University of Sheffield spinout company to pioneer novel therapies for genetic neurological diseases | News

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BlackfinBio has raised £2.75 million seed investment to advance revolutionary treatments for Hereditary Spastic Paraplegia (HSP).

  • BlackfinBio has raised £2.75 million seed investment to advance pioneering treatments for Hereditary Spastic Paraplegia (HSP).
  • The company will manage the development of Hereditary Spastic Paraplegia, sub-type 47 (SPG47) gene therapy and raise funds for clinical trials
  • The SPG47 disease was only characterised in 2012 and affects 103 children worldwide, although genetic analysis estimates this is closer to 3,300 people
  • There is currently no effective treatment available to address the cause of this neurodegenerative disease

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 leading gene therapy expert Professor Mimoun Azzouz, Chair of Translational Neuroscience in the internationally renowned 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.”
 


Additional information

About the funders

Professor Mimoun Azzouz Founder and Chief Science Officer. Professor Azzouz is an internationally renowned expert in the field of gene therapy. With an extensive background in neuroscience and gene therapeutics, he has made significant contributions to the development of innovative gene-based treatments for neurological disorders. His research focuses on the delivery of therapeutic genes to the central nervous system, particularly in the context of neurodegenerative diseases like Parkinson’s and motor neurone diseases. Driven by a passion for translational science, Mimoun Azzouz’s work bridges the gap between bench and bedside, aiming to bring promising therapies from the laboratory to clinical application. Through his pioneering efforts, he has emerged as a leading figure in the field of gene therapy for neurological conditions, offering hope for patients worldwide. Azzouz has also won prestigious awards in keeping with the excellence of his science, including the ERC Advanced Investigator (2011), ERC Proof-of-Concept (2017) and IMI award (2020).

Peter Nolan Founder, Chair & Interim CEO –  Formerly Director, CBO, and key executive of Oxford Biomedica since its inception; over 25 yrs experience in gene therapy commercialization; led negotiations on key deals including Novartis and Sanofi.

Chris Edwards, Founder and Chief Business Officer. Father of Robbie who is an SPG47 patient. Chris is a seasoned entrepreneur who founded, managed, and exited a series of successful startup companies during his career. He has extensive experience in building/managing teams with diverse skills sets, and with navigating complex governmental regulations and problem solving.

About BlackfinBio
In 2023, BlackfinBio emerged as a spinout from the University of Sheffield, focusing on the development of groundbreaking gene therapies aimed at addressing the significant unmet medical needs of rare genetic diseases such as SPG47. The foundation of BlackfinBio is built upon the research conducted by world-renowned scientist Professor Mimoun Azzouz based at SITraN (Sheffield Institute for Translational Neuroscience) at the University of Sheffield. BlackfinBio aims to advance genetic research methods to improve the treatment landscape for those affected by these rare debilitating conditions.
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About Cure AP-4
Cure AP-4 is a non-profit organization founded in 2016 by families of two of the known SPG47 patients. It was originally founded as Cure SPG47, but the mission has since expanded to include all four AP-4 related disorders due to shared natural history, goals and patient/family needs. 
For more information please go to:

About LifeArc
LifeArc is a self-funded, non-profit medical research organisation. We take science ideas out of the lab and help turn them into medical breakthroughs that can be life-changing for patients. We have been doing this for more than 25 years and our work has resulted in five licensed medicines and a diagnostic for antibiotic resistance. Our teams are experts in drug and diagnostics discovery, technology transfer, and intellectual property. Our work is in translational science – bridging the gap between academic research and clinical development, providing funding, research and expert knowledge, all with a clear and unwavering commitment to having a positive impact on patient lives. LifeArc is committed to spending £1.3 billion by 2030 in areas of high unmet medical need. 
For more information please go to: www.lifearc.org

About LifeArc Philanthropic Fund
The LifeArc Philanthropic Fund provides grants and funding to academic researchers working to advance new treatments and diagnostics for rare diseases. As of December 2022, the LifeArc Philanthropic Fund has awarded £14.6 million to 45 research projects since 2017. 24 of those grants have been to projects co-supported by our charity partners and three of the projects are at the clinical trial stage of development. The awarded grants comprise research that addresses 33 rare disease indications.
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