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Taysha Gene Therapies (TSHA)
NASDAQ:TSHA
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Taysha Gene Therapies
(NASDAQ:TSHA)
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Rating:53Neutral
Price Target:
$4.50
▼(-1.32% Downside)
Action:ReiteratedDate:03/19/26
TSHA’s score is held back primarily by sustained large losses and significant cash burn despite improved leverage and high gross margins. Positive earnings-call developments—strong clinical/regulatory progress and cash runway into 2028—partially offset these financial risks. Technically, indicators lean mildly weak/neutral, and valuation signals are limited due to negative earnings and no dividend.
Positive Factors
Regulatory Momentum
Breakthrough designation plus written FDA alignment on pivotal, ASPIRE and CMC comparability materially reduces development and regulatory uncertainty. Durable agency support to pool cross-trial data raises the probability of an efficient BLA pathway and allows management to optimize trial and manufacturing sequencing.
Cash Runway
A reported cash balance near $320M and management guidance to fund operations into 2028 meaningfully lowers near-term financing pressure. This multi-year runway supports completion of pivotal dosing, PPQ lots and initial commercial readiness activities, providing time to reach regulatory inflection points before seeking major new capital.
Favorable Unit Economics
A strong revenue rebound and very high gross margin (~95%) indicate favorable unit economics for the company’s therapeutics at current scale. While revenues remain small, sustained high margins suggest that, if approved, therapies could scale with meaningful operating leverage and attractive incremental profitability versus current cost base.
Negative Factors
Persistent Cash Burn
Multi-year negative operating and free cash flow of roughly -$93M TTM demonstrates persistent cash consumption from development activities. This structural burn rate makes the company reliant on external financing over the medium term, increasing dilution risk and creating execution dependence on successful trial outcomes and financing markets.
Large and Sustained Losses
Deep operating losses and an extremely negative net margin reflect structural unprofitability while programs remain pre-commercial. Persistent negative returns will continue to erode shareholder equity absent approvals or partnerships, constrain reinvestment flexibility, and make long-term value creation contingent on binary clinical and regulatory events.
Small Cohorts & Regulatory Timing Risk
Key pediatric safety data come from a three-patient ASPIRE cohort and the pivotal plan relies on pooling small cross-study cohorts. This structural reliance on limited sample sizes increases statistical uncertainty and gives the FDA reason to demand longer follow-up (e.g., 12 months), which could delay BLA timing and compress strategic optionality.
Taysha Gene Therapies (TSHA) vs. SPDR S&P 500 ETF (SPY)
Market Cap
$1.25B
Dividend YieldN/A
Average Volume (3M)2.43M
Price to Earnings (P/E)―
Beta (1Y)2.63
Revenue Growth-36.36%
EPS Growth-945.72%
CountryUS
Employees73
SectorHealthcare
Sector Strength45
IndustryBiotechnology
Share Statistics
EPS (TTM)-0.08
Shares Outstanding273,919,370
10 Day Avg. Volume2,649,383
30 Day Avg. Volume2,434,655
Financial Highlights & Ratios
PEG Ratio2.90
Price to Book (P/B)7.12
Price to Sales (P/S)179.93
P/FCF Ratio-18.74
Enterprise Value/Market Cap0.63
Enterprise Value/Revenue80.44
Enterprise Value/Gross Profit85.56
Enterprise Value/Ebitda-7.26
Forecast
1Y Price Target
$12.11Price Target Upside165.57% Upside
Rating ConsensusStrong Buy
Number of Analyst Covering9
EPS Forecast (FY)-0.44
Revenue Forecast (FY)$4.46M
Taysha Gene Therapies Business Overview & Revenue Model
Company DescriptionTaysha Gene Therapies, Inc., a gene therapy company, focuses on developing and commercializing adeno-associated virus-based gene therapies for the treatment of monogenic diseases of the central nervous system. It primarily develops TSHA-120 for the treatment of giant axonal neuropathy; TSHA-102 for the treatment of Rett syndrome; TSHA-121 for the treatment of CLN1 disease; TSHA-118 for the treatment of CLN1 disease; TSHA-105 foe the treatment of for SLC13A5 Deficiency; and TSHA-101 for the treatment of GM2 gangliosidosis. Taysha Gene Therapies, Inc. has a strategic partnership with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments. The company was incorporated in 2019 and is based in Dallas, Texas.
How the Company Makes MoneyAs a clinical-stage gene therapy company without approved products, Taysha has historically generated little to no recurring revenue from product sales; its operations have primarily been funded through capital raising and, where applicable, other non-product sources. Key ways the company can generate or report revenue/financing include: (1) Equity financing: issuing common stock and other equity-linked securities in public or private offerings to fund R&D and corporate operations. (2) Debt financing/credit facilities: borrowing (including convertible instruments, if used) to supplement cash, typically dependent on market conditions and balance sheet capacity. (3) Collaboration, licensing, or partnership income (if applicable in reported periods): upfront payments, milestone payments, cost reimbursements, or royalties from partnering its gene therapy programs, platform know-how, or regional rights with larger biopharma companies; if no such agreements are active or material, this source is null. (4) Grants and other income: research grants, government or nonprofit funding, or other ancillary income, if awarded; if not reported, this source is null. Overall, until commercialization, Taysha’s ability to "make money" in an operating sense is limited, and its financial results are primarily driven by R&D spending and its ability to raise external capital; any meaningful recurring earnings would typically depend on future regulatory approvals and subsequent product commercialization and/or monetization of programs via partnerships.
Taysha Gene Therapies Earnings Call Summary
Earnings Call Date:Mar 19, 2026
(Q4-2025)
| % Change Since: |
Next Earnings Date:May 20, 2026
Earnings Call Sentiment Positive
The call emphasized substantial clinical and regulatory progress — compelling Part A efficacy signals, initiation of the pivotal trial, FDA Breakthrough designation, written FDA alignments on trial designs and CMC comparability, and a clear commercial readiness plan — supported by a solid cash position into 2028. These positives are tempered by rising R&D and G&A expenses, a larger net loss in 2025, small sample sizes in key pediatric safety cohorts, and contingent regulatory timing that could require longer follow-up. On balance, the forward clinical and regulatory momentum materially outweighs the financial and operational risks discussed.Q4-2025 Updates
Positive Updates
Compelling Clinical Efficacy Signals in REVEAL Part A
REVEAL Part A reported a 100% response rate among the 10 treated patients across low- and high-dose cohorts, with an 83% response rate at six months in the high-dose group (5 of 6). Management reported an average of ~19 developmental milestone gains per patient from a 32-milestone set, sustained and deepening over time.
Pivotal and Pediatric Safety Trials Initiated with Favorable Tolerability
Initiated the REVEAL pivotal trial in 2025 and have dosed multiple patients with enrollment advancing; ASPIRE safety-focused trial cleared by FDA to enroll three females aged 2 to <4 years. No treatment-related serious adverse events or dose-limiting toxicities observed across REVEAL Phase 1/2 and pivotal trials as of the March 2026 data cutoff.
Regulatory Progress — FDA Breakthrough and Written Alignments
Received FDA Breakthrough Therapy designation for TSHA-102 and secured written FDA alignment on REVEAL pivotal and ASPIRE trial designs, a six-month interim analysis for the pivotal trial, as well as CMC comparability approach and PPQ strategy — enabling pooling of data across trials to support a BLA.
CMC and Manufacturing Alignment
FDA endorsed the proposed process performance qualification (PPQ) campaign, stability package, and potency assay strategy; management plans to initiate BLA-enabling PPQ lots in 2026 with completion of PPQ lots expected by year-end, supporting a streamlined BLA timeline alongside pivotal data.
Commercial Readiness and Market Opportunity
Market research showed high anticipated demand from clinicians and caregivers and a strong preference for intrathecal administration for scalability and outpatient delivery. Company estimates 15,000–20,000 Rett patients in U.S./EU/UK (6,000–9,000 in U.S.) and has begun building commercial leadership with the appointment of Brad Martin as SVP, Market Access and Value.
Cash Position and Near-Term Financing
As of 12/31/2025, cash and cash equivalents were $319.8 million. The company raised $50.0 million in Q4 2025 via its ATM program and expects current cash resources to fund planned operating expenses into 2028.
Planned Near-Term Data Milestones
Company expects a REVEAL Part A update in Q2 with 12 patients and a minimum of 12 months of follow-up, plus a six-month interim analysis for the REVEAL pivotal once all pivotal patients reach six months — providing multiple potential regulatory pathways toward BLA submission.
Negative Updates
Rising Operating Expenses
Research and development expenses increased to $86.4 million in 2025 from $66.0 million in 2024 — a $20.4 million increase (approximately +30.9%), driven by higher headcount, clinical trial and GMP expenses. General and administrative expenses rose to $33.9 million from $29.0 million — a $4.9 million increase (approximately +16.9%).
Widening Net Loss
Net loss increased to $109.0 million for 2025 versus $89.3 million in 2024, an increase of $19.7 million (about +22.1%). Loss per share improved slightly to $0.34 from $0.36 (approximately -5.6%), implying dilution/changes in share count despite a larger absolute loss.
Small Sample Sizes and Reliance on Extrapolation
Key planned pediatric safety data from ASPIRE will enroll only three patients aged 2 to <4 years, with efficacy in the 2 to <6 population to be extrapolated from REVEAL pivotal data. The pivotal design also relies on pooling limited-size datasets (e.g., Part A, pivotal, ASPIRE), which increases reliance on cross-study comparability and statistical assumptions.
Regulatory Timing Risk — Potential Need for Longer Follow-Up
Management noted possible scenarios where FDA could request 12-month data rather than accept a six-month interim for BLA filing, which could delay submission; while FDA alignment exists, filing timing remains contingent on the interim results and subsequent agency discussions.
Funding and Dilution Considerations
While $50.0 million was raised via ATM in Q4 2025 to support a potential commercial inventory build, using an ATM and having term loan-related costs recorded in G&A indicate ongoing financing needs and potential shareholder dilution as the company advances toward commercialization.
Competitive and Geographic Reimbursement Uncertainties
Management discussed potential read-throughs from other programs (e.g., CHMP opinions and competing entrants) and acknowledged typical European pricing pressure; uncertainties remain around EU reimbursement and pricing despite management optimism based on the data and high unmet need.
Company Guidance
On the March 19, 2026 call (full-year 2025 results) Taysha reiterated regulatory and development guidance: REVEAL pivotal was initiated in 2025 with multiple patients dosed and dosing expected to complete in 2026, a six‑month interim analysis (FDA‑aligned) is planned and could support a BLA filing (alternatively a 12‑month or rolling approach remains possible); ASPIRE will enroll three females aged 2 to <4 years, use a brain‑volume‑scaled high dose (equivalent to 1e15 total vector genomes), requires a minimum of 3 months’ safety data for the BLA, and is on track to complete dosing in 2026; the FDA agreed to pool data from REVEAL Part A/Phase 1/2, the pivotal and ASPIRE given CMC comparability, endorsed the PPQ/process validation and stability/potency plan (PPQ lots to initiate in 2026 and complete by year‑end), and the program holds Breakthrough Therapy designation. Clinical metrics highlighted: Part A earlier showed a 100% response rate in 10 treated patients, an 83% response at six months (5/6 high‑dose patients), ~19 average milestone gains per patient across 32 developmental milestones, a planned pivotal cohort of 15 patients aged 6 to <52 testing against a ~6.3–6.7% spontaneous‑improvement benchmark (spontaneous gains up to ~85% at ages 1–5); Q2 2026 will report comprehensive data on all 12 Part A pediatric patients with ≥12‑month follow‑up. Financial guidance/metrics: FY2025 R&D $86.4M, G&A $33.9M, net loss $109.0M ($0.34/sh) vs. $89.3M ($0.36) in FY2024, cash & equivalents $319.8M as of 12/31/2025 (plus $50M gross ATM raised in Q4) and management expects cash to fund operations into 2028; estimated addressable Rett population ~15,000–20,000 (U.S. ~6,000–9,000).Taysha Gene Therapies Financial Statement Overview
Summary
Revenue rebounded strongly and gross margin is very high, and leverage is now low-to-moderate (debt-to-equity ~0.32). However, the core profile is still dominated by very large operating losses (EBIT about -$110.5M; net margin about -1,584%) and heavy cash burn (TTM operating cash flow about -$93.1M; FCF about -$93.8M), implying continued reliance on external funding.Income Statement
TTM (Trailing-Twelve-Months) revenue rebounded strongly (+54.9%), and gross margin remains very high (TTM ~95%), reflecting favorable unit economics on a small revenue base. However, profitability is deeply negative: TTM net margin is about -1,584% with large operating losses (EBIT roughly -$110.5M) that have persisted for years. Overall, the business is showing top-line momentum but is still far from breakeven and remains highly dependent on funding.22
Negative
Balance Sheet
The balance sheet has improved materially versus prior years, with low-to-moderate leverage in TTM: debt is about $18.2M against equity of ~$246.9M (debt-to-equity ~0.32), a sharp improvement from 2022 when equity was very low and leverage was extreme. That said, returns remain deeply negative (TTM return on equity about -67%), indicating continued value erosion from ongoing losses. Asset base is sizable (~$343.3M), but sustained losses are the key risk to longer-term balance sheet strength.62
Positive
Cash Flow
Cash burn remains heavy: TTM operating cash flow is about -$93.1M and free cash flow about -$93.8M, continuing a multi-year pattern of significant negative cash generation. Free cash flow was modestly better than the prior annual period (TTM growth ~10.2%), and free cash flow roughly tracks net loss (close to 1x), suggesting losses are not being masked by large non-cash gains. Still, the company will likely need ongoing capital to fund operations unless burn declines meaningfully.28
Negative
| Breakdown | Dec 2025 | Dec 2024 | Dec 2023 | Dec 2022 | Mar 2022 |
|---|---|---|---|---|---|
Income Statement | |||||
| Total Revenue | 9.77M | 8.33M | 15.45M | 2.50M | 0.00 |
| Gross Profit | 0.00 | 8.33M | 15.45M | 15.00K | 0.00 |
| EBITDA | -110.50M | -87.95M | -105.20M | -159.73M | -172.60M |
| Net Income | -109.00M | -89.30M | -111.57M | -166.01M | -174.52M |
Balance Sheet | |||||
| Total Assets | 343.32M | 160.36M | 172.73M | 126.28M | 213.96M |
| Cash, Cash Equivalents and Short-Term Investments | 319.77M | 139.04M | 143.94M | 87.88M | 149.10M |
| Total Debt | 18.17M | 63.18M | 61.11M | 59.93M | 63.09M |
| Total Liabilities | 96.38M | 88.84M | 97.79M | 125.33M | 118.57M |
| Stockholders Equity | 246.94M | 71.53M | 74.94M | 949.00K | 95.38M |
Cash Flow | |||||
| Free Cash Flow | -93.83M | -81.60M | -80.39M | -113.26M | -138.60M |
| Operating Cash Flow | -93.09M | -81.22M | -73.02M | -88.39M | -117.04M |
| Investing Cash Flow | -604.00K | -363.00K | -7.35M | -24.93M | -21.55M |
| Financing Cash Flow | 274.59M | 76.68M | 136.39M | 52.10M | 39.08M |
Taysha Gene Therapies Technical Analysis
Positive
4.56
Price Trends
4.67
Negative
4.78
Negative
3.95
Positive
Market Momentum
-0.05
Negative
48.15
Neutral
48.01
Neutral
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For TSHA, the sentiment is Positive. The current price of 4.56 is below the 20-day moving average (MA) of 4.59, below the 50-day MA of 4.67, and above the 200-day MA of 3.95, indicating a neutral trend. The MACD of -0.05 indicates Negative momentum. The RSI at 48.15 is Neutral, neither overbought nor oversold. The STOCH value of 48.01 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Positive sentiment for TSHA.
Taysha Gene Therapies Risk Analysis
Taysha Gene Therapies disclosed 83 risk factors in its most recent earnings report. Taysha Gene Therapies reported the most risks in the "Tech & Innovation" category.
Finance & Corporate - Financial and accounting risks. Risks related to the execution of corporate activity and strategy
Latest Risks Added 0 New Risks
Taysha Gene Therapies Peers Comparison
UnderperformOutperform
Sector (51)
Name | Overall Rating | Market Cap | P/E Ratio | ROE | Dividend Yield | Revenue Growth | EPS Growth |
|---|---|---|---|---|---|---|---|
59 Neutral | $1.79B | -5.25 | -43.17% | ― | -16.50% | 33.02% | |
53 Neutral | $1.25B | -16.13 | -56.64% | ― | -36.36% | -945.72% | |
53 Neutral | $819.20M | -6.70 | -100.29% | ― | 5459.66% | -59.70% | |
51 Neutral | $7.86B | -0.30 | -43.30% | 2.27% | 22.53% | -2.21% | |
50 Neutral | $2.36B | -8.83 | -57.61% | ― | -4.44% | 63.58% | |
50 Neutral | $664.43M | -2.57 | -178.61% | ― | 647.13% | 26.79% | |
46 Neutral | $845.97M | -4.22 | -141.99% | ― | ― | 31.16% |
* Healthcare Sector Average
TSHA
Taysha Gene Therapies
4.56
2.92
178.05%
RLAY
Relay Therapeutics
10.00
6.94
226.80%
IMNM
Immunome
20.83
12.65
154.65%
SPRY
ARS Pharmaceuticals
8.25
-4.11
-33.25%
SANA
Sana Biotechnology
3.17
0.86
37.23%
PRME
Prime Medicine, Inc.
3.68
1.73
88.72%
Glossary
BuyA stock rated as a "Buy" is expected to perform better than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock is likely to deliver higher returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
HoldA stock rated as a "Hold" is expected to perform in line with the overall market or a specific benchmark. This rating indicates that the stock is neither particularly compelling nor unfavorable for investment. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
SellA stock rated as a "Sell" is expected to perform worse than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock may deliver lower returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
Disclaimer
This AI Analyst Stock Report is automatically generated by our AI systems using advanced algorithms and publicly available financial, technical, and market data. While the information provided aims to be accurate and insightful, it is intended for informational purposes only and should not be considered financial advice. Any content created by an AI (Artificial Intelligence) system may contain inaccuracies and/or contain errors. Investing in stocks carries inherent risks, and past performance is not indicative of future results. This report does not account for your personal financial circumstances, objectives, or risk tolerance. Always conduct your own research or consult with a qualified financial advisor before making investment decisions. The analysis and recommendations provided are based on historical and current data and may not fully reflect future market conditions or unexpected developments. Neither the creators of this report nor its affiliated entities guarantee the accuracy, completeness, or reliability of the information presented. Use this report at your own discretion and risk.