It has been a bumpy road but Novartis has made a major leap in advancing an intrathecal version of its spinal muscular atrophy (SMA) gene therapy Zolgensma which would significantly expand the number of people who would be eligible to receive the transformational drug.
Novartis Hopes To Broaden SMA Patient Pool After Intrathecal Zolgensma Success
The Swiss giant’s blockbuster gene therapy is currently only administered intravenously into patients with spinal muscular atrophy under two years of age. Its intrathecal candidate, called OAV-101, has just passed a Phase III test which could expand its use in children and young adults.
More from Gene Therapies
• By
Deal Snapshot: Lilly is the third company to sign a licensing deal for STAC-BBB with Sangamo, which also aims to secure a deal for its Fabry disease program in the second quarter.
• By
The company closed a $150m series C funding round shortly after opening the second part of its Phase I/II study of ATSN-201 and partnering with a Nippon Shinyaku on ATSN-101.
• By
The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
• By
The founder and CEO of Ultragenyx talked with Scrip as the company approaches a pivotal data readout and awaits US FDA action on its first gene therapy.
More from Therapy Areas
• By
Investing heavily in antibody-drug conjugates, AbbVie is pioneering a dual STEAP1xPSMA-targeting approach in prostate cancer, as other candidates advance.
• By
Strong sales growth for the German group’s SGLT2 inhibitor in 2024
• By
The study’s failure in the overall MDD population was not a surprise, but solriamfetol’s efficacy in MDD with EDS provided a rationale for testing it in that subpopulation.