Clinical Trials for SMA

Intro

In these pages, we outline the drugs that have reached human clinical trial stages and show promise in treating SMA (see pipeline).

There are currently four main approaches used to develop drugs for SMA (see table below), with others being explored in pre-clinical development:

Replacement of the SMN1 gene, to increase production of full length, functional SMN protein, for example, onasemnogene abeparvovec-xioi (Zolgensma)
Enhancement of the SMN2 gene, to increase production of full length, functional SMN protein, as done by nusinersen (Spinraza) and risdiplam (Evrysdi)
Enhancement of muscle growth and strength, as done by apitegromab and taldefgrobep alfa
Protection of the motor neurons

In addition, combinations of approaches are being explored, such as enhancement of the SMN2 gene together with enhancement of muscle growth and function.

Combination Therapy

In focus

SMA Drug Pipeline

By Treatment Target, June 2023

Download pipeline

: Mechanism of action
Zolgensma Novartis Gene Therapies:: Increases SMN
Spinraza (Nusinersen) - Biogen:: Increases SMN
Risdiplam (Evrysdi) - Roche:: Increases SMN
Apitegromab (SRK-015) - Scholar Rocks:: SMN independent

: Strategy
Zolgensma Novartis Gene Therapies:: SMN1 gene replacement
Spinraza (Nusinersen) - Biogen:: SMN2 splicing modifier (gene enhancement)
Risdiplam (Evrysdi) - Roche:: SMN2 splicing modifier (gene enhancement)
Apitegromab (SRK-015) - Scholar Rocks:: Muscle enhancement

: Drug type
Zolgensma Novartis Gene Therapies:: Gene therapy
Spinraza (Nusinersen) - Biogen:: ASO
Risdiplam (Evrysdi) - Roche:: Small molecule
Apitegromab (SRK-015) - Scholar Rocks:: Antibody

: Delivery method
Zolgensma Novartis Gene Therapies:: IV
Spinraza (Nusinersen) - Biogen:: IT
Risdiplam (Evrysdi) - Roche:: Oral
Apitegromab (SRK-015) - Scholar Rocks:: IV

SMA Drug Pipeline

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