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FDA Lifts Clinical Hold on Advanced Cell Technology's Phase I/II Clinical Trial for Stargardt's Macular Dystrophy

Strategic Insights

— 2 min read

Advanced Cell Technology, Inc., a biotechnology company applying cellular technology in the field of regenerative medicine, has announced that the US Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application to initiate a Phase I/II multicenter clinical trial using retinal cells derived from human embryonic stem cells to treat patients with Stargardt's Macular Dystrophy (SMD), a common form of juvenile macular degeneration.

The Phase I/II trial will be a prospective, open-label study designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation to patients with advanced SMD. A total of twelve patients will be enrolled in the study at multiple clinical sites, including the Jules Stein Eye Institute at UCLA, the Casey Eye Institute in Portland, Oregon, and the University of Massachusetts Memorial Medical Center in Worcester, Massachusetts.

The company's CEO, William M. Caldwell IV, stated, "Initiating our macular degeneration clinical trial represents a significant milestone in the progress of developing human embryonic stem cell-based therapies aimed at large worldwide markets." The research aims to treat a disease that affects millions worldwide, with approximately 30% of patients 75-85 years old suffering from macular degeneration.

Key Takeaways:

  • The FDA has lifted the clinical hold on Advanced Cell Technology's Phase I/II clinical trial for Stargardt's Macular Dystrophy, allowing the company to initiate the trial using retinal cells derived from human embryonic stem cells.
  • The trial will be a prospective, open-label study designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation to patients with advanced SMD.
  • Twelve patients will be enrolled in the study, with sites currently under consideration including the Jules Stein Eye Institute at UCLA, the Casey Eye Institute in Portland, Oregon, and the University of Massachusetts Memorial Medical Center in Worcester, Massachusetts.
  • The company has gained Orphan Drug designation for the company's RPE cells, allowing them to receive benefits such as tax credits and accelerated FDA approval.
  • The trial is a significant milestone in the progress of developing human embryonic stem cell-based therapies aimed at large worldwide markets.

Statistics:

  • Approximately 30 million people worldwide suffer from macular degeneration.
  • 10% of people ages 66 to 74 will have symptoms of macular degeneration.
  • The prevalence of macular degeneration increases to 30% in patients 75 to 85 years old.
  • The worldwide market for macular degeneration treatments is estimated to be $25-30 billion.

Sources:

  • Advanced Cell Technology, Inc. press release (no date specified)
  • Food and Drug Administration (FDA) (no date specified)
  • Jules Stein Eye Institute at UCLA (no date specified)
  • Casey Eye Institute in Portland, Oregon (no date specified)
  • University of Massachusetts Memorial Medical Center in Worcester, Massachusetts (no date specified)

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