Novartis believes all people with spinal muscular atrophy (SMA) – a rare, genetic, neuromuscular disease – should have access to gene therapy that addresses the root cause of their disease. 

This is one reason why we’re conducting the STEER clinical trial, a global research study of the intrathecal (IT) administration of OAV101, an investigational gene therapy. 

STEER is exploring the efficacy, safety, and tolerability of OAV101 IT in children and teens between 2 to <18 years of age who have SMA Type 2.1

All compounds under investigation are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established for this new use. There is no guarantee that they will become commercially available for the use(s) under investigation.

Introducing STEER

Child hugs dad next to dog on couch, while visiting clinician looks on and smiles

About the STEER study

STEER is a Novartis research study evaluating the clinical efficacy, safety and tolerability of OAV101 IT in patients with SMA Type 2 between 2 to <18 years of age who are able to sit but never walked, and have never received treatment for SMA. 

This research study is a randomized, double-blind, sham-controlled, global Phase 3 clinical trial.1

In STEER, patients will be randomly assigned to one of two possible treatment groups, including:1

  • Treatment with OAV101 IT
  • Sham procedure, a needle prick in the lower back without the administration of OAV101 IT

Eligible patients who have the sham procedure in the first period of the study will receive OAV101 IT in the second period of the study.

Intrathecal administration is an injection into the spine. This is performed using a thin needle.2

A sham control is a method used in research studies to help compare results, in an unbiased way, in those who receive a therapy to those who do not. The sham procedure mimics the treatment procedure but, unlike the treatment procedure, the sham procedure does not provide active treatment.3 The sham procedure is a needle prick in the lower back without any administration of OAV101 IT. The needle will break the skin, but no needle insertion for lumbar puncture will occur.1
Eligible patients who have the sham procedure in the first period of the study will receive OAV101 IT in the second period of the study. 

The treatment procedures are blinded – patients and researchers do not know who is receiving OAV101 IT or the sham – and will be assigned at random.

Check eligibility for the STEER research study here.

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Spinal Muscular Atrophy

SMA: a rare genetic disease affecting around 1 in 10,000 births worldwide4,5

  • SMA is caused by the lack of a functional survival motor neuron 1 (SMN1) gene6,7
  • Untreated, SMA leads to irreversible loss of motor neurons, affecting breathing, swallowing, and basic movement in its most severe forms6,7
  • The severity of SMA varies across a spectrum of types (Type 1–4)8

The STEER research study focuses on Type 2.

SMA Type 2

  • Accounts for approximately 30% of all SMA cases4
  • Typically presents between 6 and 18 months of age4
  • Patients learn to sit independently, but often lose this ability by their adolescent years9 

Untreated, people with SMA Type 2 will never walk without support and will need a wheelchair or other mobility aids.10

Check eligibility for the STEER research study here.

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Genes are the building blocks of DNA. Gene therapy addresses the root cause of a genetic disease by restoring the function of a missing or faulty gene with a new, working copy.11,12,13 As SMA is caused by a missing or non-functioning SMN1 gene, gene therapy can be used to treat it.6

The STEER research study will explore an investigational gene therapy known as OAV101 IT that aims to restore the function of that missing or non-functioning gene.

Middle-aged female physician evaluates young girl with braided pigtails in wheelchair, in hospital setting


OAV101 IT consists of a modified virus called adeno-associated virus (AAV), which transports a gene to motor neurons in the spinal cord to replace SMN protein production.11 Doctors call this a “vector.” AAV is not known to cause any disease in humans.

AAV9 vectors

  • They can cross the blood-brain barrier to treat cells of the central nervous system11
  • They can yield high levels of therapeutic genes and protein expression in targeted tissues12
  • They can be delivered directly to the tissue of interest; for diseases of the central nervous system, gene therapy may be administered via IT administration14
  • The size of the genetic material that can be packaged inside AAV vectors is relatively small12

Gene therapy is a useful approach to treating diseases, like SMA, that are caused by mutations in a single gene. 

How STEER works

Dad pushes pre-teen boy, wearing red sweatshirt, in wheelchair through park, as boy smiles up at mom.

Participating in the STEER study

Eligible patients who take part in the STEER study will receive:

  • Investigational gene therapy OAV101 IT and a sham procedure
  • Care and monitoring during the study
  • Support with travel to and from the site where the study is conducted

Patients will be asked to stay in the STEER research study for about 15 months.  

The research study includes two treatment periods and two follow-up periods. If an eligible patient does not receive OAV101 IT in the first period, he or she will receive it in the second period. Similarly, eligible patients who receive OAV101 IT in the first treatment period, will receive sham procedure in the second period.1

At the end of the research study, patients will be eligible to enroll in a long-term follow-up study to monitor long-term safety and efficacy.1

STEER research diagram

Staying informed during the study

Caregivers and/or patients will be able to discuss any questions or concerns with the study doctors.

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Learn more about the STEER research study.


Inclusion criteria: who can take part in the study?

To take part in the STEER research study, patients must:1

  • Be diagnosed with SMA Type 2
  • Be between 2 to <18 years of age
  • Have never received treatment with any SMN targeting therapies, such as risdiplam or nusinersen
  • Be able to sit independently but have never walked independently

Additional inclusion and exclusion criteria apply. For more information about the STEER research study and key inclusion and exclusion criteria, visit the STEER research study page

Clinical trials are voluntary research studies conducted in people and designed to answer specific questions about the safety or efficacy of drugs, therapies, or new ways of using other existing treatments.15,16  Clinical trial results are important because they give researchers more information about the risks and benefits of an investigational therapy.

Participation in a clinical trial means patients may also benefit from:

  • Greater clinical monitoring
  • Access to an investigational treatment16

Research studies are closely monitored to ensure they are carried out correctly and that the rights of the patients are protected.17

Participation in a research study is always voluntary.

Learn more about clinical trials by visiting the Novartis Clinical Trials FAQs.

The individual study sites will lead recruitment of study participants. For more information, visit the STEER research study.


  1. Novartis Gene Therapies, Inc. Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) (STEER). identifier: NCT05089656. Updated June 28, 2022. Accessed July 1, 2022.
  2. MedLink Neurology. Intrathecal administration of drugs. Available at: Accessed July 1, 2022.
  3. Brim RL, Miller FG. The potential benefit of the placebo effect in sham-controlled trials: implications for risk-benefit assessments and informed consent. J Med Ethics. 2013;39:703-707.
  4. Verhaart IEC, Robertson A, Wilson IJ, et al. Prevalence, incidence and carrier frequency of 5q–linked spinal muscular atrophy –a literature review. Orphanet J Rare Dis. 2017;4;12(1):124.
  5. Verhaart IEC, Robertson A, Leary R, et al. A multi-source approach to determine SMA incidence and research ready population. J Neurol. 2017;264(7):1465-1473.
  6. Anderton RS and Mastaglia FL. Advances and challenges in developing a therapy for spinal muscular atrophy. Expert Rev Neurother. 2015;15(8):895–908.
  7. Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurol. 2014;83(9):810-817.
  8. Govoni A, Gagliardi D, Comi GP, Corti S. Time is motor neuron: therapeutic window and its correlation with pathogenetic mechanisms in spinal muscular atrophy. Mol Neurobiol. 2018;55(8):6307-6318.
  9. National Center for Advancing Translational Sciences. Genetic and Rare Diseases Information Center. Spinal muscular atrophy type 2. Available at:…. Accessed July 1, 2022.
  10. Darras BT, Finkel RS. Natural history of spinal muscular atrophy. In: Sumner CJ, Paushkin S, Ko CP, eds. Spinal Muscular Atrophy: Disease Mechanisms and Therapy, 2nd ed. London, UK: Academic Press/Elsevier;2017:399‒421.
  11. Global Genes. A Guide to Gene Therapy.…. Accessed July 1, 2022.
  12. Saraiva J, Nobre RJ, Pereira de Almeida L. Gene therapy for the CNS using AAVs: the impact of systemic delivery by AAV9. J Control Release. 2016;241:94–109.
  13. NIH. National Library of Medicine. How does gene therapy work? Accessed July 1, 2022.
  14. Naso MF, Tomkowicz B, Perry WL 3rd, Strohl WR. Adeno-associated virus (AAV) as a vector for gene therapy. BioDrugs. 2017;31(4):317-334.
  15. U.S. Food and Drug Administration. Clinical trials: What patients need to know. Accessed July 1, 2022.
  16. Friedman LM, Furberg CD, DeMets DL, Reboussin DM, Granger CB. Fundamentals of clinical trials. Switzerland: Springer International Publishing; 2015. Available at:…. Accessed July 1, 2022.
  17. Novartis. Clinical Trials FAQs. Available at: . Accessed July 1, 2022.