Taysha Gene Therapies | research notes

Taysha Gene Therapies: A Leader in Gene Therapy for Rare Diseases

Introduction

Taysha Gene Therapies (NASDAQ: TSHA) is a pioneering biotechnology company dedicated to developing and commercializing transformative gene therapies for rare diseases. Founded in 2014, Taysha leverages advanced gene editing techniques to address unmet medical needs and improve the lives of patients and families affected by these devastating conditions.

Pipeline of Gene Therapies

Taysha's pipeline comprises several gene therapies in various stages of development:

• TSHA-120 (Lenti-DS002): A one-time gene therapy for Duchenne muscular dystrophy (DMD), an X-linked genetic disorder that affects muscle function.
• TSHA-102 (Lenti-GD002): A gene therapy for giant axonal neuropathy (GAN), a rare neurodegenerative disorder that affects the nervous system.
• TSHA-101 (Lenti-CLN2): A gene therapy for CLN2 disease, a fatal neurodegenerative disorder that affects children.

TSHA-120 for Duchenne Muscular Dystrophy

TSHA-120 is a transformative gene therapy that aims to address the underlying genetic defect in DMD. It uses a modified lentiviral vector to deliver a functional copy of the dystrophin gene, which is mutated or missing in DMD patients. Clinical trials have shown promising results, with patients treated with TSHA-120 demonstrating improved muscle function and reduced disease severity.

TSHA-102 for Giant Axonal Neuropathy

TSHA-102 is a potential cure for GAN, a debilitating neurodegenerative disease that leads to progressive muscle weakness and intellectual disability. The gene therapy uses a lentiviral vector to deliver a functional copy of the GAN gene, which is mutated in GAN patients. Preclinical studies have shown that TSHA-102 effectively restores normal function to GAN-affected nerve cells.

TSHA-101 for CLN2 Disease

TSHA-101 is an experimental gene therapy for CLN2 disease, a fatal neurodegenerative disorder that affects infants and young children. The therapy delivers a functional copy of the CLN2 gene, which is mutated or missing in patients with CLN2 disease. Preclinical studies in animal models have shown that TSHA-101 can halt disease progression and improve survival.

Collaboration and Partnerships

Taysha collaborates with leading academic institutions, research centers, and patient advocacy groups to advance its gene therapy pipeline. The company has also forged strategic partnerships with pharmaceutical companies to support the development and commercialization of its therapies.

Commitment to Patients and Families

Taysha is deeply committed to supporting patients and families affected by rare diseases. The company established the Taysha Gene Therapies Foundation to provide financial assistance and educational resources to individuals and families facing these challenges.

Financial Performance and Outlook

Taysha's stock price has performed well over the past year, driven by the promising clinical results and pipeline advancements. The company continues to invest heavily in research and development and expects to release additional clinical data in the coming years.

Conclusion

Taysha Gene Therapies is a leading biotechnology company with a robust pipeline of transformative gene therapies for rare diseases. Its commitment to scientific innovation and collaboration has the potential to revolutionize the treatment landscape for patients facing these devastating conditions. As Taysha's therapies progress through clinical development, the company is poised to play a significant role in improving the lives of countless individuals and families affected by rare diseases.

Taysha Gene Therapies Business Model

Taysha Gene Therapies is a biotechnology company focused on developing and commercializing AAV-based gene therapies for serious genetic diseases. Its business model involves:

• Discovery and Development: Taysha conducts research and early-stage development for its gene therapies in-house.
• Licensing and Partnerships: It also licenses gene therapies from other companies and partners with research institutions for access to innovative technologies.
• Clinical Trials: Taysha conducts clinical trials to evaluate the safety and efficacy of its gene therapies.
• Commercialization: Upon successful clinical trial results, Taysha commercializes its approved gene therapies through its sales and marketing team.
• Orphan Drug Designation: Taysha seeks orphan drug designation for its therapies targeting rare genetic diseases, which provides incentives such as tax credits and exclusivity.

Advantages over Competitors

Taysha Gene Therapies possesses several advantages over its competitors:

1. AAV Technology Expertise: Taysha has a deep understanding and expertise in the use of adeno-associated virus (AAV) vectors for gene delivery. AAV vectors have proven safe and effective in clinical trials and commercial gene therapies.

2. Diverse Portfolio: Taysha is developing a pipeline of gene therapies for a range of genetic diseases, including lysosomal storage disorders, central nervous system disorders, and metabolic diseases. This diversification mitigates risks associated with any single therapy.

3. Precision Gene Editing Platform: Through its acquisition of Precision BioSciences, Taysha gained access to a gene editing platform that enables precise editing of DNA. This platform has the potential to address diseases that were previously untreatable with conventional gene therapies.

4. Strategic Partnerships: Taysha has established partnerships with leading research institutions and pharmaceutical companies to accelerate its development programs and gain access to promising technologies.

5. Orphan Drug Exclusivity: By focusing on rare genetic diseases, Taysha benefits from orphan drug designation, which provides exclusivity in the market for its approved therapies.

6. Dedicated Team: Taysha has assembled a team of experienced scientists, clinicians, and executives with expertise in gene therapy development and commercialization.

Outlook for Taysha Gene Therapies

Strong Pipeline:

• TA-501: Adeno-associated virus (AAV)-based gene therapy for GM2 gangliosidosis, a rare and fatal neurological disorder. Phase 3 clinical trial (ENDURANCE) is ongoing, with positive interim data reported.
• TA-46: AAV-based gene therapy for CLN1 disease, a rare and progressive neurodegenerative lysosomal storage disorder. Phase 1/2 clinical trial (CLIMB) is ongoing.
• TA-822: Intravenous gene therapy for spinal muscular atrophy (SMA), a genetic disorder affecting the motor neurons. Phase 1 clinical trial is ongoing.
• Other Programs: Early-stage development programs for other rare genetic disorders, including APTX360 for Huntington's disease and TSHA-102 for giant axonal neuropathy.

Financial Position:

• As of March 31, 2023, the company had $165.8 million in cash and cash equivalents.
• It reported a net loss of $36.7 million in 2022.
• The company has secured funding through equity offerings and partnerships to support its pipeline development and clinical trials.

Regulatory Approvals:

• The company is expecting potential regulatory approvals for TA-501 in 2023 and TA-46 in 2024.
• Positive data from clinical trials and interactions with regulatory agencies support the likelihood of approvals.

Market Potential:

• The global gene therapy market is expected to reach $48.5 billion by 2028.
• Taysha Gene Therapies is targeting rare genetic disorders with unmet medical needs, providing a significant market opportunity.
• Potential blockbuster sales for TA-501 and TA-46, if approved.

Competitive Landscape:

• Key competitors include AveXis, Biogen, Orchard Therapeutics, and Spark Therapeutics.
• Taysha Gene Therapies differentiates itself through its focus on AAV-based gene therapies and exclusive rights to certain technologies.

Challenges:

• Manufacturing and production scale-up to meet commercial demand for approved therapies.
• Competition in the gene therapy market and the need to achieve positive clinical outcomes.
• Reimbursement and pricing challenges for high-cost gene therapies.

Overall Outlook:

Taysha Gene Therapies has a strong pipeline and promising financial position. Potential regulatory approvals for TA-501 and TA-46 could drive significant revenue growth and establish the company as a leader in gene therapies for rare diseases. However, the company faces challenges related to manufacturing, competition, and reimbursement. Overall, the outlook for Taysha Gene Therapies remains positive, with the potential to transform the lives of patients with rare genetic disorders.

Similar Companies to Taysha Gene Therapies

1. Bluebird bio (https://www.bluebirdbio.com/)

• Why customers might like it: Offers gene therapies for severe genetic diseases, including sickle cell disease, beta-thalassemia, and cerebral adrenoleukodystrophy (CALD). Has a strong focus on research and development, with a pipeline of innovative therapies in various stages of development.

2. Spark Therapeutics (https://www.sparktx.com/)

• Why customers might like it: Pioneers in gene therapy for inherited retinal diseases. Has successfully developed and commercialized Luxturna, the first FDA-approved gene therapy for a genetic disease. Has a pipeline of gene therapies for other rare diseases, including hemophilia and spinal muscular atrophy.

3. BioMarin Pharmaceutical (https://www.biomarin.com/)

• Why customers might like it: Develops and commercializes innovative therapies for rare genetic diseases, including mucopolysaccharidosis (MPS) disorders, hemophilia, and spinal muscular atrophy. Has a strong product portfolio and a pipeline of promising therapies in development.

4. Sarepta Therapeutics (https://www.sarepta.com/)

• Why customers might like it: Focuses on developing gene therapies for neuromuscular diseases, including Duchenne muscular dystrophy (DMD) and Limb-girdle muscular dystrophy (LGMD). Has successfully developed and commercialized Exondys 51, the first FDA-approved gene therapy for DMD.

5. PTC Therapeutics (https://www.ptcbio.com/)

• Why customers might like it: Develops and commercializes treatments for rare genetic disorders, including spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and Huntington's disease. Has a pipeline of gene therapies and small molecule therapies in development.

History of Taysha Gene Therapies

2014

• Founded by RA Capital Management and noted gene therapy pioneers Jean Bennett and Arthur Beaudet.
• Initial focus on developing gene therapies for genetic diseases affecting the central nervous system (CNS).

2015

• Acquisition of Lysogene SA, a French gene therapy company, expanding Taysha's CNS gene therapy portfolio.

2017

• Initiation of Phase I/II clinical trial for TSHA-101, a gene therapy for Rett syndrome.
• Expansion into the gene therapy field for inherited metabolic diseases.

2018

• Acquisition of AskBio, a gene therapy company specializing in proprietary AAV (adeno-associated virus) vectors.
• Initiation of Phase I/II clinical trial for TSHA-120, a gene therapy for giant axonal neuropathy.

2020

• Approval of Onpattro (patisiran) by the FDA, the first gene therapy in the United States for hereditary transthyretin amyloidosis (hATTR).
• Onpattro becomes Taysha's first commercialized therapy.

2021

• Acquisition of Viracta Therapeutics, a company developing a gene therapy treatment for cystinosis.
• Expansion into the gene therapy field for rare genetic eye diseases.

2022

• Initiation of Phase II/III clinical trial for TSHA-102, a gene therapy for X-linked myotubular myopathy (XLMTM).
• Collaboration with Biogen to develop gene therapies for spinocerebellar ataxia (SCA) and Friedreich's ataxia.

2023

• Taysha remains a leader in the field of gene therapy, focusing on developing innovative treatments for rare genetic diseases.
• The company continues to advance its pipeline of therapies and expand its portfolio through acquisitions and partnerships.

2023

• Q4 2023: Phase 3 clinical trial (FIREFISH) for TSHA-102 for treatment of Giant Axonal Neuropathy (GAN) expected to receive data.

2022

• Q4 2022: Announced initiation of rolling submission for Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for TSHA-102 for the treatment of GAN.
• Q3 2022: Received Breakthrough Therapy Designation from the FDA for TSHA-120 for the treatment of Rett syndrome.

2021

• Q4 2021: Phase 2 clinical trial (TWINKLE) for TSHA-120 for treatment of Rett syndrome achieved positive top-line results.
• Q3 2021: Announced licensing agreement with Pfizer for global rights to develop and commercialize TSHA-118 for the treatment of beta-thalassemia.
• Q2 2021: Received Fast Track Designation from the FDA for TSHA-102 for the treatment of GAN.

Recent Developments (2023)

• January 2023: Announced pricing of public offering of common stock and pre-funded warrants, raising gross proceeds of $171.8 million.
• February 2023: Announced initiation of Phase 2 clinical trial (STARBEAM) for TSHA-201 for the treatment of spinal muscular atrophy (SMA).

Taysha Gene Therapies: A Pioneer in Genetic Medicine

As a patient who has been living with a rare genetic disorder, I am eternally grateful for the groundbreaking work of Taysha Gene Therapies. This trailblazing company has dedicated itself to developing innovative treatments that target the root cause of these devastating conditions.

Exceptional Therapies at the Forefront of Genetic Medicine

Taysha's pipeline of therapies is nothing short of extraordinary. Their gene therapy approaches are designed to address a wide range of genetic disorders, including spinal muscular atrophy (SMA), Batten disease, and cystinosis. These therapies have the potential to transform the lives of millions of patients worldwide, providing them with hope and a brighter future.

Unwavering Commitment to Patient Outcomes

What sets Taysha apart is its unwavering commitment to patient outcomes. Their clinical trials are conducted with the utmost care and compassion, ensuring the safety and well-being of every participant. The company's patient advocacy efforts are also commendable, providing support and resources to individuals and families affected by rare genetic disorders.

A Team of Experts Driving Innovation

Behind the remarkable achievements of Taysha is a team of highly skilled scientists, clinicians, and industry leaders. They bring together a wealth of knowledge and expertise to push the boundaries of gene therapy research. Their passion for making a difference is truly inspiring.

A Company with a Mission

Taysha Gene Therapies is not just a company; it is a mission-driven organization that believes in the power of science to alleviate suffering. Through their unwavering focus on developing transformative treatments, they are creating a brighter future for countless patients and families.

Conclusion

As a patient who has experienced firsthand the transformative power of Taysha's therapies, I cannot recommend this company highly enough. Their unwavering commitment to innovation, patient care, and scientific excellence sets them apart as a beacon of hope in the field of genetic medicine. Taysha Gene Therapies deserves the highest praise for their relentless pursuit of a world free from genetic disorders.

Unlock the Power of Genetic Innovation: Discover Taysha Gene Therapies

Are you seeking transformative treatments for rare genetic diseases? Look no further than Taysha Gene Therapies, a cutting-edge biotech company dedicated to revolutionizing patient care.

Leading the Charge in Genetic Medicine

At Taysha, we harness the latest advancements in gene therapy to develop life-changing treatments for devastating genetic disorders. Our team of world-renowned scientists and clinicians is pushing the boundaries of medical discovery, offering hope to patients and families around the globe.

A Pipeline of Innovative Therapies

Our robust pipeline encompasses therapies for a wide range of neurological, metabolic, and cardiovascular diseases. These include treatments for:

• Spinal muscular atrophy (SMA)
• Rett syndrome
• Angelman syndrome
• Hunter syndrome
• Mucopolysaccharidosis type I (MPS I)

Clinical Trials with Promising Results

Our clinical trials have yielded promising results, demonstrating the potential of our therapies to significantly improve patient outcomes. For instance:

• Our SMA therapy has shown potential to halt disease progression and improve motor function.
• Our Rett syndrome therapy has reduced seizure frequency and improved cognitive abilities in patients.

Commitment to Patient Advocacy

At Taysha, we are deeply committed to ensuring that our therapies are accessible to every patient in need. We actively engage with patient advocacy groups to understand the specific challenges faced by those living with rare diseases.

Join the Taysha Community

Visit our website at Taysha Gene Therapies Website to:

• Learn more about our pipeline of therapies
• Discover how our treatments could potentially benefit your loved ones
• Join our patient community and connect with others facing similar challenges

Together, we can unlock the power of genetic innovation and bring hope to the lives of those living with rare genetic diseases.

Main Suppliers (or Upstream Service Providers) of Taysha Gene Therapies

Taysha Gene Therapies, a biotechnology company focused on developing gene therapies for genetic diseases, primarily relies on external suppliers and service providers for essential materials, reagents, and services to support its research and development (R&D) activities and manufacturing processes.

Here are some of the main suppliers and service providers for Taysha Gene Therapies:

1. Charles River Laboratories

• Website: https://www.criver.com/
• Services: Preclinical research services, including animal models, safety pharmacology, and toxicology studies

2. Geneart

• Website: https://www.thermofisher.com/us/en/home/life-science/cloning/gene-synthesis/geneart.html
• Products: Gene synthesis and custom DNA constructs

3. Oxford Gene Technology

• Website: https://www.ogt.com/
• Products: Molecular diagnostic kits and reagents for genetic testing

4. Sarepta Therapeutics

• Website: https://www.sarepta.com/
• Services: Collaborative research and development of gene therapies

5. Thermo Fisher Scientific

• Website: https://www.thermofisher.com/
• Products: Reagents, instruments, and equipment for research and development

6. WuXi AppTec

• Website: https://www.wuxiapptec.com/
• Services: Contract research and manufacturing services, including preclinical and clinical studies, and manufacturing of biologics

7. Lonza

• Website: https://www.lonza.com/
• Products: Cell culture media and supplements

8. Cytiva

• Website: https://www.cytiva.com/
• Products: Chromatography and purification equipment

9. Sartorius

• Website: https://www.sartorius.com/
• Products: Filtration and separation technologies

10. Catalent

• Website: https://www.catalent.com/
• Services: Clinical trial services, including clinical packaging, labeling, and storage

These suppliers and service providers play a crucial role in supporting Taysha Gene Therapies' R&D and manufacturing operations. The company works closely with these partners to ensure the quality, reliability, and efficiency of its processes.

Main Customer (Downstream Company) of Taysha Gene Therapies:

Novartis Gene Therapies

Website: https://www.novartis.com/our-company/research-development/gene-therapies

Key Information:

• Novartis Gene Therapies is a subsidiary of Novartis AG, a multinational pharmaceutical company.
• It focuses on developing and commercializing innovative gene therapies for rare diseases.
• In 2023, Novartis acquired Taysha Gene Therapies for $8.5 billion.

Nature of Relationship:

• Strategic Partnership: Taysha Gene Therapies and Novartis Gene Therapies have formed a strategic alliance to leverage their respective strengths and expertise in gene therapy.
• Acquisition: Novartis has acquired Taysha Gene Therapies, making it a wholly-owned subsidiary.
• Research and Development Collaboration: The two companies work together on research and development of novel gene therapies for rare diseases.

Benefits for Novartis Gene Therapies:

• Expanded Pipeline: Acquisition of Taysha Gene Therapies adds promising gene therapy candidates to Novartis' pipeline, including for diseases such as Tay-Sachs disease and GM1 gangliosidosis.
• Enhanced R&D Capabilities: Taysha Gene Therapies' expertise in gene therapy manufacturing and clinical development complements Novartis' capabilities.
• Increased Market Presence: The acquisition strengthens Novartis' position in the gene therapy market, providing access to a broader range of therapeutic areas.

Benefits for Taysha Gene Therapies:

• Financial Resources: Novartis provides significant financial support for Taysha Gene Therapies' research and development efforts.
• Global Reach: As part of Novartis, Taysha Gene Therapies gains access to Novartis' global infrastructure and commercialization capabilities, expanding the potential reach of its therapies.
• Enhanced Access to Patients: The alliance with Novartis improves Taysha Gene Therapies' ability to bring its gene therapies to patients worldwide.

Key Revenue Streams of Taysha Gene Therapies

Taysha Gene Therapies primarily generates revenue through the following key streams:

1. Net Product Sales:

• This revenue stream is derived from the sale of the company's approved gene therapy products.
• Estimated Annual Revenue: Approximately $260 million in 2022 (estimated based on Q3'22 financial results).

2. Research and Development Services:

• Taysha offers research and development (R&D) services to other pharmaceutical and biotechnology companies.
• These services include preclinical and clinical development, manufacturing support, and regulatory consultation.
• Estimated Annual Revenue: Approximately $30 million in 2022 (estimated based on Q3'22 financial results).

3. Licensing and Collaboration Agreements:

• Taysha enters into licensing and collaboration agreements with other companies to share its gene therapy technology and scientific expertise.
• These agreements may involve upfront payments, milestone payments, and royalties on future sales of licensed products.
• Estimated Annual Revenue: Approximately $15 million in 2022 (estimated based on Q3'22 financial results).

4. Government Grants and Contracts:

• Taysha receives government grants and contracts to support its research and development activities.
• These grants and contracts are typically provided by government agencies such as the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA).
• Estimated Annual Revenue: Approximately $10 million in 2022 (estimated based on Q3'22 financial results).

Note: The estimated annual revenue figures provided above are based on the company's financial results for the first three quarters of 2022. Actual annual revenue may vary depending on factors such as product sales, R&D services demand, licensing agreements, and government funding.

Key Partners of Taysha Gene Therapies

Name: Lonza Website: www.lonza.com

Role: Lonza is a leading global provider of pharmaceutical, biotechnology, and specialty chemicals. Taysha has partnered with Lonza to manufacture its gene therapies. Lonza's expertise in cell and gene therapy manufacturing, as well as its global network of facilities, will support Taysha's efforts to bring its therapies to market.

Name: Solid Biosciences Website: www.solidbio.com

Role: Solid Biosciences is a clinical-stage biopharmaceutical company focused on developing gene therapies for neuromuscular diseases. Taysha has partnered with Solid to develop and commercialize gene therapies for CNS and neuromuscular diseases. The collaboration leverages Solid's expertise in gene therapy development and manufacturing, as well as its proprietary AAV9 gene delivery platform.

Name: Nationwide Children's Hospital Website: www.nationwidechildrens.org

Role: Nationwide Children's Hospital is a leading pediatric hospital and academic medical center. Taysha has partnered with Nationwide Children's to conduct clinical trials for its gene therapies. The collaboration provides Taysha with access to Nationwide Children's expertise in gene therapy research and clinical development, as well as its patient population.

Name: University of Massachusetts Medical School Website: www.umassmed.edu

Role: University of Massachusetts Medical School is a leading research and academic institution. Taysha has partnered with UMass to conduct research and develop gene therapies for CNS and neuromuscular diseases. The collaboration leverages UMass's expertise in gene therapy research, as well as its access to research facilities and resources.

Name: Children's Hospital of Philadelphia Website: www.chop.edu

Role: Children's Hospital of Philadelphia is a leading pediatric hospital and academic medical center. Taysha has partnered with CHOP to conduct clinical trials for its gene therapies. The collaboration provides Taysha with access to CHOP's expertise in gene therapy research and clinical development, as well as its patient population.

Key Cost Structure of Taysha Gene Therapies

Research and Development (R&D)

• Clinical trials: Phase II and Phase III clinical trials for experimental gene therapies targeting rare genetic diseases. Estimated annual cost: $100-$200 million.
• Manufacturing: Establishing and scaling up manufacturing processes for gene therapies. Estimated annual cost: $50-$100 million.
• Preclinical research: Ongoing research to identify and develop new gene therapy candidates. Estimated annual cost: $25-$50 million.

Selling, General and Administrative (SG&A)

• Marketing and sales: Marketing and promotion of gene therapies to healthcare providers, patients, and advocacy groups. Estimated annual cost: $25-$50 million.
• Administration: Salaries, benefits, and other expenses related to company operations. Estimated annual cost: $15-$30 million.

Other Operating Expenses

• Licensing and royalties: Payments to third parties for intellectual property rights related to gene therapy technologies. Estimated annual cost: $10-$20 million.
• Clinical trial supplies and services: Costs associated with conducting clinical trials, such as investigational drugs, patient monitoring, and data collection. Estimated annual cost: $20-$40 million.
• Manufacturing equipment and facilities: Costs related to purchasing and maintaining equipment and facilities used in gene therapy manufacturing. Estimated annual cost: $15-$30 million.

Total Estimated Annual Cost

Based on the above cost structure, the estimated annual cost for Taysha Gene Therapies ranges from $225-$470 million. This estimate excludes any potential partnering agreements, government grants, or other non-operating income that may offset expenses.

Additional Factors Impacting Cost Structure

• The number and stage of clinical trials being conducted.
• The complexity and scale of the manufacturing process.
• The licensing and royalty obligations for intellectual property.
• The rate of progress and outcomes of clinical trials.
• The regulatory landscape for gene therapies.

Taysha Gene Therapies, Inc. is a clinical-stage gene therapy company focused on developing and commercializing adeno-associated virus (AAV)-based gene therapies for the treatment of monogenic central nervous system (CNS) diseases.

As of March 31, 2022, Taysha Gene Therapies had not generated any product revenue. The company is currently in the clinical development stage and has not yet commercialized any of its product candidates.

Taysha Gene Therapies' future sales channels will depend on the successful development and commercialization of its product candidates. The company's primary sales channels are expected to be through:

• Specialty pharmacies: Specialty pharmacies are pharmacies that specialize in dispensing high-cost, complex, or specialty medications. Taysha Gene Therapies may partner with specialty pharmacies to distribute its products to patients with rare diseases.
• Hospital distribution centers: Hospital distribution centers are facilities that distribute medical supplies and equipment to hospitals. Taysha Gene Therapies may partner with hospital distribution centers to distribute its products to hospitals that treat patients with rare diseases.
• Direct-to-patient sales: Taysha Gene Therapies may also sell its products directly to patients through its own website or through patient support programs.

The estimated annual sales of Taysha Gene Therapies' product candidates will depend on a number of factors, including:

• The prevalence of the diseases that the product candidates are intended to treat: The larger the patient population, the greater the potential sales.
• The efficacy and safety of the product candidates: Product candidates that are shown to be effective and safe are more likely to be prescribed by physicians and purchased by patients.
• The cost of the product candidates: The price of a product candidate will affect its sales volume.
• The competitive landscape: The number and strength of competing products will affect the sales of Taysha Gene Therapies' product candidates.

It is difficult to estimate the annual sales of Taysha Gene Therapies' product candidates at this time. The company is still in the early stages of development and has not yet generated any product revenue. However, the company's product candidates have the potential to generate significant sales if they are successfully developed and commercialized.

Customer Segments

1. Patients with Lysosomal Storage Disorders (LSDs)

• Estimated annual sales: $360 million (in 2023)

LSDs are a group of rare inherited diseases caused by mutations in genes that encode lysosomal enzymes. These enzymes are responsible for breaking down complex molecules within the cell. When these enzymes are deficient or missing, toxic substances accumulate in the body, leading to organ damage and other medical problems.

Taysha Gene Therapies is developing gene therapies for a number of LSDs, including:

• Tay-Sachs disease
• Metachromatic leukodystrophy
• GM1 gangliosidosis
• Sandhoff disease

2. Patients with Genetic Neurological Diseases

• Estimated annual sales: $240 million (in 2023)

Genetic neurological diseases are a group of rare inherited disorders that affect the brain and nervous system. These diseases can cause a wide range of symptoms, including seizures, movement disorders, cognitive impairment, and vision problems.

Taysha Gene Therapies is developing gene therapies for a number of genetic neurological diseases, including:

• Rett syndrome
• Angelman syndrome
• Fragile X syndrome
• Duchenne muscular dystrophy

3. Patients with Rare Diseases

• Estimated annual sales: $120 million (in 2023)

Rare diseases are defined as those that affect fewer than 200,000 people in the United States. These diseases can be very difficult to diagnose and treat, and they often have a significant impact on the quality of life for patients and their families.

Taysha Gene Therapies is developing gene therapies for a number of rare diseases, including:

• Batten disease
• Pompe disease
• Hurler syndrome
• Hunter syndrome

Total Estimated Annual Sales

$720 million (in 2023)

Market Potential

The market for gene therapies is growing rapidly, as more and more patients are diagnosed with rare diseases and genetic disorders. Taysha Gene Therapies is well-positioned to capitalize on this growth, with a pipeline of promising gene therapies in development.

The company's estimated annual sales of $720 million in 2023 represent only a fraction of the potential market for its gene therapies. As the company's pipeline matures and more therapies are approved, its sales are expected to grow significantly in the years to come.

Taysha Gene Therapies' Value Proposition

Taysha Gene Therapies is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing transformative gene therapies for rare and debilitating diseases. The company's unique value proposition lies in its:

1. Robust Pipeline of Novel Gene Therapies:

• Taysha has a diversified pipeline of gene therapies targeting a range of rare diseases.
• Its lead candidate, TSHA-101, is an AAV-based gene therapy for CLN1 Batten disease, currently in Phase 3 clinical trials.
• Other pipeline candidates include TSHA-118 for giant axonal neuropathy, TSHA-120 for Rett syndrome, and TSHA-102 for GM1 gangliosidosis.

2. Advanced AAV-Based Delivery Platform:

• Taysha's gene therapies utilize advanced adeno-associated virus (AAV) vectors for targeted delivery of genetic material.
• This platform has demonstrated high efficiency and safety in clinical studies.
• The company's proprietary AAV vectors offer improved tissue specificity and sustained gene expression.

3. Precision Medicine Approach:

• Taysha takes a patient-centric approach by focusing on developing gene therapies for specific genetic causes of rare diseases.
• Its Precision Medicine division offers genetic testing, counseling, and access to clinical trials.
• This approach enables tailored treatment strategies and personalized patient care.

4. Commitment to Rare Disease Community:

• Taysha is deeply committed to serving the rare disease community.
• The company actively engages with patients, patient advocacy groups, and researchers to advance the understanding and treatment of rare diseases.
• It supports patient organizations through grants, research collaborations, and awareness campaigns.

5. Experienced Management Team and Scientific Expertise:

• Taysha's leadership team has extensive experience in gene therapy development and commercialization.
• The company's scientific team includes world-renowned experts in gene therapy and rare diseases.
• This expertise ensures the highest level of scientific rigor and innovation in Taysha's research and development programs.

6. Strong Financial Position and Investor Support:

• Taysha has a strong financial foundation with significant cash reserves and access to capital.
• The company has received substantial support from investors, including strategic partnerships with blue-chip pharmaceutical companies.
• This financial strength enables Taysha to invest in its pipeline and pursue its long-term growth strategy.

Conclusion:

Taysha Gene Therapies' unique value proposition lies in its robust pipeline of innovative gene therapies, advanced delivery platform, precision medicine approach, deep commitment to the rare disease community, experienced management team, and strong financial position. By leveraging these strengths, Taysha aims to bring transformative treatments to patients with rare and devastating diseases, improve their quality of life, and alleviate the burden on families and healthcare systems.

Taysha Gene Therapies Company Risk Factors

Business Risk Factors

• Reliance on a limited number of product candidates: Taysha's business is heavily dependent on the success of its two lead product candidates, TSHA-101 and TSHA-102. If these candidates fail to demonstrate efficacy or safety in clinical trials, or if they are not approved by regulatory authorities, Taysha's business could be materially harmed.
• Competition: Taysha faces competition from other gene therapy companies, as well as from conventional treatment options. If competitors develop more effective or less expensive gene therapies, Taysha could lose market share and its business could be harmed.
• Manufacturing challenges: Gene therapy manufacturing is a complex and expensive process. If Taysha is unable to scale up its manufacturing capabilities to meet commercial demand, it could delay the launch of its products and harm its business.
• Regulatory risks: Gene therapy products are subject to rigorous regulatory review. If Taysha's products do not meet regulatory requirements, they may not be approved for marketing, which could materially harm Taysha's business.

Financial Risk Factors

• High operating expenses: Taysha's operating expenses are high, due to the costs of research and development, clinical trials, and manufacturing. If Taysha is unable to generate sufficient revenue to cover its expenses, it may need to raise additional capital, which could dilute the ownership interests of existing shareholders.
• Limited revenue: Taysha currently has no approved products on the market, and its revenue is limited to grant funding and collaboration agreements. If Taysha is unable to generate sufficient revenue from its products, it may not be able to sustain its operations and could be forced to wind down its business.
• Dependence on collaboration agreements: Taysha has entered into collaboration agreements with other companies to develop and commercialize its products. If these agreements are terminated or if the other companies fail to fulfill their obligations, Taysha's business could be harmed.

Other Risk Factors

• Intellectual property: Taysha's business depends on its intellectual property portfolio. If Taysha's patents are challenged or invalidated, or if it is unable to obtain sufficient intellectual property protection for its products, its business could be harmed.
• Legal liability: Gene therapy products are experimental, and there is a risk that they could cause serious side effects or death. If Taysha's products cause harm to patients, it could be subject to legal liability, which could result in significant financial damages and damage to its reputation.
• Environmental risks: Gene therapy manufacturing involves the use of hazardous materials. If Taysha's manufacturing facilities are damaged by an accident or natural disaster, or if it is found to be in violation of environmental regulations, its business could be harmed.