University spin-out company to pioneer novel therapies for genetic neurological diseases

A new spin-out company from the University of Sheffield is set to pioneer novel therapies for rare genetic neurological diseases.

BlackfinBio has raised £2.75 million seed investment to advance revolutionary treatments for Hereditary Spastic Paraplegia (HSP). The company will manage the development of gene therapy for the HSP sub-type 47 (SP47) and raise funds for clinical trials.

SPG47 is a slowly-progressing rare disease that causes malformation of the brain. It is caused by a specific mutation in the SPG47 gene that creates a deficiency of the AP4B1 protein which normally transports other molecules around inside the cells of the nervous system. Symptoms such as muscle weakness, developmental delays, impaired speech and progressive motor skills often show up in early childhood. There is currently no cure and no effective treatment for this debilitating disease.

BlackfinBio is built on research from gene therapy expert Professor Mimoun Azzouz, Chair of Translational Neuroscience in the Sheffield Institute for Translational Neuroscience at the University of Sheffield.

The company was conceived when initial research performed by Professor Azzouz, funded by US charity Cure AP-4, provided proof-of-concept for the potential to effectively restore the AP4B1 gene through gene therapy. Based on these results, further funding from the LifeArc Philanthropic Fund was awarded to Professor Azzouz to validate the therapy in pre-clinical models and produce the gene therapy batches under clinical grade manufacturing required to move on to clinical trials in the near future.

This pioneering research showed that through AP4B1 gene replacement therapy it is possible to put a correct copy of the faulty gene back into the patients to stop their nervous systems being damaged further. This treatment will be carried out through injected gene therapy based on a harmless viral vector (carrier). One dose will be enough to treat a patient for a lifetime.

Led by experienced executive Peter Nolan, formerly of the multinational gene therapy company, Oxford Biomedica the company will use the initial funding to advance gene therapy treatments for the SPG47 disease before proceeding with in-human clinical trials.

Professor Mimoun Azzouz, Academic Founder of BlackfinBio, Professor and Chair of Translational Neuroscience at the University of Sheffield, Founder and Director of Gene Therapy Innovation and Manufacturing Centre (GTIMC), said: “The launch of BlackfinBio is a significant milestone which will enable the progress of the translational pipeline being developed by the company towards potential life-changing therapies for patients with HSP and other diseases.

“I am delighted by the overwhelming support by Cure AP-4 and LifeArc to progress the SPG47 gene therapy project through the clinical development pathway. BlackfinBio is a great addition to the favourable and dynamic gene therapy ecosystem at the University of Sheffield.”

Peter Nolan, Co-Founder, Chair & Interim CEO, said: “I am delighted to be able to work again with Professor Azzouz and help him translate his world-class research into successful gene therapy products for the benefit of patients who have genetic diseases where there are no current treatments. BlackfinBio will seek to build a pipeline of such gene therapies for a range of neurological conditions.”

Chris Edwards, Co-Founder and Chief Business Officer and President of Cure AP-4, said: “Cure AP-4’s initial priority was to develop a therapeutic to halt the trajectory of this devastating disease. Launching a commercial venture in partnership with the University of Sheffield became the most expedient way to advance the promising treatment developed by Professor Azzouz and his team.

“We are delighted to be a part of this important milestone, and being positioned to advance this potentially life changing treatment into the clinic through BlackfinBio.”

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