First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration

计划入组 12 人 · 首次发布于 2025 年 12 月 8 日

Clinical Registry Investigating Bardet-Biedl Syndrome

计划入组 1,200 人 · 首次发布于 2014 年 12 月 31 日

Genetics and Clinical Characteristics of Bardet-Biedl Syndrome

计划入组 194 人 · 首次发布于 2004 年 2 月 19 日

Bardet-biedl syndrome therapeutics(Rescindo Therapeutics, Inc.)

AAV8-RK-BBS10

Setmelanotide(Imcivree)

C₄₉H₆₈N₁₈O₉S₂

ALPORT SYNDROME FOUNDATION

United States

NHI-1

United States

International Rett Syndrome Foundation

United States

$2 Million Grant Awarded to Advance Gene Therapy Trial for Rare Childhood Blindness

A Race Against Blindness Awards $1 Million to Advance AXV-101 Gene Therapy for Childhood Blindness

FDA Expands Setmelanotide Approval to Include Children Aged 2-5 with Rare Obesity Forms