Taysha Gene Therapies

What Taysha Gene Therapies does

Taysha Gene Therapies is a Dallas, Texas-based clinical-stage biotechnology company (NASDAQ: TSHA) founded in 2020 that develops AAV-based gene therapies for severe monogenic diseases of the central nervous system. Its lead program is TSHA-102, a self-complementary, intrathecally delivered AAV9 gene therapy using the proprietary miRARE (microRNA-Responsive Auto-Regulatory Element) platform plus a miniaturized MECP2 transgene to treat Rett syndrome, with the REVEAL pivotal trial (15 female patients aged 6 to <22) fully dosed and the ASPIRE trial enrolling pediatric patients aged 2 to <4 years.

The lead program has accumulated a dense regulatory moat: FDA Breakthrough Therapy, RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease designations, plus EMA Orphan Drug and UK MHRA ILAP. Manufacturing uses a scalable HEK293 triple-transfection suspension process, and intrathecal administration enables outpatient delivery at lower doses than systemic routes. Clinical data show 100% of evaluable patients achieving developmental milestone gains at 12 months with no treatment-related serious adverse events.

The company currently generates minimal recurring revenue (FY2025: $9.77M; Q1 2026: $7.5M) primarily from residual collaboration/licensing income from the now-expired Astellas agreement; Taysha has no approved products. The business model is structured around a one-time intrathecal gene therapy treatment for ultra-rare Rett syndrome (estimated 15,000–20,000 patients in the US/EU/UK), with pricing expected to follow rare-disease value-based frameworks. Capital is sourced through repeated public equity offerings (~$475M raised across June 2024, May 2025, and June 2026) and a prior $150M private placement in August 2023, with cash of $276.6M as of Q1 2026 guiding runway into 2028 — sufficient to reach a projected H1 2027 BLA filing and potential Q4 2027 US launch.

Name

Taysha Gene Therapies

Legal name

Taysha Gene Therapies, Inc.

Website

https://tayshagtx.com

Company type

Public

Founded year

2020

Operating status

Operating

Headcount range

101–250 employees

Short description

Taysha Gene Therapies is a clinical-stage biotechnology company (NASDAQ: TSHA), founded in 2020 and based in Dallas, Texas, that develops AAV-based gene therapies for severe monogenic CNS diseases, with lead program TSHA-102 (AAV9/miRARE) in pivotal development for Rett syndrome.

Ownership category

akta.pro rank

Product category

Gene Therapy for Rare CNS Disorders

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Neurology/CNS Gene Therapies (HLAAACAH)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), AAV Vector Gene Therapy Developers (HLAAACAA), Gene Therapy Delivery Devices & Administration Systems (catheters, intrathecal, ocular delivery) (HLAAACAL)

Keywords

• AAV gene therapy
• Monogenic CNS diseases
• Intrathecal gene delivery
• Rare pediatric therapeutics
• Clinical-stage biotechnology

Headquarters

HQ city

Dallas

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Others

Revenue model

1. Gene Therapy Product Sales (Anticipated): Taysha is a pre-commercial clinical-stage biotech company developing TSHA-102 for Rett syndrome. It has no approved products yet and generates minimal revenue (collaboration/licensing income). The company is building commercial capabilities in anticipation of a potential BLA approval and launch of TSHA-102 as a one-time gene therapy treatment.
2. Collaboration and Licensing Revenue: The company generates occasional collaboration revenue from licensing agreements, such as the former Astellas partnership. Q1 2025 revenue was $5.49M and FY2025 revenue was $9.77M, primarily from collaboration activities. Future revenue will depend on clinical progress and regulatory approvals.

Go-to-market motion2 records

Distribution channels1 record

Marketing channels5 records

Core offering

Taysha Gene Therapies is a clinical-stage biotechnology company developing AAV-based gene therapies for severe and life-threatening monogenic diseases of the central nervous system, with its lead candidate TSHA-102 (a self-complementary intrathecally delivered AAV9 gene therapy incorporating miRARE auto-regulatory technology) in pivotal clinical development for Rett syndrome. The platform combines miniaturized MECP2 transgene delivery, cell-by-cell expression regulation to avoid toxic overexpression, and a scalable HEK293 manufacturing process, targeting a one-time disease-modifying treatment for patients aged 2 years and older.

Product overview

Taysha Gene Therapies is a clinical-stage biotechnology company developing AAV-based gene therapies for monogenic diseases of the central nervous system. The company's core product is TSHA-102, a self-complementary intrathecally delivered AAV9 gene therapy for Rett syndrome that uses the proprietary miRARE technology to enable cell-by-cell regulation of MECP2 expression. The clinical program includes the REVEAL Pivotal Trial (Part B), REVEAL Phase 1/2 Trials (Part A) for adolescent/adult and pediatric populations, and the ASPIRE Trial for patients aged 2 to <4 years. The technology platform is built on an AAV9 capsid delivery system with intrathecal administration route. The company is laser-focused on advancing its lead clinical program in Rett syndrome, with a pipeline of early-stage gene therapy programs targeting other CNS diseases.

Differentiator

Problem solved

Functional benefit

Products and services

• TSHA-102 A self-complementary intrathecally delivered AAV9 gene therapy in clinical evaluation for Rett syndrome, designed as a one-time treatment to address the genetic root cause by delivering a functional form of MECP2 to cells in the central nervous system. Incorporates miRARE (microRNA-Responsive Auto-Regulatory Element) technology to prevent MeCP2 overexpression and is being developed for females aged 2 years and older.

Quantifiable outcome

• 100% response rate at 12 months in REVEAL Phase 1/2 Part A — all 10 patients achieved at least one developmental milestone gain
• +4 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Scale indicators10 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Sarepta Therapeutics: Commercial-stage gene therapy company with Elevidys (AAV-based gene therapy for Duchenne muscular dystrophy) and a pipeline of AAV programs for rare neuromuscular diseases. Directly comparable as a clinical-to-commercial AAV gene therapy company targeting rare pediatric CNS/neuromuscular indications with similar regulatory and reimbursement dynamics.
• Regenxbio: Clinical and commercial-stage AAV gene therapy company developing RGX-202 for Duchenne, ABBV-RGX-314 for wet AMD (with AbbVie), and CNS gene therapy programs. Comparable as an AAV vector platform company advancing CNS and rare disease gene therapies through pivotal trials.
• BioMarin Pharmaceutical: Commercial-stage rare disease biotech with Roctavian (AAV5 gene therapy for hemophilia A) and a deep pipeline of AAV gene therapies for CNS and metabolic indications. Directly comparable for AAV gene therapy development, manufacturing, and commercialization of ultra-rare disease therapies.
• uniQure: Clinical-stage gene therapy company developing AAV-based therapies for hemophilia B (etranacogene dezaparvovec, approved as Hemgenix via CSL partnership) and CNS indications including Huntington's disease. Closely comparable as an AAV gene therapy developer focused on rare diseases with CNS expansion ambitions.
• Solid Biosciences: Clinical-stage AAV gene therapy company developing SGT-501 (a microdystrophin gene therapy for Duchenne) and CNS programs. Comparable as a smaller clinical-stage AAV gene therapy peer with similar single-asset/single-indication concentration and capital intensity.
• MeiraGTx Holdings: Clinical-stage gene therapy company with CNS and ocular programs (Parkinson's, ALS, inherited retinal diseases). Comparable as an AAV gene therapy developer pursuing CNS rare disease indications with multiple parallel programs.
• Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based therapies for CNS diseases including Parkinson's, Huntington's, and Alzheimer's. Comparable as an AAV-CNS gene therapy developer with a capsid-engineering platform approach similar in spirit to Taysha's miRARE technology.
• Passage Bio: Clinical-stage AAV gene therapy company focused on CNS monogenic diseases including GM1 gangliosidosis and frontotemporal dementia. Comparable as a clinical-stage AAV gene therapy peer targeting rare pediatric CNS disorders with intrathecal delivery approaches.

Broad incumbents

• Pfizer: Large biopharmaceutical company with commercial gene therapy Beqvez (fidanacogene elaparvovec for hemophilia B, acquired via Spark Therapeutics) and a broader rare disease pipeline. Comparable as a large pharma incumbent operating in the AAV gene therapy space, particularly relevant to Taysha's commercialization competitive dynamics.
• Novartis: Large biopharmaceutical company commercializing Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy - the first FDA-approved gene therapy for a monogenic CNS disease. Relevant as the strategic acquirer of AveXis and the benchmark for CNS gene therapy commercialization that Taysha's leadership helped build.

Market position

Strengths6 records

Weaknesses4 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xtwitter.com/tayshagtx
• LinkedInlinkedin.com/company/taysha-gene-therapies

Compliance7 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds10 records

Investors23 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Taysha Gene Therapies