Onasemnogene abeparvovec is a Gene Therapy owned by Novartis Gene Therapies, and is involved in 12 clinical trials, of which 6 were completed, 5 are ongoing, and 1 is planned.

Onasemnogene abeparvovec is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression.

Onasemnogene abeparvovec acts as survival motor neuron protein activator. It is comprised of complementary DNA (cDNA) of the human SMN gene, NAV rAAV9 vectors and promoter. AAV9’s components are removed and replaced with the SMN transgene. CB promoter and BGH polyadenylation signal (BGH pA) are added for mRNA stability and protection from hydrolytic enzymes. The viral vector will deliver the SMN transgene into the motor neurons and produce full-length, functional SMN protein and treats spinal muscular atrophy.

The revenue for Onasemnogene abeparvovec is expected to reach a total of $34.7bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Onasemnogene abeparvovec NPV Report.

Onasemnogene abeparvovec was originated by Ohio State University and Nationwide Children’s Hospital and is currently owned by Novartis Gene Therapies. Novartis is the other company associated in development or marketing of Onasemnogene abeparvovec.

Onasemnogene abeparvovec Overview

Onasemnogene abeparvovec (Zolgensma) is an adeno-associated viral vector-based gene therapy produced in human embryonic kidney cells by recombinant DNA technology. It is formulated as suspension and solution for intravenous route of administration. Zolgensma is indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Onasemnogene abeparvovec (scAAV9.CB.SMN) is under development for the treatment of spinal muscular atrophy type 1, type 2 and type 3. It is administered intravenously and intrathecal route. It is developed by using NAV-vector technology. It carries a transgene into the cells of the body using NAV rAAV9 vectors (Adeno-associated virus) (AAV).

Novartis Overview

Novartis is a healthcare company that focuses on the discovery, development, manufacture and marketing of prescription and generic pharmaceutical products and eye care products. It provides drugs for the treatment of cancer, cardiovascular diseases, dermatological conditions, neurological disorders, ophthalmic and respiratory diseases, immune disorders, and infections, among others. The company offers generic medicines and biosimilars through Sandoz. Novartis conducts research in various disease areas through The Novartis Institutes for BioMedical Research (NIBR). The company operates through a network of subsidiaries and offices across the Americas, Europe, the Middle East, Africa, and Asia-Pacific. Novartis is headquartered in Basel, Switzerland.

The company reported revenues of (US Dollars) US$52,877 million for the fiscal year ended December 2021 (FY2021), an increase of 6% over FY2020. In FY2021, the company’s operating margin was 22.1%, compared to an operating margin of 20.3% in FY2020. In FY2021, the company recorded a net margin of 45.4%, compared to a net margin of 16.2% in FY2020. The company reported revenues of US$12,842 million for the third quarter ended September 2022, a decrease of 1.9% over the previous quarter.

Quick View – Onasemnogene abeparvovec

Report Segments
  • Innovator (NME)
Drug Name
  • Onasemnogene abeparvovec
Administration Pathway
  • Intrathecal
  • Intravenous
Therapeutic Areas
  • Central Nervous System
Key Companies
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.