Celosia Therapeutics, an Australian biotech company located in Sydney, has garnered an impressive $16.75 million in one of the country’s largest-ever Series A funding rounds for a biotech startup. The company has plans to use the vast investment to further develop the groundbreaking treatment for amyotrophic lateral sclerosis (ALS), a neurodegenerative disease causing the progressive loss of motor neurons.
The Series A round was led by Uniseed, Australia’s longest-standing research commercialization fund, with support from UniSuper. The majority of the funding came from hedge fund manager Iomar Barrett, who is also an ALS patient. This round of funding will accelerate the advancement of Celosia’s leading candidate, CTx1000, a novel gene therapy designed to target TDP-43, a protein strongly implicated in ALS pathology.
ALS is the most common form of Motor Neuron Disease and leads to progressive degeneration of motor neurons in the brain and spinal cord. This has resulted in a gradual loss of muscle control and ultimately death. There is as yet no cure to reverse the damage caused by ALS or halt its progression. However, preclinical studies have indicated that Celosia’s CTx1000 therapy might not only stop the disease’s progression but also reverse some of the neurological damage already done.
The gene therapy builds on breakthrough research led by Professors Yazi Ke and Lars Ittner from Macquarie University’s Dementia Research Centre. The result, published in Neuron February 2024, they pinpointed a specific interactor protein targeting toxic TDP-43 forms: in an exclusive interaction may, therefore, offer new, unique possibilities to possibly clean up the neurons harmed through ALS and restore healthy activity of normal TDP-43 function.
There was evidence in mouse models for showing that CTx1000 at advanced stages of ALS has halted the progression of ALS while, in some instances, reversing damage partially. Breakthrough treatment in the form of a “game-changer” seems to emerge for the illness of ALS, presenting, for the first time ever, a realistic opportunity in addressing the root cause and not merely the symptoms.
Celosia Therapeutics CEO Dr. Kathryn Sun said she was thrilled with the funding, which confirms that the company’s innovative approach is correct and that Australia plays an important role in ALS research around the world. “This funding will accelerate the translation of discoveries made by Professor Ittner and Professor Ke into the clinic. Our objective is to provide life-changing therapies to ALS patients who desperately need new treatment options,” she said.
Investor Iomar Barrett believes Celosia’s approach represents a significant step toward finding a cure for ALS. With over 20 years of experience in the investment sector, Barrett recognizes the potential of CTx1000 to make a meaningful impact on ALS treatment. “I believe this therapy can rewrite the narrative for ALS patients worldwide,” he said.
Uniseed, which has a long history of supporting innovative Australian startups, also expressed confidence in Celosia’s future. Dr. Peter Devine, CEO of Uniseed, emphasized the importance of addressing the unmet medical need in ALS. “We are proud to support Celosia as they advance toward clinical trials and work to develop a transformative treatment for ALS,” Devine said.
This new investment marks an exciting milestone for Celosia Therapeutics as it readies its gene therapy to enter clinical stages. The new funding will be used to further develop CTx1000, based on 15 years of research from Macquarie University, to bring this revolutionary treatment closer to ALS patients.
The importance of this funding cannot be overstated, as it offers new hope to people living with ALS. Instead of just palliating the symptoms, CTx1000 deals with the root cause of the disease, which marks a big step forward in the quest for a cure. If clinical trials are confirmed to be effective, this therapy could revolutionize the treatment of ALS and change the lives of millions of people around the world who suffer from the disease.
