Patient Family Creates BlackfinBio to Develop Gene Therapies

Rare Daily Staff

Cure AP-4 co-founders Kasey and Chris Edwards have entered into a business partnership that includes the University of Sheffield and Cure AP-4 to launch BlackfinBio, which will advance gene therapy programs into human clinical trials.

BlackfinBio will use a platform approach developed by Principal Investigator Mimoun Azzouz of the Sheffield Institute for Translational Neuroscience. The company will initially focus on its lead program for SPG47, a form of the neurodevelopmental condition spastic paraplegia, but said it plans to advance gene therapy research for additional disorders of the central nervous system by insights gained from that work.

Pathological variants in the AP4B1, AP4E1, AP4M1 and AP4S1 genes cause AP-4-associated hereditary spastic paraplegia (AP-4 HSP). Disease symptoms become apparent in infancy, generally presenting with loss of muscle tone progressing to spasticity and loss of lower and, sometimes, upper limb function, accompanied by global developmental disability, and seizures. Cure AP-4 (formerly Cure SPG47) was co-founded in 2016 by Kasey and Chris Edwards and Angela and Kevin Duffy after their daughters were diagnosed with SPG47.

Cure AP-4 provided financing to Azzouz’s SPG47 gene therapy, which provided the nonprofit an equity stake.

“The initial priority was to develop a therapeutic to halt the trajectory of the disease threatening the futures of Robbie and Molly,” said Chris Edwards. “Launching our own venture became the most expedient way to advance the promising treatment developed by Dr. Azzouz and the University of Sheffield.”

The Edwards family has agreed to provide seed capital for the venture, with Chris Edwards serving as interim chief business officer and board member.