Neurogene plummets 35% on gene therapy trial data for Rett syndrome
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Nov 11 2024
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Neurogene's Stock Reaction: Neurogene's stock fell approximately 35% after releasing interim phase 1/2 data for its gene therapy NGN-401, which showed promising results in low doses but raised concerns due to a serious adverse event in a high-dose patient.
Competitor Performance: Taysha Gene Therapies, developing a competing treatment for Rett syndrome, TSHA-102, saw its stock rise about 28% following Neurogene's announcement.
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Analyst Views on TSHA
Wall Street analysts forecast TSHA stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for TSHA is 11.60 USD with a low forecast of 8.00 USD and a high forecast of 19.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
11 Analyst Rating
11 Buy
0 Hold
0 Sell
Strong Buy
Current: 4.570
Low
8.00
Averages
11.60
High
19.00
Current: 4.570
Low
8.00
Averages
11.60
High
19.00
About TSHA
Taysha Gene Therapies Inc is a clinical-stage biotechnology company, which is focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. The Company’s lead clinical program, TSHA-102, is in development for the treatment of Rett syndrome, a rare neurodevelopmental disorder. The Company is evaluating TSHA-102 in the REVEALPhase I/II adolescent and adult clinical trial, which is a first-in-human, open-label, randomized, dose escalation and dose-expansion, multicenter study evaluating the safety and preliminary efficacy of TSHA-102 in female patients aged 12-years and older with Rett syndrome. It has acquired a worldwide right to a clinical-stage, intrathecally dosed AAV9 gene therapy program, TSHA-120, for the treatment of giant axonal neuropathy (GAN). TSHA-105 is a gene replacement therapy in development for the treatment of SLC13A5 deficiency, a rare autosomal recessive epileptic encephalopathy.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Taysha Gene Therapies Doses First Patient in TSHA-102 Pivotal Trial for Rett Syndrome
- Trial Progress: Taysha Gene Therapies successfully dosed the first patient in the TSHA-102 pivotal trial in Q4 2025, with plans to complete dosing for all 15 patients by Q2 2026, marking a significant milestone in the treatment of Rett syndrome.
- FDA Alignment: The company reached written alignment with the FDA to include three months of safety data from the ASPIRE trial in its planned BLA submission, aiming to enable broad labeling for patients aged two and older, which will expedite the path to market.
- Market Opportunity: With an estimated 15,000 to 20,000 patients affected by Rett syndrome across the U.S., EU, and U.K., Taysha aims to redefine the treatment paradigm for this high unmet need disease through compelling clinical data from TSHA-102, presenting a significant market opportunity.
- Long-term Data Update: An update on long-term safety and efficacy data from the REVEAL trial is expected in H1 2026, which will provide crucial support for the company's future treatment strategies and enhance its competitive position in the market.
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Taysha Grants 212,000 Stock Options to New Employees
- Employee Incentive Program: On December 3, 2025, Taysha Gene Therapies granted 212,000 stock options to two new employees at an exercise price of $4.49 per share, aimed at attracting and retaining key talent, thereby enhancing the company's competitive edge in the gene therapy sector.
- Option Structure: These options will be valid for 10 years and vest over four years, with 25% vesting on the first anniversary and the remaining 75% vesting in equal monthly installments over the subsequent 36 months, ensuring continued employee contributions during their tenure.
- Compliance Assurance: The stock option grant complies with Nasdaq Listing Rule 5635(c)(4), demonstrating the company's commitment to transparency and compliance in talent acquisition, which can bolster investor confidence.
- Strategic Goals: Taysha focuses on developing gene therapies for severe monogenic diseases, particularly its clinical program TSHA-102 for Rett syndrome, and aims to accelerate research and development through these incentive measures to address unmet medical needs.
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