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Jun 30, 2022
Taysha Gene Therapies Q2 2022 Earnings Report
Reported financial results for the second quarter ended June 30, 2022, and provided a corporate update.
Key Takeaways
Taysha Gene Therapies reported progress in giant axonal neuropathy (GAN) with TSHA-120, demonstrating durable neurophysiological improvements. Positive feedback from MHRA supports regulatory strategy and manufacturing approach. Preclinical data for TSHA-102 in Rett syndrome showed near normalization of survival and behavior in neonatal knockout Rett mice.
TSHA-120-treated patients with GAN demonstrated durable neurophysiological improvements in the sensory nerve action potential (SNAP), a definitive clinical endpoint, compared to rapid and irreversible decline in sensory function early in life in untreated patients based on natural history.
Pathology from nerve biopsies in all evaluable samples confirmed the presence of regenerative nerve fibers in 100% of TSHA-120-treated patients (n=11).
TSHA-120 commercial grade and clinical trial material considered comparable across all key quality attributes as assessed by an extensive panel of release assays and next-generation sequencing.
Late-breaking positive preclinical data for TSHA-102 in Rett syndrome demonstrated near normalization of survival and normalization of behavior in neonatal knockout Rett mice, a model significantly more severe than the human phenotype
Taysha Gene Therapies
Taysha Gene Therapies
Forward Guidance
Taysha anticipates further regulatory update, including from FDA, for TSHA-120 in GAN and first-in-human preliminary Phase 1/2 data for TSHA-102 in Rett syndrome by year-end 2022.
Positive Outlook
- Further regulatory update, including from FDA, for TSHA-120 in GAN by year-end 2022
- First-in-human preliminary Phase 1/2 data for TSHA-102 in Rett syndrome by year-end 2022