia’s Daybue comes on strong as others in the works, too

Jan. 18, 2024

By Randy Osborne

After the U.S. FDA granted clearance in March 2023, sales began promptly of Acadia Pharmaceuticals Inc.’s new Rett syndrome therapy, Daybue (trofinetide), and questions during the recent J.P. Morgan Healthcare Conference in San Francisco had to do with – among other matters – sales guidance from the firm, which is hardly alone in seeking treatments for the disease. Other players include Anavex Life Sciences Corp., Neurogene Inc. and Taysha Gene Therapies Inc.

Anavex rallies a bit after phase II/III failure

Jan. 3, 2024

By Lee Landenberger

Anavex Life Sciences Corp.’s stock (NASDAQ:AVXL) sunk 35% on Jan. 2 after a phase II/III study failure in Rett syndrome but posted a next-day rebound of 8% on Jan. 3 with shares closing at $6.53 each. Top-line data from the randomized, double-blind, placebo-controlled study of the company’s lead asset, Anavex 2-73 (blarcamesine), showed improvement but did not reach statistical significance on the co-primary endpoint, the Rett Syndrome Behaviour Questionnaire.