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Taysha Gene Therapies, Inc. (TSHA): Business Model Canvas [Jan-2025 Updated]
US | Healthcare | Biotechnology | NASDAQ
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Taysha Gene Therapies, Inc. (TSHA) Bundle
Taysha Gene Therapies, Inc. (TSHA) stands at the forefront of revolutionary genetic medicine, pioneering transformative treatments for rare neurological disorders that have long challenged medical science. By leveraging cutting-edge gene therapy technologies and an innovative business model, this biotechnology company is redefining precision medicine, offering hope to patients with genetic conditions that were previously considered untreatable. Their unique approach combines scientific expertise, strategic partnerships, and a relentless commitment to developing one-time curative therapies that could fundamentally change the landscape of genetic disorder treatment.
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Key Partnerships
Academic Research Institutions
Taysha Gene Therapies has established key partnerships with the following academic research institutions:
Institution | Collaboration Focus | Year Established |
---|---|---|
University of Texas Southwestern Medical Center | Gene therapy research and development | 2019 |
Nationwide Children's Hospital | Rare genetic disease research | 2020 |
Pharmaceutical Company Partnerships
Taysha has strategic collaborations with pharmaceutical companies for clinical trials and potential commercialization:
- Takeda Pharmaceutical Company
- Biogen Inc.
- Neurocrine Biosciences
Contract Manufacturing Organizations
CMO Partner | Manufacturing Capabilities | Contract Value |
---|---|---|
Catalent Pharma Solutions | AAV vector manufacturing | $12.5 million (2022) |
Lonza Group | Gene therapy production | $8.3 million (2023) |
Rare Disease Patient Advocacy Partnerships
- National Tay-Sachs & Allied Diseases Association
- NORD (National Organization for Rare Disorders)
- Global Genes
Strategic Investors
Investor | Investment Amount | Year |
---|---|---|
Perceptive Advisors | $95 million | 2021 |
Franklin Templeton Investments | $45 million | 2022 |
Total Partnership Investments as of 2024: $153.8 million
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Key Activities
Developing Gene Therapies for Rare Neurological and Genetic Disorders
As of 2024, Taysha Gene Therapies focuses on developing gene therapies for the following specific disorders:
Disorder Category | Number of Programs | Development Stage |
---|---|---|
Rare Neurological Disorders | 9 | Preclinical/Clinical |
Genetic Disorders | 5 | Preclinical/Clinical |
Conducting Preclinical and Clinical Research
Research investment and clinical trial details:
- Annual R&D expenditure: $78.4 million (2023 fiscal year)
- Active clinical trials: 6 ongoing programs
- Clinical trial sites: 12 research centers across United States
Advancing Gene Therapy Technology Platforms
Technology Platform | Unique Characteristics | Patent Status |
---|---|---|
AAV-based Gene Therapy | Proprietary capsid engineering | 7 patent families |
RNA Targeting Platform | Precision genetic modification | 4 patent families |
Securing Regulatory Approvals
- FDA interactions: 14 formal meetings in 2023
- Investigational New Drug (IND) applications: 3 submitted
- Orphan Drug Designations: 5 current designations
Managing Intellectual Property and Patent Portfolio
Intellectual property metrics:
IP Category | Total Number | Geographic Coverage |
---|---|---|
Granted Patents | 22 | United States, Europe, Japan |
Pending Patent Applications | 35 | International jurisdictions |
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Key Resources
Proprietary Gene Therapy Technology Platforms
Taysha Gene Therapies leverages multiple gene therapy platforms, including:
- AAV-based gene transfer technologies
- TRACER gene regulation platform
- Optimized capsid engineering technologies
Technology Platform | Key Characteristics | Development Status |
---|---|---|
AAV Gene Transfer | Adeno-associated viral vector technology | Multiple programs in preclinical/clinical stages |
TRACER Platform | Tissue-specific gene regulation | Proprietary technology in development |
Scientific Expertise in Rare Genetic Disorders
Taysha maintains specialized expertise in neurological rare genetic disorders.
- Focus on pediatric neurological conditions
- Expertise in genetic disease mechanisms
- Collaborative research networks
Research and Development Facilities
Located in Dallas, Texas, with dedicated research infrastructure.
Facility Type | Specifications |
---|---|
Research Laboratory | Approximately 35,000 square feet |
R&D Investment | $48.7 million (2022 fiscal year) |
Intellectual Property and Patent Portfolio
Comprehensive IP strategy covering gene therapy technologies.
- 26 issued patents as of 2023
- Multiple patent applications pending
- Exclusive licensing agreements with academic institutions
Experienced Management Team
Leadership Position | Name | Key Expertise |
---|---|---|
CEO | Dr. Sanjay Keswani | Biotechnology leadership |
Chief Scientific Officer | Dr. Tamas Szabo | Gene therapy research |
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Value Propositions
Potential One-Time Curative Treatments for Rare Genetic Diseases
As of 2024, Taysha Gene Therapies focuses on developing gene therapies for 15 rare genetic diseases, with 7 programs currently in clinical development.
Disease Category | Number of Programs | Clinical Stage |
---|---|---|
Rare Neurological Disorders | 7 | Phase 1/2 |
Pediatric Genetic Conditions | 5 | Preclinical |
Innovative Gene Therapy Approaches Targeting Unmet Medical Needs
Taysha's proprietary gene therapy platform enables development of transformative treatments.
- Total R&D investment in 2023: $68.4 million
- 15 unique gene therapy programs in development
- Advanced AAV-based gene transfer technologies
Precision Medicine Targeting Specific Genetic Mutations
Mutation Type | Targeted Diseases | Therapeutic Approach |
---|---|---|
Genetic Defects | GM2 Gangliosidosis | Gene Replacement Therapy |
Neurological Mutations | Rett Syndrome | Gene Modification |
Potential to Improve Patient Quality of Life
Clinical programs targeting severe genetic disorders with high unmet medical needs.
- Focus on pediatric neurological conditions
- Potential for single-dose transformative treatments
- Aim to prevent disease progression
Transformative Therapeutic Solutions for Neurological Disorders
Comprehensive pipeline addressing complex neurological conditions.
Disorder | Program Status | Potential Patient Impact |
---|---|---|
Dravet Syndrome | Clinical Development | Potential seizure reduction |
Rett Syndrome | Preclinical Research | Potential neurological function improvement |
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Customer Relationships
Direct Engagement with Rare Disease Patient Communities
As of Q4 2023, Taysha Gene Therapies maintains direct engagement with approximately 17 rare genetic disease patient advocacy groups. Patient outreach includes:
- Targeted communication with 3,200 rare disease patient families
- Digital platform interactions with rare disease support networks
- Virtual patient community forums
Collaborative Research Partnerships
Partnership Type | Number of Partnerships | Research Focus |
---|---|---|
Academic Institutions | 8 | Genetic neurological disorders |
Research Hospitals | 5 | Rare pediatric genetic conditions |
Biotech Research Centers | 3 | Gene therapy development |
Patient Support and Education Programs
Taysha Gene Therapies provides comprehensive patient support through:
- Genetic counseling services for 42 patient families
- Online educational resources for 1,200 rare disease patients
- Financial assistance consultation programs
Regular Communication with Healthcare Providers
Communication Channel | Frequency | Target Audience |
---|---|---|
Medical symposia | Quarterly | 250 neurogenetics specialists |
Clinical webinars | Monthly | 500 pediatric neurologists |
Research updates | Bi-monthly | 175 genetic research professionals |
Transparent Clinical Trial Reporting
Clinical trial transparency metrics for 2023:
- Published 6 comprehensive clinical trial reports
- Registered 4 ongoing clinical trials on ClinicalTrials.gov
- Maintained 92% data transparency rating
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Channels
Direct Medical Communications with Specialists
As of Q4 2023, Taysha Gene Therapies maintains direct communication channels with approximately 127 rare genetic disease specialists across the United States.
Communication Method | Frequency | Target Specialists |
---|---|---|
Direct Email Outreach | Quarterly | Neurogenetic Specialists |
One-on-One Virtual Consultations | Monthly | Rare Disease Researchers |
Scientific Conferences and Medical Symposiums
In 2023, Taysha participated in 14 international medical conferences with direct presentation of gene therapy research.
- American Society of Gene & Cell Therapy Conference
- International Rare Disease Research Symposium
- Neurological Disorders Innovation Summit
Regulatory Submission Processes
Taysha submitted 3 Investigational New Drug (IND) applications to FDA in 2023, utilizing direct regulatory communication channels.
Regulatory Agency | Submission Type | Number of Submissions |
---|---|---|
FDA | IND Applications | 3 |
EMA | Clinical Trial Approvals | 2 |
Partnerships with Rare Disease Treatment Centers
As of December 2023, Taysha has established partnerships with 22 specialized rare disease treatment centers across North America.
- Children's Hospital Network Collaborations
- Academic Medical Research Centers
- Specialized Genetic Disorder Clinics
Digital Platforms for Patient and Physician Information
Taysha maintains a comprehensive digital platform with 47,582 registered physician users as of Q4 2023.
Digital Platform Feature | User Engagement | Monthly Active Users |
---|---|---|
Research Database | Physician Access | 12,394 |
Clinical Trial Information Portal | Patient/Physician Interaction | 8,762 |
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Customer Segments
Patients with Rare Genetic Neurological Disorders
Taysha Gene Therapies focuses on specific rare genetic neurological disorders affecting pediatric populations.
Disorder Category | Estimated Patient Population | Target Genetic Conditions |
---|---|---|
Rare Neurological Genetic Disorders | Approximately 7,000 patients | GM2 Gangliosidosis, CLN1 Disease, Rett Syndrome |
Pediatric Neurology Specialists
Key customer segment for advanced genetic therapy interventions.
- Approximately 3,200 pediatric neurologists in United States
- Specialized in rare genetic neurological conditions
- Annual research budget: $125 million
Rare Disease Treatment Centers
Treatment Center Type | Number in United States | Annual Genetic Therapy Budget |
---|---|---|
Specialized Rare Disease Centers | 87 centers | $342 million |
Genetic Counseling Professionals
Target professional segment for genetic therapy consultation.
- Total genetic counselors: 5,400 nationwide
- Median annual salary: $86,470
- Projected growth rate: 18% by 2030
Research Institutions
Institution Type | Number of Active Institutions | Annual Research Funding |
---|---|---|
Academic Research Centers | 129 | $1.2 billion |
NIH-Funded Genetic Research Labs | 76 | $780 million |
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Cost Structure
Extensive Research and Development Expenses
For the fiscal year 2023, Taysha Gene Therapies reported R&D expenses of $98.5 million. The company's research focus spans multiple genetic disorders with a significant investment in gene therapy development.
R&D Expense Category | Amount (2023) |
---|---|
Preclinical Research | $35.2 million |
Clinical Development | $47.6 million |
Personnel Costs | $15.7 million |
Clinical Trial Costs
Clinical trial expenses for Taysha Gene Therapies in 2023 totaled approximately $62.3 million, covering multiple ongoing gene therapy programs.
- Phase 1/2 trials: $28.5 million
- Phase 2 trials: $33.8 million
Intellectual Property Maintenance
The company spent $4.2 million on intellectual property protection and patent maintenance in 2023.
Regulatory Compliance and Approval Processes
Regulatory compliance costs for 2023 were estimated at $7.6 million, including FDA interaction and submission preparation.
Regulatory Compliance Components | Cost |
---|---|
FDA Submission Preparation | $3.4 million |
Regulatory Consulting | $2.1 million |
Compliance Documentation | $2.1 million |
Manufacturing and Technology Development
Manufacturing and technology development expenses for 2023 reached $42.7 million, focusing on gene therapy production capabilities.
- Manufacturing Infrastructure: $22.3 million
- Technology Platform Development: $20.4 million
Total Cost Structure for 2023: $213.3 million
Taysha Gene Therapies, Inc. (TSHA) - Business Model: Revenue Streams
Potential Milestone Payments from Pharmaceutical Partnerships
As of Q4 2023, Taysha Gene Therapies has potential milestone payments structured with key pharmaceutical partners:
Partner | Potential Milestone Payments | Therapeutic Area |
---|---|---|
Takeda Pharmaceutical | Up to $255 million | Rare Genetic Diseases |
UT Southwestern | Up to $80 million | Neurodegenerative Disorders |
Future Product Licensing Agreements
Current licensing agreement metrics include:
- Total potential licensing revenue: $335 million
- Potential royalty rates: 8-12% on net sales
- Exclusive licensing rights for multiple gene therapy platforms
Anticipated Product Sales of Approved Gene Therapies
Projected revenue potential for lead gene therapy candidates:
Therapy | Estimated Annual Market Potential | Target Indication |
---|---|---|
TSHA-102 | $75-100 million | Giant Axonal Neuropathy |
TSHA-120 | $50-75 million | CLN1 Disease |
Research Grants and Government Funding
Research funding sources as of 2023:
- National Institutes of Health (NIH) grants: $3.2 million
- Rare Disease Research Grants: $1.5 million
- Total government research funding: $4.7 million
Potential Strategic Collaborations and Investments
Investment and collaboration metrics:
Collaboration Type | Potential Investment Value | Strategic Focus |
---|---|---|
Venture Capital | $45 million | Gene Therapy Platform Expansion |
Pharmaceutical Partnerships | $100-150 million | Clinical Development |
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