Adrenomyeloneuropathy

FDA Grants Fast Track Designation to SBT101, the First Investigational AAV-Based Gene Therapy for Patients With Adrenomyeloneuropathy (AMN)

Retrieved on: 
Wednesday, February 16, 2022

AMN is an adult-onset progressive, degenerative spinal cord disease caused by mutations in the ABCD1 gene leading to loss of mobility, incontinence and pain.

Key Points: 
  • AMN is an adult-onset progressive, degenerative spinal cord disease caused by mutations in the ABCD1 gene leading to loss of mobility, incontinence and pain.
  • We look forward to continuing to advance SBT101 as we work toward our goal of bringing life-changing treatments to patients.
  • The Fast Track process is designed to facilitate the development and expedite the review of investigational treatments that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions.
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions.

SwanBio Therapeutics Announces First Patient Enrolled in Natural History Study to Evaluate Patients with Adrenomyeloneuropathy

Retrieved on: 
Tuesday, June 29, 2021

CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD) occurring in adulthood.

Key Points: 
  • CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD) occurring in adulthood.
  • This observational, multinational study will prospectively evaluate patients to assess the course of the disease and provide insights into potential endpoints and designs for future clinical trials.
  • We are pleased to initiate this natural history study for AMN, which has been strategically designed with patients in mind, and allows for the majority of monitoring to be conducted virtually from each patients home.
  • The global study will enroll approximately 80 adult male patients with a confirmed diagnosis of adrenoleukodystrophy (ALD).