Drug Profile

NOGA 101

Alternative Names: BTK-001; NOGA-101

Latest Information Update: 11 Aug 2025

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At a glance

Originator Noga therapeutics
Class Gene therapies
Mechanism of Action Gene transference

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

Highest Development Phases

Preclinical Agammaglobulinaemia

Most Recent Events

11 Aug 2025 Pharmacodynamics data from a preclinical trial in Agammaglobulinaemia released by Noga Therapeutics (Noga Therapeutics pipeline; August 2025 )
13 Apr 2023 Preclinical trials in Agammaglobulinaemia in Israel (Parenteral) (Noga Therapeutics pipeline, April 2023)
12 May 2020 Noga therapeutics and Lonza enters into an R&D agreement for lentiviral gene therapies for Primary immune deficiencies

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