Drug intelligence / Profile preview

tofersen + 99mTc-MAG3-BIIB067

Development stage
Unknown
Lead developer
Biogen
Modality
Antibody-Radionuclide Conjugates → Antibody Conjugates → Antibody-Based Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intrathecal, Intravenous
01

Overview

**Tofersen + 99mTc-MAG3-BIIB067** is a combination of an antisense oligonucleotide drug (tofersen), used for the treatment of amyotrophic lateral sclerosis (ALS) with confirmed SOD1 mutation, and a technetium-99m–labeled mercaptoacetyltriglycine (99mTc-MAG3), a radiopharmaceutical. This specific combination is utilized for imaging and in vivo distribution studies to assess the CNS biodistribution of tofersen, with 99mTc-MAG3 serving as a radiotracer. Tofersen acts by binding SOD1 mRNA, reducing SOD1 protein synthesis and thereby targeting the underlying pathogenic mechanism in SOD1-ALS. 99mTc-MAG3 is a renal imaging agent commonly used for nuclear medicine assessments, here chemically conjugated for the specific purpose of tracing tofersen pharmacokinetics via SPECT/CT imaging.

Other names
tofersen + 99mTc-MAG3-BIIB067
02

Targets

SOD (Manganese Superoxide Dismutase)

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