Novartis Gene Therapies
Illinois
United States
106 articles with Novartis Gene Therapies
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Health Alert for Parents: How one boy is thriving following treatment with a gene therapy after receiving an early diagnosis
10/12/2022
Two years ago, a mother received a phone call with a devastating diagnosis. That mother shares her son's experience with spinal muscular atrophy, a rare genetic disease, and how early diagnosis and treatment transformed his life.
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Novartis released new data Monday from its Phase III SPR1NT trial that reinforces the benefits of Zolgensma.
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After guiding the commercial rollout of embattled Alzheimer’s drug Aduhelm for Biogen, Johanna Rossell has departed the company for a leadership role at Enzyvant.
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From the continuing fight against COVID-19 to new companies emerging in exciting therapeutic areas to the people who mattered most, here’s a look at just some of the biggest successes, most dramatic flops – and a few that fall somewhere in between.
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Novartis is the subject of a DOJ inquiry over the company’s marketing and pricing of Entresto over the past five years, receiving a Civil Investigative Demand from the government.
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The drug is limited to patients who test at least 20% for a Ki-67 score on an FDA-approved test.
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Why not take advantage of the perks of a lower cost of living, slower pace, and opportunities to make a big impact in some important disease areas?
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Philip Morris will acquire Vectura to gain access to its inhaled drug formulas to bolster its Beyond Nicotine strategy.
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Scorpion Therapeutics announced its new CEO Dr. Axel Hoos while Tenax Therapeutics named Christopher Giordano as its new chief executive.
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New data shows Zolgensma, Novartis’ gene therapy for spinal muscular atrophy, has the potential to be used presymptomatically in juveniles.
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Molecular Partners announced yesterday it has filed for a $100 million initial public offering with the Securities and Exchange Commission in the U.S., funding which will go toward supporting the company's work in the development of protein-based treatments for COVID-19 and various cancers.
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AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)
5/19/2020
AveXis, a Novartis company, today announced the European Commission (EC) granted conditional approval for Zolgensma® (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.
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Beyond COVID-19: Biotech Beach Companies Reinvigorate Small Molecules, Cannabinoids and More
4/15/2020
The COVID-19 battle dominates the news, but it doesn’t constitute all the news. Here’s what several Southern California Biotech Beach companies are doing that you might have missed. -
AveXis receives positive CHMP opinion for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)
3/27/2020
AveXis, a Novartis company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending conditional marketing authorization of Zolgensma® (onasemnogene abeparvovec).
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Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing
3/24/2020
AveXis, a Novartis company, today announced a one-time infusion of Zolgensma® (onasemnogene abeparvovec-xioi) showed rapid, significant and clinically meaningful therapeutic benefit in patients with spinal muscular atrophy (SMA) across a range of studies, including in patients treated presymptomatically, and sustained durability in patients now up to five years post-dosing and some patients more than five years of age.
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AveXis presents AVXS-101 IT data demonstrating remarkable increases in HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type 2
3/24/2020
AveXis, a Novartis company, today announced that new data from the Phase 1/2 STRONG study demonstrated a one-time intrathecal (IT) administration of AVXS-101 in patients ≥2 years and <5 years old with spinal muscular atrophy (SMA) Type 2 who received Dose B (1.2 x 1014 vg) met the primary efficacy endpoint.
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Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma® the only gene therapy for patients with spinal muscular atrophy (SMA)
3/19/2020
Novartis Pharma K.K. (“Novartis Pharma”) today announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) approved Zolgensma® (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA) in patients under the age of two, including those who are pre-symptomatic at diagnosis.
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Clinical Catch-Up: October 7-11
10/14/2019
It was a busy week for clinical trial announcements. Here’s a look. -
In a 56-page response to the investigation from the U.S. Food and Drug Administration, Novartis said the Kaspar brothers personally manipulated or instructed others to alter some of the preclinical data that ultimately led to the approval of the $2.1 million priced SMA therapy.
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Clinical Catch-Up: September 16-20
9/23/2019
Last week was an unusually busy week for clinical trial news, with numerous companies presenting results at conferences. Here’s a look at the top stories.