What Tern Therapeutics does

Tern Therapeutics, LLC is a privately-held clinical-stage biotechnology company founded in 2023 and headquartered in Washington, DC, developing one-time adeno-associated virus (AAV9) gene therapies for CLN2 Batten disease, a rare pediatric neurodegenerative disorder caused by TPP1 enzyme deficiency. The company's pipeline consists of two in-licensed assets acquired from REGENXBIO in August 2024: TTX-381, an intravitreal AAV9 gene therapy designed to deliver a working copy of the TPP1 gene directly to the retina for treatment of the ocular manifestations of CLN2 disease (progressive vision loss); and TTX-181, an AAV gene therapy designed to deliver TPP1 to the central nervous system via cerebrospinal fluid administration for treatment of the CNS manifestations (seizures, motor and cognitive decline). Tern has demonstrated clinical proof-of-concept data including 100% photoreceptor preservation in treated eyes of TTX-381 patients through 12 months, a 27- to 55-fold increase in CSF TPP1 levels and 90% seizure frequency reduction with TTX-181, and no serious adverse events related to either product.

The company is pre-revenue and operates a clinical development and eventual commercialization model. TTX-381 is currently in an open-label Phase I/II trial at Great Ormond Street Hospital in London and University Medical Center Hamburg Eppendorf in Germany, with regulatory engagement spanning the FDA (Fast Track, RMAT, Rare Pediatric Disease, Orphan Drug, CDRP selection) and the UK MHRA (Innovation Passport under ILAP). The go-to-market motion centers on clinical trial execution at specialized pediatric centers, supplemented by a global expanded access program for compassionate use, patient advocacy partnerships with BDFA, BDSRA, and BDSRA Australia to support enrollment, and scientific communication through major medical conferences including WORLDSymposium, the International Congress on NCL, and ARVO.

Tern was founded by three former REGENXBIO executives — CEO Alex M. Bailey, PhD, CMO Christina Ohnsman, MD, and CFO/CAO Matthew Rosini — all of whom were integral leaders in the original CLN2 programs at their prior employer. The company raised $15 million in seed financing in August 2024 led by ATW Partners and Steve Oliveira of Nemean Asset Management, and operates with 1-10 employees. Future revenue is expected to derive from one-time gene therapy product sales and/or licensing economics, contingent on regulatory approval in major markets.

Name

Tern Therapeutics

Legal name

Tern Therapeutics, LLC

Website

https://terntx.com

Company type

Private

Founded year

2023

Operating status

Operating

Headcount range

1–10 employees

Short description

Tern Therapeutics is a privately-held, clinical-stage biotechnology company developing one-time AAV9 gene therapies (TTX-381 and TTX-181) to treat ocular and CNS manifestations of CLN2 Batten disease in pediatric patients.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

akta.pro secondary industries

Ophthalmology Gene Therapies (HLAAACAG), Neurology/CNS Gene Therapies (HLAAACAH), AAV Vector Gene Therapy Developers (HLAAACAA), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• AAV gene therapy
• Rare disease therapeutics
• CLN2 Batten disease
• Ocular gene therapy
• CNS gene therapy

Headquarters

HQ city

Washington

HQ country

United States

HQ region

North America

Offices1 record

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 Development and Commercialization: Pre-revenue clinical stage biotechnology company developing one-time gene therapies for rare diseases. The company is focused on advancing TTX-381 and TTX-181 through clinical development toward regulatory approval and commercialization.

Go-to-market motion1 record

Distribution channels4 records

Marketing channels4 records

Core offering

Tern Therapeutics develops one-time AAV-based gene therapies targeting rare genetic diseases, with an initial pipeline focused on CLN2 (a form of Batten disease). Its lead candidate TTX-381 delivers a working copy of the TPP1 gene to the retina to address ocular manifestations (vision loss), and TTX-181 delivers TPP1 to the central nervous system to address neurological degeneration. Both programs are investigational, in clinical development, and were in-licensed from REGENXBIO.

Product overview

Tern Therapeutics is a privately-held biotechnology company developing transformative one-time AAV gene therapies for rare diseases. The company's initial therapeutic pipeline consists of two gene therapy products: TTX-381, a one-time gene therapy for the ocular manifestations of CLN2 Batten disease (targeting vision loss), and TTX-181, a one-time gene therapy for the CNS manifestations of CLN2 Batten disease (targeting neurological degeneration). Both products deliver a working copy of the TPP1 gene to address enzyme deficiency in patients with CLN2 disease.

Differentiator

Problem solved

Functional benefit

Products and services

• TTX-381 Investigational one-time AAV9 gene therapy that delivers a working copy of the TPP1 gene directly to the retina, intended as a durable source of TPP1 enzyme activity to treat ocular manifestations (vision loss) in patients with CLN2 Batten disease. Currently in Phase I/II clinical testing in the UK and Germany.
• TTX-181

Quantifiable outcome

• 100% of treated eyes (6/6) showed photoreceptor preservation with stabilization or improvement in ellipsoid zone loss at 12 months
• +4 more outcomes

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Seven partnerships are on record, tiered core and minor.

• REGENXBIO Inc.coreTechnology or Integration · 27 August 2024Tern Therapeutics entered into a global licensing agreement with REGENXBIO for RGX-381 and RGX-181 (now designated TTX-381 and TTX-181 respectively) to form its initial therapeutic pipeline. These are novel one-time gene therapy products for treatment of ocular and CNS manifestations of CLN2 disease.
• Batten Disease Family Association (BDFA)coreStrategic or Co-development PartnerBDFA is the only patient organization in the UK for families affected by Batten disease. They expressed support for Tern's advancement of CLN2 programs and the ocular trial in the UK.
• Batten Disease Support, Research & Advocacy (BDSRA)coreStrategic or Co-development PartnerBDSRA is a major US patient advocacy organization for Batten disease. They expressed strong support for the transition of gene therapy programs to Tern's team, noting the team's familiarity with programs and connection to CLN2 community.
• BDSRA AustraliaminorStrategic or Co-development PartnerAustralian affiliate of BDSRA providing support and collaboration for Tern's CLN2 programs in the Australian region.
• Great Ormond Street Hospital (GOSH)coreStrategic or Co-development PartnerWorld-renowned children's hospital in London, UK serving as the primary clinical trial site for TTX-381 Phase I/II study. Dr. Robert Henderson serves as Principal Investigator and Consultant Retinal Surgeon.
• University Medical Center Hamburg Eppendorf (UKE)coreStrategic or Co-development PartnerLeading European center for CLN2 research with the largest cohort of CLN2 patients worldwide. UKE received CTA approval from Germany's Paul-Ehrlich-Institut and opened as second clinical trial site for TTX-381 in October 2025. Dr. Angela Schulz serves as Principal Investigator.
• Hospital de Clinicas Porto AlegreminorStrategic or Co-development PartnerBrazilian hospital where physician investigators conducted a single-patient, investigator-initiated study of TTX-181 in late 2022. Dr. Carolina Fischinger Moura de Souza and Dr. Roberto Giugliani presented the two-year follow-up data.

Scale indicators3 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Voyager Therapeutics: Voyager Therapeutics is a clinical-stage gene therapy company developing AAV-based treatments for severe neurological diseases, including rare CNS indications. Comparable in AAV platform, neurological focus, and pipeline-stage maturity.
• Abeona Therapeutics: Abeona Therapeutics is a clinical-stage gene therapy company developing AAV-based treatments for rare diseases including CLN1 (infantile Batten disease) and CLN3 (juvenile Batten disease). Directly comparable as a Batten disease gene therapy peer with similar regulatory pathway and ultra-rare indication focus.
• Passage Bio: Passage Bio is a clinical-stage AAV gene therapy company focused on rare CNS disorders using AAV9 and AAV1 vectors. Comparable in technology platform (AAV), therapeutic area (rare CNS), and clinical-stage positioning.
• Taysha Gene Therapies: Taysha is a clinical-stage gene therapy company using AAV9 vectors to develop treatments for rare CNS diseases including CLN1 and CLN7 forms of Batten disease. Comparable in technology platform, vector choice (AAV9), and focus on ultra-rare CNS indications.
• REGENXBIO: REGENXBIO is the originator of the TTX-381 and TTX-181 programs that Tern licensed, and a leading AAV gene therapy developer with a broad pipeline including CNS and ocular assets. It is the most directly comparable peer given shared technology platform and overlapping therapeutic areas.
• Neurogene: Neurogene is a clinical-stage genetic medicines company developing AAV-based gene therapies for rare neurological diseases, including programs in Batten disease. Comparable in therapeutic focus (rare neurological/ocular disease), modality (AAV gene therapy), and stage (clinical).

Broad incumbents

• Spark Therapeutics (Roche): Spark Therapeutics developed and commercialized Luxturna, the first FDA-approved gene therapy for an inherited disease (RPE65-mediated retinal dystrophy), and was acquired by Roche. Comparable as a pioneer in ocular gene therapy with regulatory and commercial experience directly relevant to TTX-381.
• Ultragenyx Pharmaceutical: Ultragenyx is a commercial-stage rare disease company with gene therapy programs and a strong ultra-rare disease commercialization infrastructure. Comparable for the orphan disease regulatory pathway and ultra-rare pricing strategy that Tern's products would follow.
• BioMarin Pharmaceutical: BioMarin is an established rare disease biotech with a commercial gene therapy (Roctavian for hemophilia A) and multiple rare disease franchises. Comparable as a broader rare-disease-focused biotech with experience commercializing high-cost, ultra-rare therapies and managing payer relationships.

Emerging players

• Solid Biosciences: Solid Biosciences is a clinical-stage genetic medicines company developing AAV gene therapies for rare neuromuscular diseases. Comparable in AAV gene therapy modality, clinical stage, and rare disease therapeutic focus.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

Compliance6 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds3 records

Investors1 record

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 Tern Therapeutics