The US Food and Drug Administration has cleared BlackfinBio’s investigational new drug (IND) application for a Phase I/II trial of BFB-101, an adeno-associated virus gene therapy designed to treat hereditary spastic paraplegia type 47 (SPG47).
The open-label, single-centre trial will take place at Boston Children’s Hospital, with recruitment anticipated to start by the end of this year.
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It will primarily evaluate the safety and efficacy of BFB-101 when delivered via intra-cisterna magna (ICM) administration, which involves injecting the drug into the cerebrospinal fluid at the brainstem base near the spinal cord.
This method of administration is intended to allow the therapy to be delivered rapidly to the central nervous system.
Up to five children with AP4B1-associated SPG47 will receive the treatment, with the trial’s secondary objectives due to include assessing its impact on motor function, development, and quality of life related to health.
BFB-101 has received orphan drug and rare paediatric disease designations from the FDA for SPG47, a rare condition caused by mutations in the AP4B1 gene that results in progressive lower-limb spasticity and developmental delays in children.
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By GlobalDataThe gene therapy is tailored to deliver the AP4B1 gene’s functional copy to halt or reverse the progression of the disease.
BlackfinBio CEO and founder Peter Nolan said: “The FDA’s clearance of the BFB-101 IND is an important milestone for our rare neurological disease programme and the company.
“We look forward to initiating enrolment in the US later this year and are working closely with the investigator team at Boston Children’s Hospital to evaluate the therapeutic utility of this gene therapy in children with SPG47.”
Based in Sheffield, UK, BlackfinBio is a clinical-stage gene therapy company that develops treatments for rare neurological diseases.
The company’s academic founder, Professor Mimoun Azzouz, developed BFB-101 with support from LifeArc and Cure AP-4.
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