Drug Profile

TW 002

Alternative Names: AAV5-GDNF gene therapy; ALS gene theapy - uniQure/Treeway; TW-002

Latest Information Update: 28 Oct 2023

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At a glance

Originator uniQure
Developer Treeway
Class Gene therapies; Nerve growth factors
Mechanism of Action Gene silencing; Gene transference; Glial cell line-derived neurotrophic factor agonists

Orphan Drug Status

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

No
New Molecular Entity No

28 Oct 2023 No recent reports of development identified for preclinical development in Amyotrophic-lateral-sclerosis in Netherlands (Parenteral)
27 Oct 2021 TW 002 is still in preclinical trials for Amyotrophic lateral sclerosis in Netherlands (Treeway pipeline, October 2021)
28 Feb 2020 No recent reports of development identified for preclinical development in Amyotrophic-lateral-sclerosis in Netherlands (Parenteral)

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