AMN

Data show SBT101 effectively delivered a functional copy of the ABCD1 gene to spinal cord tissues with resultant protein expression, addressing the underlying cause of disease and improving function in preclinical models.

Highlighted in peer-reviewed journal Molecular Therapy Methods & Clinical Development, these data supported the advancement of SBT101 into a Phase 1/2 clinical trial.

Spur Therapeutics today announced the publication of preclinical proof-of-concept data for SBT101, its potential first-in-class gene therapy program for the treatment of adrenomyeloneuropathy (AMN), in Molecular Therapy Methods & Clinical Development. AMN is a rare and devastating neurodegenerative disease caused by a mutation in the ABCD1 gene and characterized by progressive muscle weakness and sensory loss, leading to impaired mobility, increased risk of falls, incontinence and debilitating pain.

"People living with AMN currently have no treatment options to slow or alter the progression of this devastating disease, and gene therapy presents the opportunity to address the disease at its root cause,” said Pamela Foulds, M.D., Chief Medical Officer at Spur. "These data demonstrate SBT101's potential to halt disease progression by delivering a functioning ABCD1 gene to generate the adrenoleukodystrophy protein (ALDP) lacking in AMN. We are currently advancing SBT101 in a Phase 1/2 trial with the aim of developing a life-changing gene therapy for people with AMN.”