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Representative image of neuroscience and gene therapy research, reflecting Taysha Gene Therapies’ TSHA-102 Rett syndrome update and the race to develop disease-modifying treatments for rare neurodevelopmental disorders.

Pharma & Biotech

Why Taysha’s Rett syndrome data could become a major gene therapy test case

by Soujanya Ravi
June 22, 2026
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Find out how Taysha’s TSHA-102 Rett syndrome update could shape gene therapy development, BLA planning and future disease-modifying care.

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POPULAR NEWS

Representative image of a clinician reviewing skeletal imaging, illustrating Mirum Pharmaceuticals and Incyte’s zilurgisertib Phase 2 progress in fibrodysplasia ossificans progressiva and the rare disease treatment questions facing regulators.

Pharma & Biotech

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Niagen Bioscience’s NB4168 has secured U.S. FDA Rare Pediatric Disease and European orphan designations as the company advances the experimental ataxia telangiectasia therapy toward human trials. Representative image.

Pharma & Biotech

Niagen wins twin rare-disease designations, but can NB4168 turn nicotinamide riboside evidence into a medicine?

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Representative image of biopharma laboratory research. Gilead Sciences and Merck’s latest Phase 3 updates highlight the promise of once-weekly HIV treatment with islatravir and lenacapavir, while raising fresh questions over Trodelvy’s lung cancer expansion strategy.

Pharma & Biotech

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clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immuno-oncology immunotherapy Inc. Johnson & Johnson medical devices oncology precision oncology rare disease U.S. Food and Drug Administration United States Food and Drug Administration

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