Drug Profile

AVR RD 04

Alternative Names: AVR-RD-04; CTNS-RD-04

Latest Information Update: 05 Mar 2025

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At a glance

Originator GenStem Therapeutics
Developer Novartis; Papillon Therapeutics
Class Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
Mechanism of Action CTNS protein stimulants; CTNS replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Cystinosis
New Molecular Entity No

Highest Development Phases

Phase I/II Cystinosis

Most Recent Events

05 Mar 2025 Phase-I/II clinical trials in Cystinosis in USA (IV) (Papillon Therapeutics Pipeline, March 2025)
20 Jun 2024 AVROBIO has been merged with Tectonic Therapeutic to form Tectonic Therapeutic
12 Jul 2023 Suspended - Phase-I/II for Cystinosis in USA (IV) as AVROBIO is exploring strategic alternatives

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