5 Key Takeaways

• 1

  Six companies are developing therapies for Stargardt disease, affecting approximately 60,000 people in the U.S.

• 2

  Therapies under development include gene therapy, vitamin A modulation, and optogenetics, with promising early results.

• 3

  Belite Bio's tinlarebant aims to reduce toxic vitamin A delivery, stabilizing disease progression despite some visual side effects.

• 4

  Nanoscope Therapeutics' MCO-010 showed functional vision gains in a phase 2 study, with a phase 3 trial planned.

• 5

  The rapid expansion of clinical programs reflects a competitive pipeline for Stargardt disease, with no approved therapies yet.