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Novartis, but exclusively focused on gene therapies for rare brain and nervous system disorders.

Like a clinical-stage Sarepta Therapeutics, developing gene therapies for a different range of severe neurological genetic conditions.

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• TSHA-120: An investigational gene therapy designed to deliver a functional *GAN* gene to treat Giant Axonal Neuropathy (GAN).
• TSHA-102: An investigational gene therapy aimed at delivering a functional *MECP2* gene to address the underlying cause of Rett Syndrome.
• TSHA-118: An investigational gene therapy intended to deliver a functional *PPT1* gene for the treatment of CLN1 disease, a severe lysosomal storage disorder.
• TSHA-101: An investigational gene therapy designed to deliver functional *HEXA* and *HEXB* genes to treat GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases).

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• Astellas Pharma Inc. (ALPMY)
  Astellas has an exclusive option and license agreement for Taysha's lead program, TSHA-102, a gene therapy for Rett syndrome. If Astellas exercises its option, it would assume responsibility for global development and commercialization.
• Sanofi (SNY)
  Sanofi acquired Taysha's GM2 gangliosidosis gene therapy program (TSHA-120) in 2024. This was an outright sale of the program and its associated intellectual property and assets.

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• Lonza (SWX: LONN)

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Sean Nolan, Chief Executive Officer and Chairman

Sean Nolan was appointed CEO of Taysha Gene Therapies in December 2022. He brings over 30 years of experience in the biopharmaceutical industry. Prior to Taysha, Nolan served as CEO of the gene therapy company AveXis, where he led the company through an initial public offering and transformed it into a fully integrated global organization. AveXis was subsequently acquired by Novartis. He currently holds the position of Executive Chairman at Jaguar Gene Therapy, a privately held early-stage gene therapy company, and is a board member for several other companies.

Kamran Alam, Chief Financial Officer and Corporate Secretary

Kamran Alam serves as the Chief Financial Officer and Corporate Secretary of Taysha Gene Therapies. With over two decades of experience in finance and strategic leadership within the pharmaceutical and other industries, he has a strong background in financial analysis, budgeting, and mergers & acquisitions. Alam previously served as VP Finance at AveXis, where he was instrumental in its acquisition by Novartis. Most recently, he was the Senior Vice President of Finance and Principal Financial Officer at Rocket Pharmaceuticals.

Sukumar Nagendran, M.D., President, Head of Research and Development, and Director

Dr. Sukumar Nagendran was appointed President and Head of Research and Development in December 2022. He is a physician, drug developer, and biotech executive with more than 30 years of experience in areas such as gene therapy development, clinical development strategy, medical affairs, and diagnostics. His previous roles include Chief Medical Officer and President of R&D at Jaguar Gene Therapy, and Chief Medical Officer and Senior Vice President at AveXis before its acquisition by Novartis. Dr. Nagendran has also held senior management positions at various pharmaceutical companies including Pfizer and Novartis.

Tracy M. Porter, Chief People Officer

Tracy M. Porter holds the title of Chief People Officer at Taysha Gene Therapies.

Emily McGinnis, Chief Patient and External Affairs Officer

Emily McGinnis serves as the Chief Patient and External Affairs Officer for Taysha Gene Therapies.

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The key risks to Taysha Gene Therapies (TSHA) largely revolve around its financial viability, the inherent challenges of clinical-stage gene therapy development, and intense market competition.

1. Financial Instability and Need for Additional Capital: Taysha Gene Therapies faces significant financial challenges, characterized by dismal financial metrics, a substantial accumulated deficit, and an unsustainable cash burn rate. The company has consistently reported heavy losses and negative profit margins, indicating a struggle to achieve operational profitability. With a projected cash runway extending to late 2026, there is a high likelihood of needing to raise additional capital, which could lead to shareholder dilution. This ongoing financial scrutiny reflects a disconnect between the company's valuation and its actual financial health.
2. High Clinical Development and Regulatory Risks: As a clinical-stage biotechnology company, Taysha Gene Therapies has no commercialized products, making its success highly dependent on the successful clinical development and regulatory approval of its pipeline assets, particularly TSHA-102 for Rett syndrome. The regulatory pathway for gene therapies is complex and demanding, with stringent safety and efficacy standards that can lead to delays or prevent commercialization. The company previously discontinued its TSHA-120 program due to feasibility challenges, underscoring the high binary risk associated with clinical trials. Unforeseen long-term side effects could also impact product viability.
3. Intense Competition and Market Pressures: Taysha operates within a highly competitive biotechnology sector, specifically in the rapidly evolving gene therapy landscape. Numerous companies are vying for market share, including those developing treatments for Rett syndrome. Breakthroughs in alternative therapies by competitors could reduce Taysha's potential market share. Furthermore, gene therapies often face significant pricing pressures and reimbursement challenges due to their high cost, which could limit market penetration.

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Taysha Gene Therapies (TSHA) is primarily focused on developing gene therapies for severe monogenic diseases of the central nervous system, with its lead clinical program, TSHA-102, targeting Rett syndrome.

TSHA-102 for Rett Syndrome

The global addressable market for Rett Syndrome is substantial. In 2024, the global Rett Syndrome market size was valued at approximately USD 380.91 million and is projected to reach USD 1.09 billion by 2033, growing at a compound annual growth rate (CAGR) of 12.4%. Other estimates place the global market size at USD 330 million in 2024, projected to grow to USD 1.368 billion by 2035 at a CAGR of 9.8%. North America is identified as the largest market for Rett Syndrome treatments, accounting for approximately 60% of the global market share. In the U.S. alone, there are over 10,000 patients affected by Rett syndrome. The prevalence is estimated to be between 15,000 and 20,000 patients across the U.S., EU, and UK.

Previously Deprioritized Programs

Taysha Gene Therapies has previously deprioritized several other gene therapy programs and transferred their rights to other entities or collaborators. While these are no longer Taysha's main products, market size information for some of the associated conditions includes:

• CDKL5 Deficiency Disorder (CDD): The global CDKL5 Deficiency Disorder market was valued at approximately USD 120 million in 2023 and is projected to reach USD 220 million by 2032. Another source indicates a global market size of US$ 111.4 million in 2024, expected to reach US$ 155.7 million by 2031. North America holds the largest market share for CDD. There are less than 4,500 individuals with CDKL5 deficiency in the United States.
• Batten Disease (including CLN1 and CLN7 Batten disease): The global Batten Disease Treatment Market is projected to grow from USD 426.42 million in 2024 to USD 634.3 million by 2032, with a CAGR of 5.09%. Another estimate valued the global market at around USD 40.2 million in 2023, predicted to grow to about USD 65.2 million by 2032. The worldwide prevalence of Batten disease is approximately 1 in 100,000 live births.
• Giant Axonal Neuropathy (GAN), GM2 gangliosidosis, SURF1-associated Leigh syndrome, and APBD: Market sizes for these specific conditions were not readily available in monetary terms. Taysha transferred the Investigational New Drug (IND) application for TSHA-120 in GAN to the National Institute of Neurological Disorders and Stroke (NINDS) and transferred rights for other programs back to their originating institutions.

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Taysha Gene Therapies (TSHA) is a clinical-stage biotechnology company, and as such, its future revenue growth over the next 2-3 years will be primarily driven by the advancement and potential commercialization of its lead gene therapy candidate, TSHA-102, for Rett Syndrome.

Expected Drivers of Future Revenue Growth:

1. Regulatory Approval and Commercial Launch of TSHA-102 for Rett Syndrome: The most significant driver of future revenue growth for Taysha Gene Therapies is the successful regulatory approval and subsequent commercial launch of TSHA-102. The company has made significant progress in its REVEAL clinical trials for TSHA-102, which has demonstrated encouraging results and a generally well-tolerated safety profile in pediatric, adolescent, and adult patients. TSHA-102 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, which are expected to expedite its development and review process. Taysha is preparing for a pivotal trial, with the first patient dosing occurring in Q4 of 2025, and a 6-month interim analysis may accelerate the Biologics License Application (BLA) submission by at least two quarters. Longer-term data are anticipated in the first half of 2026. The company anticipates beginning to dose younger patients (2-5 years old) in the middle of 2026, aiming for a broad label that includes this population upon BLA submission.
2. Expansion of TSHA-102 Indication to Broader Patient Populations: Beyond the initial target population, the potential to expand the use of TSHA-102 to a broader range of Rett Syndrome patients, including younger age groups (2-5 years old), represents a key growth driver. The company's strategy includes the inclusion of this younger population in the BLA submission. Positive clinical data across various age groups would support a wider label and increased market penetration.
3. Global Market Expansion for TSHA-102: Taysha Gene Therapies has regained full global rights to the TSHA-102 program, which positions the company to pursue commercialization beyond the United States. While initial focus will likely be on major markets like the U.S., successful regulatory approvals and launches in the EU and other significant regions for Rett Syndrome patients would substantially contribute to revenue growth over the next 2-3 years and beyond. The estimated patient population for Rett syndrome caused by a pathogenic MECP2 mutation is between 15,000 and 20,000 patients in the U.S., EU, and U.K.
4. Advancement of Other Pipeline Candidates (Longer-Term Potential): While TSHA-102 is the primary near-term driver, successful advancement of other gene therapy candidates in Taysha's pipeline could provide additional revenue streams in the longer term. However, within the next 2-3 years, Taysha has streamlined its pipeline, focusing efforts on TSHA-102 and out-licensing or transferring rights for other deprioritized programs (such as TSHA-120, TSHA-101, TSHA-104, and TSHA-112) to other entities for continued development. Therefore, significant revenue from new product launches from other pipeline candidates is less likely within the immediate 2-3 year timeframe.

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Share Issuance

• Taysha Gene Therapies has experienced a substantial increase in its shares outstanding, growing from 63.2 million in 2022 to approximately 273.92 million by November 2025.
• In August 2023, the company raised approximately $150 million through a Post-IPO funding round.
• In June 2024, Taysha announced an underwritten public offering of common stock and pre-funded warrants, which was expected to generate gross proceeds of approximately $75 million.

Inbound Investments

• Taysha Gene Therapies' successful Initial Public Offering (IPO) in September 2020 provided significant initial capital for its early research and development efforts.
• The company entered into a strategic collaboration and investment agreement with Astellas Pharma in 2022, securing $50 million upfront.
• A Post-IPO funding round in August 2023 brought in approximately $150 million from various investors, including Perceptive Advisors, RA Capital Management, RTW Investments, and Casdin Capital.

Capital Expenditures

• Capital expenditures amounted to approximately $20.6 million in 2022.
• In 2023, capital expenditures were approximately $3.9 million.
• For 2024 and 2025 (year-to-date), capital expenditures were relatively low, reported at approximately $0.4 million for each year.