BlackfinBio Limited is a spinout company from the University of Sheffield set to pioneer novel therapies for rare genetic neurological diseases.
Today, the company announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its novel adeno-associated virus (AAV) gene therapy targeting Hereditary Spastic Paraplegia, Type 47 (SPG47).
In 2023, BlackfinBio 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.
BlackfinBio’s AAV-based gene therapy aims to address the underlying genetic cause of SPG47 by delivering a functional copy of the AP4B1 gene, with the goal of halting or reversing disease progression.
“The IND submission marks a significant milestone for BlackfinBio and the SPG47 community. This achievement is the culmination of years of dedicated research and collaboration. We are deeply grateful to LifeArc, Cure AP-4, and our private investors, whose support has been instrumental in advancing this program to the clinic” says Peter Nolan, CEO of BlackfinBio Limited.
Pending regulatory clearance, BlackfinBio plans to initiate a Phase 1/2 clinical trial in the United States to assess the safety, tolerability, and preliminary efficacy of its AAV gene therapy in patients with SPG47.
“We are thrilled to take this important step forward in our mission to develop transformative therapies for patients with rare neurological diseases. We look forward to working closely with the FDA and the broader medical community as we advance this program” adds Professor Mimoun Azzouz, Scientific Founder of BlackfinBio.
For more information, please contact BlackfinBio Limited at enquiries@blackfin.bio
Additional information
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.
For more information please go to: https://blackfin.bio
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: https://cureap4.org
About LifeArc:
LifeArc is a not-for-profit medical research organisation that turns promising scientific research into impact for people living with rare diseases and global infectious diseases.
We form partnerships, and provide scientific expertise and funding to help break down the barriers preventing scientific breakthroughs from becoming life-transforming treatments and cures. We have been doing this for more than 30 years and our work has resulted in five licensed medicines, including cancer drug pembrolizumab and lecanemab for Alzheimer’s disease.
Our goal is a world where no one with a rare disease or a global infectious disease misses out on life-changing innovation because of complexity, cost or risk.
LifeArc is a company limited by guarantee (registered in England and Wales under no. 2698321) and a charity (registered in England and Wales under no. 1015243 and in Scotland under no. SC037861).
