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����app Therapeutics (����app), a biotechnology company developing novel therapeutics that reverse mitochondrial dysfunction to arrest the progression of degenerative diseases, announces the final close of its seed extension financing round, with £4m raised to date from existing investors.

The company’s first-in-class therapeutic pipeline targets mitochondria dysfunction linked to the progression of rare diseases such as the neuromuscular disorder Duchenne muscular dystrophy (DMD), the neurodegenerative disorder Huntington’s disease (HD), and also implicated in common neurodegenerative diseases. The Company has promising preclinical results, which validate its platform in a mammalian model of severe neuromuscular disease. The platform is extendable to other degenerative diseases, and additional evidence has been generated in a mammalian respiratory disease model through its academic collaborators.

Dr. Jon Rees, Chief Executive Officer of ����app, said, “Degenerative diseases place great burdens on patients and healthcare systems. We are the first biotech to reverse the impairment in sulfide-signaling driving mitochondrial dysfunction, which underlies the pathophysiology of degenerative disease in muscular dystrophy as well as the dementias. Our approach has the potential to finally address the huge unmet clinical need in these diseases. This seed extension financing round enables us to advance a lead asset toward the clinic and provides a firm foundation to secure a Series A to take us to the clinic within two years. We are also seeking partners to develop our assets which demonstrate anti-fibrotic and anti-inflammatory properties in COPD and other respiratory diseases."

Oliver Sexton, Investment Director at the UK Innovation & Science Seed Fund, said, “����app’s approach to mitochondrial dysfunction will potentially develop a treatment for degenerative diseases that have a hugely harmful impact. Such diseases affect both young and elderly patients who desperately need more treatment options. UKI2S is delighted to continue supporting ����app’s development.”

����app’s Chief Development Officer, Dr Christine Charman, said, “Interim results in our Duchenne program demonstrate that our muscle-penetrative lead asset preserves strength in oxidative muscle, and confirms mitochondrial modulation. These results will be presented at the Muscular Dystrophy Association Clinical and Scientific Conference in Orlando during March 2024.”

����app’s academic founder, and Chief Scientific Officer, Professor Matt Whiteman of the University of Exeter and collaborators, led by Prof Phil Hansbro at Australia’s Centenary/UTS Centre for Inflammation, published an abstract at the 10th Annual Meeting of the International Cytokine and Interferon Society, on research which demonstrated anti-fibrotic activity and confirmed the preservation of lung function in a model of COPD (1). Prof Whiteman and collaborators have previously demonstrated neuroprotection in a mammalian model of Alzheimer’s disease, through the restoration of sulfide-signalling (2).

This seed extension financing round was backed by a broad range of investors including the UK Innovation & Science Seed Fund, Wren Capital, Longevitytech.fund, The Fink Family Office, the Science Angel Syndicate Network, Oxford Technology Management, as well as angel investors. It will be used to fund pre-clinical work in HD, the activation of the company’s first neurodegenerative disease program, and to explore research collaborations and partnerships. The company expects to see preclinical readouts in DMD, HD, and COPD, by the end of Q2 2024.