Background: Tackling ALS with Gene Therapy

Amyotrophic lateral sclerosis (ALS), the most common form of motor neuron disease, remains a devastating condition with no cure. Celosia Therapeutics, a Sydney-based biotech, is developing CTx1000, a novel gene therapy targeting TDP-43, a protein implicated in ALS pathology. The therapy is based on 15 years of research from Macquarie University’s Dementia Research Centre.

On November 25, 2024, Celosia announced the close of a $16.75 million Series A funding round, one of the largest ever for an Australian biotech startup.

Funding Breakdown and Strategic Backers

• Lead Investor: Uniseed, Australia’s longest-running research commercialization fund
• Key Contributor: Hedge fund manager and ALS patient Iomar Barrett
• Institutional Support: UniSuper, representing the tertiary education sector

The funding will accelerate clinical development of CTx1000 and support regulatory engagement and manufacturing scale-up.

Expert Commentary

Dr. Kathryn Sunn, CEO of Celosia, stated:

“This investment validates our approach and positions Australia as a global leader in ALS research. We’re not just slowing progression—we’re aiming to reverse it.”

Mr. Barrett added:

“CTx1000 represents a realistic pathway to a cure. As a fund manager, I see this as a transformative technology with global impact.”

Scientific Foundation and Clinical Outlook

• Mechanism: CTx1000 targets TDP-43 aggregation, a hallmark of ALS pathology
• Preclinical Data: Shows potential to halt and reverse motor neuron damage
• Next Steps: IND-enabling studies and Phase I trial initiation in 2025

 Implications for Australia’s Biotech Ecosystem

Celosia’s success highlights the growing maturity of Australia’s biotech sector, especially in neurodegenerative disease research. It also reflects a shift toward patient-driven investment and translational science.