What GeneTx Biotherapeutics does

GeneTx Biotherapeutics is a biotechnology company founded in 2017 to develop therapeutics for Angelman syndrome, a rare neurogenetic disorder with no approved disease-modifying treatments. The company's core technology centers on gene therapy modalities, including antisense oligonucleotide (ASO) approaches, with GTX-102 (now branded UX143 under Ultragenyx) as its lead clinical-stage candidate targeting Angelman syndrome. GeneTx was acquired by Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) in August 2024 and operates as a wholly-owned subsidiary, leveraging Ultragenyx's integrated rare disease drug development platform spanning discovery, gene therapy manufacturing, and global commercialization infrastructure across 18+ countries.

GeneTx's business model is pre-revenue and clinically-oriented: the company historically financed operations through venture capital, including a $75 million Series A round in 2020 co-led by RA Capital Management and OrbiMed Advisors with participation from Fidelity Management and Research Company, alongside an earlier ~$6.3 million raise in 2018. Its customer segment is narrow by design — Angelman syndrome patients and their caregivers — with planned commercialization expected to flow through Ultragenyx's specialty pharmacy and hospital-based administration channels under the ultra-orphan gene therapy commercial model. Pricing for gene therapies of this class typically ranges $2-3 million per patient as one-time treatments, though GTX-102-specific pricing is not publicly disclosed.

The company's value proposition is rooted in addressing severe unmet medical need in ultra-rare genetic disease, where standard pharmaceutical economics break down but pricing power and regulatory exclusivity are correspondingly high. As a subsidiary of Ultragenyx, GeneTx benefits from its parent's diversified gene therapy pipeline (DTX401, DTX301, UX701, UX111, UX016, UX055), established rare disease advocacy infrastructure (ultrarareadvocacy.com), and operational scale in clinical development and manufacturing. The leadership team — co-founders Allyson Berent (COO) and Paula Evans (CEO), alongside CMO Scott Stromatt — brings rare disease development expertise that anchored the company's clinical progression prior to acquisition.

Name

GeneTx Biotherapeutics

Legal name

GeneTx Biotherapeutics, Inc.

Website

https://genetxbio.com

Company type

Private

Founded year

2017

Operating status

Acquired

Headcount range

1,001–5,000 employees

Short description

GeneTx Biotherapeutics, a 2017-founded biotech and wholly-owned subsidiary of Ultragenyx (NASDAQ: RARE) since August 2024, develops gene therapies for Angelman syndrome, a rare neurogenetic disorder with no approved disease-modifying treatments, with lead candidate GTX-102 (now UX143) in clinical development.

Ownership category

akta.pro rank

Product category

Gene Therapy / Rare Disease Biopharmaceuticals

NAICS

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

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

akta.pro secondary industries

Neurology/CNS Gene Therapies (HLAAACAH), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH)

Keywords

• Rare disease therapeutics
• Gene therapy development
• Antisense oligonucleotide therapy
• Angelman syndrome treatment
• Neurological genetic diseases

Headquarters

HQ city

Downers Grove

HQ country

United States

HQ region

North America

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Supply Chain

Marketing channels1 record

Core offering

GeneTx Biotherapeutics develops GTX-102, an antisense oligonucleotide gene therapy candidate in clinical development for Angelman syndrome, a rare neurogenetic disorder with no currently approved disease-modifying therapies. The company operates as a wholly-owned subsidiary of Ultragenyx Pharmaceutical Inc., focused exclusively on advancing this single therapeutic program targeting the UBE3A gene expression pathway for patients with Angelman syndrome.

Differentiator

Problem solved

Functional benefit

Products and services

• GTX-102 (now UX143)

Quantifiable outcome

• The global gene therapy market was valued at $7.74 billion in 2023 and is projected to exceed $38.76 billion by 2032.

Segments1 record

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

One partnership is on record.

• UltragenyxcoreStrategic or Co-development PartnerGeneTx Biotherapeutics is a subsidiary of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE). Ultragenyx acquired GeneTx in 2020 to expand its rare disease gene therapy pipeline, specifically to advance GTX-102 for Angelman syndrome. GeneTx operates as part of Ultragenyx's integrated platform, leveraging the parent company's development, manufacturing, and commercial capabilities.

Recent moves5 records

Expansion highlights5 records

Broad incumbents

• Ultragenyx Pharmaceutical: Parent company of GeneTx; publicly-traded rare-disease biopharma with an integrated gene-therapy platform, multiple clinical-stage programs across metabolic and neurologic rare diseases, and global commercial infrastructure that GeneTx now leverages for GTX-102 development.
• Sarepta Therapeutics: Established commercial-stage gene therapy company with FDA-approved AAV-based therapies for rare neuromuscular diseases; comparable as a rare-disease genetic medicine developer with demonstrated regulatory and commercial track record.
• PTC Therapeutics: Established rare-disease biopharma with both small-molecule and gene-therapy programs across CNS and metabolic indications; relevant comparable for a clinical-stage rare-disease asset at the integration/launch stage.
• BioMarin Pharmaceutical: Commercial-stage rare-disease biopharma with approved gene and enzyme-replacement therapies (e.g., ROCTAVIAN for hemophilia A); comparable in commercialization of ultra-orphan gene therapies and pricing/reimbursement strategy.

Direct peers

• Ionis Pharmaceuticals: Leader in antisense oligonucleotide (ASO) therapeutics with a deep pipeline in rare neurological and genetic diseases; directly relevant given that GTX-102 is described in funding details as an ASO therapy for Angelman syndrome.
• Taysha Gene Therapies: Clinical-stage gene therapy company focused on rare CNS and neurodevelopmental disorders using AAV delivery; most directly comparable to GeneTx on indication area (rare CNS monogenic disease) and modality.

Emerging players

• Passage Bio: Clinical-stage AAV gene therapy developer targeting rare CNS disorders; comparable therapeutic modality and indication focus to GeneTx/GTX-102 for Angelman syndrome.
• Encoded Therapeutics: Gene therapy company developing AAV-delivered regulatory-element-based therapies for severe CNS disorders; overlaps with GeneTx's modality and ultra-rare CNS indication strategy.
• Ovid Therapeutics: Clinical-stage rare-disease biotech focused on neurological and neurodevelopmental disorders; comparable patient population focus and orphan-drug development approach.
• Wave Life Sciences: Clinical-stage RNA therapeutics company developing stereopure oligonucleotides for rare neurological and neuromuscular genetic diseases; comparable to GTX-102 if treated as an oligonucleotide-based modality.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights5 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds1 record

Investors

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 GeneTx Biotherapeutics