July 9, 2026 Apertura Gene Therapy Announces Cooperative Research and Development Agreement with Institutes of the NIH to Test Investigational Gene Therapy for Niemann-Pick Disease Type C1 — Cooperative Research and Development Agreement (CRADA) will utilize Apertura’s TfR1 CapX™ Capsid — — Ara Parseghian Medical Research Fund, a non-profit organization dedicated to finding a treatment …
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Research News
Mandos Submits New Drug Application to U.S. FDA for Adrabetadex in Infantile-Onset NPC!
• Comprehensive submission package includes evidence of substantially improved survival among adrabetadex-treated patients with infantile-onset Niemann Pick disease type C (NPC), intended to serve as a single adequate and well-controlled study, and confirmatory evidence of biomarkers and nonclinical data demonstrating effects on the underlying pathology • Priority Review was requested on the basis that the …
Adrabetadex (Cyclodextrin) receives FDA Breakthrough Therapy Designation for Individuals with Infantile-Onset NPC
Breakthrough Therapy Designation is based on FDA’s review of survival analyses comparing adrabetadex-treated patients with external controls and marks a significant regulatory milestone ahead of an New Drug Apllication submission. THOUSAND OAKS, Calif.– December 9, 2025 – Beren Therapeutics P.B.C.®, a Public Benefit Corporation and parent company of Mandos LLC®, today announced that Mandos received Breakthrough Therapy Designation …