Celosia Therapeutics

What Celosia Therapeutics does

Celosia Therapeutics is a private Australian clinical-stage biotechnology company developing AAV-based gene therapies targeting toxic TDP-43 protein for neurodegenerative diseases. The company is headquartered in Sydney, Australia, with 1-10 employees, and was founded based on 15+ years of neuroscience research conducted by Professors Lars Ittner and Yazi Diana Ke at Macquarie University. The core technology was exclusively licensed to Celosia in February 2024 following publication in the Neuron journal.

The company's pipeline is anchored by CTx1000, a first-in-class AAV gene therapy designed to selectively bind and clear pathological forms of TDP-43 — a protein implicated in the majority of ALS cases and a significant subset of FTD. Preclinical studies demonstrated that CTx1000 halted disease progression in multiple ALS models and partially reversed manifestations in some cases. The Phase 1b KOANEWA clinical trial dosed its first participant in March 2026 at Macquarie University Hospital. Three additional programs extend the platform: CTx2000 (ALS, preclinical), CTx3000 (tau-driven Alzheimer's disease, discovery), and CTx4000 (epilepsy syndromes, discovery). The company is led by CEO Dr. Kathryn Sunn alongside founders Professor Lars Ittner (CSMO) and Dr. Yazi Diana Ke (COO/Inventor).

Celosia operates a pre-revenue biotech business model funded by equity capital. The company has raised approximately $18.75 million across two funding rounds — a $2 million seed round in November 2022 and a $16.75 million round led by Uniseed in November 2024. Revenue generation is contingent on future therapeutic commercialization, out-licensing arrangements, or co-development partnerships with pharmaceutical companies. The company engages with investors and potential partners through direct outreach, its corporate website, LinkedIn, and YouTube channels. Its clinical trial site at Macquarie University Hospital represents the primary operational footprint, with no evidence of multi-site or international clinical expansion at this stage.

Name

Celosia Therapeutics

Legal name

Celosia Therapeutics

Website

https://celosiatx.com

Company type

Private

Founded year

2022

Operating status

Operating

Headcount range

1–10 employees

Short description

Celosia Therapeutics is a private Australian clinical-stage biotech developing AAV-based gene therapies that target toxic TDP-43 protein to treat neurodegenerative diseases including ALS, FTD, Alzheimer's, and epilepsy syndromes.

Ownership category

akta.pro rank

Product category

Biopharmaceuticals

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

Neurology/CNS Gene Therapies (HLAAACAH)

akta.pro secondary industries

AAV Vector Gene Therapy Developers (HLAAACAA), Neurology & CNS Specialty Pharmaceuticals (HLAIACAC), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

Keywords

• Gene therapy development
• Neurodegenerative disease treatment
• AAV-based therapeutics
• Biopharmaceutical research
• Precision medicines

Headquarters

HQ city

Sydney

HQ country

Australia

HQ region

Oceania

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Gene Therapy Development and Commercialization: Pre-revenue biotechnology company developing proprietary gene therapies for neurodegenerative diseases. Revenue will be generated through eventual therapeutic commercialization, licensing deals, and potential partnership arrangements.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Celosia Therapeutics is a clinical-stage Australian biotechnology company developing precision AAV-based gene therapies for neurodegenerative diseases. Its lead program, CTx1000, targets toxic TDP-43 protein implicated in ALS and FTD. The company also has pipeline programs targeting ALS (CTX2000), Alzheimer's disease (CTX3000), and epilepsy syndromes (CTX4000), all built on 15+ years of neuroscience research from Macquarie University.

Product overview

Celosia Therapeutics is a biotechnology company developing precision gene therapies for neurodegenerative diseases including ALS and FTD. The company's pipeline includes CTx1000 as the lead first-in-class gene therapy targeting toxic TDP-43 protein (currently in Phase 1b clinical trial), CTX2000 for ALS (preclinical), CTX3000 for Alzheimer's disease (discovery), and CTX4000 for epilepsy syndromes (discovery). All programs are built on 15+ years of neuroscience research from Macquarie University.

Differentiator

Problem solved

Functional benefit

Brands

• CTx1000: Lead gene therapy program targeting toxic TDP-43 in ALS and FTD.
• CTx2000
• CTx3000
• CTx4000

Products and services

• CTx1000 Lead AAV-based gene therapy designed to selectively bind and clear toxic forms of the TDP-43 protein, a central pathological driver of ALS and FTD; aims to slow or halt neurodegeneration in affected nerve cells.
• CTX2000 Gene therapy program targeting disease-specific molecular mechanisms in ALS to protect nerve cells and slow disease progression; preclinical stage.
• CTX3000 Gene therapy program focusing on tau-driven neurodegeneration in Alzheimer's disease; discovery stage.
• CTX4000 Gene therapy program designed to restore neuronal balance in severe epilepsy syndromes by targeting underlying molecular drivers of hyperexcitability; discovery stage.

Quantifiable outcome

• Disease progression halted in multiple preclinical ALS models; partial reversal of disease manifestations observed in some cases
• +1 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

One partnership is on record.

• Macquarie UniversitycoreStrategic or Co-development Partner · 1 February 2024Research partnership with Macquarie University based on 15+ years of neuroscience research by Professors Lars Ittner and Yazi Diana Ke. The TDP-43 targeting technology was developed at Macquarie University and exclusively licensed to Celosia. The clinical trial is being conducted at Macquarie University Hospital.

Scale indicators3 records

Recent moves5 records

Expansion highlights5 records

Direct peers

• Passage Bio: Clinical-stage AAV-based gene therapy company focused on CNS/neurodegenerative diseases. Direct peer to Celosia on modality (AAV gene therapy) and indication area (CNS), making it the most comparable private-stage peer for translational and manufacturing strategy benchmarking.
• Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based treatments for CNS disorders including neurodegenerative diseases. Shares the AAV-CNS gene therapy modality and indication overlap with Celosia, making it a key comparable for pipeline strategy and valuation.
• Lexeo Therapeutics: Clinical-stage gene therapy company focused on CNS and cardiac diseases, including Alzheimer's and other neurodegenerative programs. Direct peer on modality (AAV gene therapy) and overlap on neurodegenerative CNS indications.
• uniQure: Clinical-stage AAV gene therapy company with CNS programs (including Huntington's disease). Comparable on AAV-CNS modality and clinical-stage gene therapy operations relevant to Celosia's pipeline strategy.
• REGENXBIO: Commercial-stage AAV gene therapy platform company with CNS programs (e.g., MPS II) and a broad AAV technology licensing platform. Comparable to Celosia on AAV vector modality and as a benchmark for AAV platform valuation.

Broad incumbents

• Biogen: Large biotech with leading ALS commercial franchise (Spinraza, Qalsody). Most established player in ALS therapeutics and a likely strategic acquirer/partner for late-stage ALS gene therapy assets like Celosia's CTx1000.
• Ionis Pharmaceuticals: Established antisense oligonucleotide developer with tofersen (Qalsody) for SOD1-ALS, directly relevant to the ALS competitive landscape. A potential partner or acquirer interested in expanding its ALS franchise beyond SOD1 into TDP-43-driven disease.
• Cytokinetics: Clinical-stage biopharmaceutical company focused on muscle function and ALS (e.g., reldezemtide partnership with Biogen). Direct comparator on ALS pipeline strategy and as a potential partner for combination or disease-modifying ALS regimens.

Emerging players

• Krystal Biotech: Commercial-stage AAV gene therapy company with a CNS-pivoting pipeline strategy (e.g., KB-407 for cystic fibrosis, exploration of CNS targets). Comparable on AAV manufacturing platform and gene therapy operational scale-up.
• AavantiBio: AAV gene therapy developer with CNS indications in its pipeline. Comparable emerging AAV gene therapy peer for benchmarking on platform development and clinical translation in CNS indications.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• YouTubeyoutube.com/@CelosiaTherapeutics
• LinkedInlinkedin.com/company/celosia-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds2 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 Celosia Therapeutics