ALS gene therapy biotech startup Celosia Therapeutics raises $16.75m

Sydney-headquartered Celosia Therapeutics has completed one of the largest Series A raises for an Australian biotech startup, securing $16.75 million to further its program for treating amyotrophic lateral sclerosis (ALS) disease.

The financing round for the Macquarie University spin-out was led by Uniseed, Australia’s longest-running research commercialisation fund, supported by UniSuper and with the majority of the investment coming from hedge fund manager and ALS patient Iomar Barrett.

The funds will be used to advance the development of Celosia Therapeutics’ lead candidate, CTx1000, a novel gene therapy that targets TDP-43 - a protein directly linked to ALS pathology.

ALS is the most common form of Motor Neuron Disease (MND) and causes the progressive loss of motor neurons in the brain and spinal cord, resulting in the loss of muscle control and eventual death.

Currently there is no cure to reverse damage to motor neurons or to cure the disease. But Celosia's CTx1000 therapy has shown the potential to not only stop the progression of the disease but in fact reverse the damage.

This form of therapy is based on a discovery made by Professor Yazi Ke and Professor Lars Ittner from Macquarie University’s Dementia Research Centre, which attracted a seed investment from the university in 2022.

"CTx1000 is a novel disease-modifying therapy for ALS, differentiating itself from other treatments that only target symptoms of the disease," says Ittner.

"We firmly believe in the potential of this therapy, and are excited to continue exploring its efficacy at clinical stages."

Celosia director Professor Dan Johnson says the completion of this funding round represents a "crucial step" in accelerating the development of therapies that could bring meaningful change to those living with ALS.

"Macquarie University created and seed-funded Celosia Therapeutics to facilitate the translation of pioneering research into tangible outcomes for the benefit of patients and to support a local entrepreneurial research ecosystem," says Johnson, who is also Pro Vice-Chancellor, Research, Innovation and Enterprise at the university.

The discovery made by Ittner and Ke, which is exclusively licensed to Celosia to develop CTx1000 as genetic medicine, was published in the prestigious journal Neuron in February this year, identifying a novel protein interactor that interacts exclusively with only toxic forms of TDP-43, a protein that is normally found in neurons in the brain and spinal cord, and in diseases such as ALS.

Celosia claims this exclusive link leads to a viable solution to clean up the neurons and reverse the damage. If it can be removed from neurons it could mean that normal functioning form of TDP-43 is restored and neuron health is preserved. Preclinical data observed that in mice models, CTx1000 is able to pause the progression of ALS at a very advanced stage, even partially reversing the disease in some cases.

“This landmark investment is a huge boost for the biotech sector, and not only validates Celosia’s innovative approach but also positions Australia at the forefront of global ALS research," says Celosia Therapeutics chief executive officer Dr Kathryn Sunn.

"Our CTx1000 gene therapy program capitalises on 15 years of research from the team at Macquarie University, led by Professor Ittner and Professor Ke.

"This new funding will fast-track clinical application of their research discoveries, enabling us to bring life-changing therapies to those who need them the most."

Investor Iomar Barrett says the CTx1000 program and Celosia team represent a "realistic pathway" to finding a cure for ALS.

"As a fund manager for over 20 years, it is my job to identify macro trends and emerging technologies that are poised to reshape the future," Barrett says.

"I believe that CTx1000 is one of such significance for patients around the world, with the promise of bringing tangible benefits and rewriting the narrative for ALS patients."

Uniseed chief executive officer Dr Peter Devine says the organisation is proud to lead the funding round.

"Our investment underscores our confidence in the company’s innovative gene therapy approach and the potential of CTx1000 to address a critical unmet need in ALS," Devine says.

"We are excited to support Celosia’s team as they advance toward clinical trials and continue making strides toward a transformative treatment for ALS patients worldwide."