Novartis Gene Therapies
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About Novartis Gene Therapies
Since 2013, Novartis Gene Therapies (formerly AveXis) has had one focus: bringing change to those devastated by genetic diseases.
The Novartis Gene Therapies culture embraces this mission. As a compassionate and dedicated team, we are enthusiastic about the science behind our work and finding answers to difficult questions. We are dedicated to communities affected by rare diseases, and these patients and families are the motivation for everything we do.
We have built a team with exceptional depth of experience, unified by a common vision; to develop gene therapies with the potential to positively impact the lives of the patients and families devastated by rare and life-threatening neurological genetic diseases. Though we are proud of what we have achieved to date, we remain relentlessly focused on making that vision a reality.
With cutting-edge technology, we are making progress in the treatment of rare and life-threatening neurological genetic diseases. Our initial gene therapy for spinal muscular atrophy (SMA) has been approved in the U.S., Japan, EU and Brazil.
We are headquartered in Bannockburn, IL with another location in Deerfield, IL. Our manufacturing facilities are located in Libertyville, IL, Durham, NC and Longmont, CO. Our research site is located in San Diego, CA. The European headquarters is located in Zurich, Switzerland and Novartis Pharma K.K. (“Novartis Pharma”) is located in Tokyo, Japan.
Stock Symbol: NVS
Stock Exchange: NYSE
96 articles with Novartis Gene Therapies
1/5/2021The new year is here and the biotech job market is hot! A new year, a new you, a new job?
AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)
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.
4/15/2020The 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)
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).
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
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.
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
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.
Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma® the only gene therapy for patients with spinal muscular atrophy (SMA)
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.
10/14/2019It 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.
9/23/2019Last 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.
While the approval of AveXis’ gene therapy for spinal muscular atrophy has been tainted by the revelation of early data manipulation, the Novartis subsidiary presented some long-term data at a conference that backs up the treatment’s efficacy.
AveXis presents new data at EPNS continuing to show significant therapeutic benefit of Zolgensma® in prolonging event-free survival now up to 5 years of age in patients with spinal muscular atrophy (SMA) Type 1
AveXis announced that new interim data from the Phase 3 SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase 3 STR1VE clinical program for Zolgensma®
AveXis presented new interim data from the Phase III SPR1NT trial of Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1.
The Midwestern region of the United States is often referred to as “flyover country” by residents of both coasts as a way to diminish or dismiss the importance of the states that make up this part of the nation. But the Midwest can be a great place to live and raise a family.
Some have wondered why the agency has hit back at Novartis so hard when the data in question didn’t have any effect on the eventual outcomes of the product’s effectiveness and safety.
In a brief announcement, AveXis said the Kaspar brothers have not been involved in any operations at AveXis since early May, seemingly ahead of the U.S. Food and Drug Administration’s approval of Zolgensma.
The Democratic senators called it "unconscionable" that AveXis and its parent company Novartis would include manipulated data in order to rush a product to market.
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Catalent Biologics Announces Agreement to Provide Additional Production of Approved Gene Therapy Treatment for Spinal Muscular Atrophy
Catalent Biologics today announced a long-term strategic agreement for the development and manufacturing of Zolgensma® (onasemnogene abeparvovec-xioi), an AveXis gene therapy treatment for spinal muscular atrophy (SMA).