Tofersen (Qalsody)

Category: compound

An antisense oligonucleotide targeting SOD1 mRNA, approved by the FDA for SOD1-mutated ALS. The first gene therapy specifically targeting an ALS-causing gene.

Mechanism Detail

Tofersen binds to SOD1 mRNA via complementary base pairing, triggering RNase H-mediated degradation of the mRNA. This reduces production of toxic mutant SOD1 protein. Administered intrathecally to bypass the blood-brain barrier.

Clinical Status

FDA accelerated approval (2023) for SOD1-ALS. ATLAS trial ongoing for presymptomatic SOD1 carriers.

Relevant Diseases

• ALS

Relevant Therapies

• Gene Therapy

Related Terms

• SOD1
• Antisense oligonucleotide
• Gene silencing
• Intrathecal delivery