Novartis Gene Therapies
Clinical trials sponsored by Novartis Gene Therapies, explained in plain language.
A subsidiary of Novartis focused on gene therapies.
Trade on eToro (ad)Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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One-Time gene therapy helps babies with deadly muscle disease sit and breathe on their own
Disease control CompletedThis phase 3 trial tested a one-time gene therapy called Zolgensma in 22 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment delivers a working copy of the missing SMN gene via an IV infusion. The main goals were …
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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One-Time gene therapy helps babies with rare muscle disease sit on their own
Disease control CompletedThis study tested a one-time gene replacement therapy in infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The main goal was to see if treated babies could sit without support for at least 10 seconds by 18 months of age. The …
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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One-Time gene therapy helps babies with fatal muscle disease sit independently
Disease control CompletedThis study tested a one-time gene replacement therapy in 33 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment aims to replace the missing SMN1 gene to improve muscle function. Key results showed that many babies…
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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Gene therapy breakthrough: one dose may help babies with rare muscle disease
Disease control CompletedThis study tested a single dose of Zolgensma gene therapy in 30 infants diagnosed with spinal muscular atrophy (SMA) before symptoms appeared. The goal was to see if the treatment helps them reach motor milestones like sitting or standing alone. The therapy delivers a working cop…
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC