Press Releases & Media Statements

Date Title
Sep 02, 2020 AveXis renamed Novartis Gene Therapies, signifying the growing importance of gene therapy to Novartis corporate strategy.

AveXis renamed Novartis Gene Therapies, signifying the growing importance of gene therapy to Novartis corporate strategy.

Jun 01, 2020 AveXis Community Statement on the Global Managed Access Program for AVXS-101

In January we launched a global Managed Access Program (MAP) for Zolgensma® (onasemnogene abeparvovec). Under this program, we are making up to 100 doses available in 2020 to eligible patients with SMA under the age of two in countries where Zolgensma is not approved. Already, the program has enabled children across Asia, Australia, Europe and North America to receive treatment who would not have otherwise had access to the therapy.

May 19, 2020 AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)

Basel, May 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.

Apr 21, 2020 AveXis Community Statement on the Coronavirus Disease (COVID-19)

Like many organizations across the United States and around the world, AveXis is closely monitoring the coronavirus disease (COVID-19) and its impact on patients, the SMA community, healthcare providers, and our employees and partners. We know that life with a rare genetic disease can bring significant challenges, and we want to be as supportive as possible, particularly during this unprecedented time. We are dedicated to the SMA community, and our patients and their families are the motivation for everything we do.

Mar 27, 2020 AveXis receives positive CHMP opinion for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)

Basel, March 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) 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.

Mar 24, 2020 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

Basel, March 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.

Mar 24, 2020 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

Basel, March 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, achieving a remarkable mean increase from baseline of 6.0 points in the Hammersmith Functional Motor Scale-Expanded (HFMSE), twice the clinically meaningful threshold established in previous SMA studies and reflecting improvement in three to six skills.

Mar 24, 2020 Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma the only gene therapy for patients with spinal muscular atrophy (SMA)

Basel, March 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.

Dec 19, 2019 AveXis Community Statement on Global Managed Access Program for AVXS-101

December 19, 2019 Since its U.S. approval, AveXis has had increasing demand from families outside of the U.S. for access to AVXS-101.

Oct 30, 2019 Novartis announces AVXS-101 intrathecal study update

Basel, October 30, 2019 - Novartis today announced the United States Food & Drug Administration (FDA) placed a partial hold on clinical trials for intrathecal administration of AVXS-101.

Oct 05, 2019 AveXis presents updated STRONG data at WMS demonstrating a higher mean increase in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores among older SMA Type 2 patients following one-time intrathecal administration of AVXS-101

Basel, October 5, 2019 – AveXis, a Novartis company, today announced new interim data from the Phase 1/2 STRONG study for intrathecal (IT) administration of AVXS-101, demonstrating older patients (> 2 years and < 5 years) with spinal muscular atrophy

Sep 24, 2019 Novartis Statement on Form 483 posting by US Food and Drug Administration (FDA)

Today the FDA posted information provided by the company to the FDA in response to its Form 483 issued on August 2, 2019. Our submission, which can be read here at ( https://www.fda.gov/media/131007/download), reiterated our firm commitment to data

Sep 19, 2019 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

BASEL, Switzerland , Sept. 19, 2019 /PRNewswire/ -- New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age appropriate major milestone gain Updated results from global STR1VE study demonstrate that Zolgensma®

Aug 14, 2019 AveXis Statement on Changes to Senior Leadership Team

AveXis shares Page Bouchard, DVM named Senior Vice President of Research and Chief Scientific Officer, AveXis, effective August 5, 2019. Dr Bouchard is a 27 year industry veteran with experience in well over 100 Investigational New Drug programs and dozens of New Drug Application/Biologics License

Aug 06, 2019 Novartis stands behind Zolgensma® (onasemnogene abeparvovec- xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy

Basel, August 6, 2019 – Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma® (onasemnogene abeparvovec-xioi). First and foremost, we are fully confident in the safety, quality and efficacy of Zolgensma.

Jul 25, 2019 AveXis Statement on Access to Zolgensma® (onasemnogene abeparvovec-xioi)

The FDA's May 2019 approval of Zolgensma®, a gene therapy for spinal muscular atrophy (SMA) in pediatric patients less than 2 years of age, marked an important milestone within the SMA community. Zolgensma offers a new treatment option for children with SMA and their families.

May 24, 2019 AveXis Announces Innovative Zolgensma® Gene Therapy Access Programs for US Payers and Families

One-time treatment with Zolgensma (onasemnogene abeparvovec-xioi) is designed to replace lifetime of chronic therapy for all pediatric patients with SMA Annualized cost of Zolgensma is USD 425,000 per year for 5 years: 50% less than multiple established value-based pricing benchmarks including

May 24, 2019 AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA)

SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2 [1],[2] Zolgensma (onasemnogene abeparvovec-xioi) is approved for the treatment of pediatric patients less

May 07, 2019 New AveXis data at AAN showed long-term durability of Zolgensma® in patients with spinal muscular atrophy (SMA) Type 1

Interim long-term follow-up data showed all enrolled Cohort 2 patients maintained motor function and milestones achieved during the Phase 1 START trial Mean age of follow-up since dosing with Zolgensma ( onasemnogene abeparvovec-xioi; AVXS-101) was nearly four years, with some patients nearing

May 05, 2019 AveXis presented robust data at AAN demonstrating efficacy of Zolgensma® in broad spectrum of spinal muscular atrophy (SMA) patients

Interim data reported for the first time from STRONG in SMA Type 2 showed rapid motor function gains and milestone achievements with intrathecal Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) - New interim data from STR1VE in SMA Type 1 continued to show prolonged event-free survival,

Apr 16, 2019 AveXis data reinforce effectiveness of Zolgensma® in treating spinal muscular atrophy (SMA) Type 1

Ph 3 STR1VE data show prolonged event-free survival, early and rapid increases in CHOP-INTEND and significant milestone achievement in SMA Type 1, consistent with START trial First-in-human biodistribution data show transduction in intended CNS targets and widespread SMN expression comparable

Apr 01, 2019 AveXis expands world-leading gene therapy manufacturing capacity with purchase of advanced biologics therapy manufacturing campus in Longmont, Colorado

Facility to become the largest of four state-of-the-art sites involved in manufacturing of AveXis gene therapies for pipeline of rare genetic diseases including spinal muscular atrophy AveXis plans to offer positions to all approximately 150 employees previously employed at the site, and to

Dec 03, 2018 Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1

The AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx) 1 , filing is supported by data from the START trial which demonstrated a dramatic increase in survival and transformative improvement in achievement of developmental milestones compared to the natural history of SMA Type 1 2 SMA

May 03, 2018 AveXis Reports First Quarter 2018 Financial and Operating Results

Significant progress across operating functions including licensing, clinical trials and regulatory interactions -- -- AVXS-101 pre-BLA meeting with FDA is scheduled to be held in June 2018 -- CHICAGO , May 03, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc.

May 01, 2018 AveXis Provides Update on Proposed Acquisition by Novartis AG

CHICAGO , May 01, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the required waiting period under the Hart-Scott-Rodino

Apr 25, 2018 AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3

CHICAGO , April 25, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the first patient has been dosed in a Phase 3 trial

Apr 24, 2018 AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology

A mean increase in CHOP-INTEND of 17.3 was observed at three months post gene therapy in SMA Type 1 U.S. pivotal trial – – All patients in the therapeutic dose cohort in the Phase 1 trial were alive and event-free at 24-month follow-up – – Patients observed in long-term follow-up from the Phase 1

Apr 19, 2018 AveXis to Present AVXS-101 Data at the Annual Meeting of the American Academy of Neurology

Includes Initial Data from SMA Type 1 Pivotal Trial (STR1VE) and 24-Month Follow-Up Data from Phase 1 Trial CHICAGO , April 19, 2018 (GLOBE NEWSWIRE) -- AveXis , Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening

Apr 09, 2018 AveXis Enters Agreement to be Acquired by Novartis AG for $8.7 billion

Novartis to acquire AveXis for $218 per share in cash AveXis’ lead product candidate, AVXS-101, expected to enhance Novartis’s position as a gene therapy and neuroscience leader Transaction expected to be completed in mid-2018 CHICAGO , April 09, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc.

Mar 27, 2018 AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1

CHICAGO , March 27, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW)

Mar 13, 2018 AveXis Enters into Licensing Agreement with Genethon

Includes exclusive worldwide rights to AAV9-SMN product and route of administration CHICAGO and ÉVRY, France , March 13, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy

Feb 27, 2018 AveXis Reports Fourth Quarter and Full Year 2017 Financial and Operating Results

On track to request pre-BLA meeting with FDA in Q2 2018 – – Intends to initiate pivotal trial in SMA Type 1 in Europe and multi-national pre-symptomatic SMA study in the first half of 2018 – – Anticipates IND submissions for Rett syndrome and genetic ALS in late 2018/early 2019 – – Conference

Feb 20, 2018 AveXis to Report Fourth Quarter and Full Year 2017 Financial and Operating Results

Conference call and webcast on February 27 at 4:30 p.m. EST CHICAGO , Feb. 20, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report financial

Jan 30, 2018 AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients

CHICAGO , Jan. 30, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that, following review of safety data and early signals of

Jan 22, 2018 AveXis Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares

CHICAGO , Jan. 22, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the closing of its previously announced underwritten public offering of 4,509,840 shares of its common stock at a price to the public of $102.00 per share before underwriting discounts and commissions, including

Jan 16, 2018 AveXis Announces Pricing of Public Offering of Common Stock

CHICAGO , Jan. 16, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced that it intends to offer and sell, subject to market conditions, up to $400 million of shares of its common stock in an underwritten public offering. The offering is subject to market and other conditions, and

Jan 16, 2018 AveXis Announces Proposed Public Offering of Common Stock

CHICAGO , Jan. 16, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the pricing of an underwritten public offering of 3,921,600 shares of its common stock at a public offering price of $102.00 per share, before underwriting discounts and commissions.

Jan 16, 2018 AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

– Company to expand study of AVXS-101 into additional SMA populations including pre-symptomatic, older pediatric Type 2 and Type 3 SMA patients – – First patient dosed in Phase 1 trial of AVXS-101 in SMA Type 2 – CHICAGO , Jan. 16, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc.

Jan 08, 2018 REGENXBIO and AveXis Announce Expansion of Relationship through Amended License Agreement for the Development and Commercialization of Treatments for Spinal Muscular Atrophy

AveXis acquires exclusive rights to entire NAV Technology Platform for the development of treatments for SMA Amended agreement permits assignment by AveXis upon a change of control without REGENXBIO’s consent REGENXBIO could receive up to $260 million , including $140 million in guaranteed upfront

Jan 04, 2018 AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1

Company to submit information requested by FDA to the IND on an on-going basis – – AveXis plans to request a pre-BLA meeting in Q2 2018 – – Conference call and webcast today at 4:30 pm EST – CHICAGO , Jan. 04, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy

Dec 13, 2017 AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101

– The FDA notified AveXis it may initiate the Phase 1 trial in SMA Type 2 based on a review of data provided by the company; trial to commence immediately – – The trial will evaluate safety, dosing and proof of concept for efficacy – – The trial will use product produced

Nov 09, 2017 AveXis Reports Third Quarter 2017 Financial and Operating Results

– First patient has been dosed in pivotal trial of AVXS-101 for SMA Type 1 – – Conference call and webcast November 9 at 4:30 p.m. EST – CHICAGO , Nov. 09, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from

Nov 02, 2017 AveXis to Report Third Quarter 2017 Financial and Operating Results

Conference call and webcast on November 9 at 4:30 p.m. EST CHICAGO , Nov. 02, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report financial

Nov 01, 2017 AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1

– Patients who received a single dose of AVXS-101 resulted in longer survival, superior achievement of motor milestones, and better motor function than in historical cohorts – – As of August 7, 2017 , all patients are alive, event-free and have reached at least 20 months of age – – As of August 7,

Oct 03, 2017 AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society

– All patients were alive and event-free at 20 months of age as of the August 7, 2017 data cut-off – – Patients in Cohort 2 continue to demonstrate improvements in motor milestones – SAINT MALO, France , Oct. 03, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy

Sep 29, 2017 AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process

– FDA notified AveXis it may initiate pivotal trial based on a review of data provided by the company following CMC Type B meeting – – Product used in the pivotal trial will be from the AveXis GMP manufacturing process; pivotal trial to commence immediately – –

Aug 10, 2017 AveXis Reports Second Quarter 2017 Financial and Operating Results

– AveXis on track to submit potency assay data to FDA in August – – Conference call and webcast August 10 at 4:30 p.m. EDT – CHICAGO , Aug. 10, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and

Aug 03, 2017 AveXis to Report Second Quarter 2017 Financial and Operating Results

-- Conference call and webcast on August 10 at 4:30 p.m. EDT -- CHICAGO , Aug. 03, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report

Jun 26, 2017 AveXis Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares

CHICAGO , June 26, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the closing of its previously announced underwritten public offering of 4,111,250 shares of its common stock at a price to the public of $70.00 per share before underwriting discounts and commissions, including

Jun 20, 2017 AveXis Announces Pricing of Public Offering of Common Stock

CHICAGO , June 20, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the pricing of an underwritten public offering of 3,575,000 shares of its common stock. The net proceeds to AveXis from the offering, after deducting the underwriting discounts and commissions and estimated

Jun 19, 2017 AveXis Announces Proposed Public Offering of Common Stock

CHICAGO , June 19, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced that it intends to offer and sell, subject to market conditions, up to $200 million of shares of its common stock in an underwritten public offering. The offering is subject to market and other conditions, and

Jun 14, 2017 AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101

– Agency is supportive of proposed analytical methods and comparability protocol – – AveXis anticipates initiating U.S. SMA Type 1 and Type 2 trials in Q3 2017 with product from new process – – AveXis manufacturing facility is fully operational for GMP product production – – Conference call and

Jun 07, 2017 AveXis and REGENXBIO Announce New Exclusive Worldwide Licenses for the Treatment of Two Rare Neurological Monogenic Disorders Using NAV AAV9 Vector

REGENXBIO grants AveXis new licenses to NAV AAV9 vector for the development and commercialization of treatments for Rett syndrome (RTT) and amyotrophic lateral sclerosis (ALS) CHICAGO and ROCKVILLE, Md. , June 07, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) and REGENXBIO Inc.

May 11, 2017 AveXis Reports First Quarter 2017 Financial and Operating Results

– Conference call of April 25 was in lieu of Q1 2017 earnings call – CHICAGO , May 11, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported

May 04, 2017 AveXis to Report First Quarter 2017 Financial and Operating Results

Results to be reported on May 11, 2017 CHICAGO , May 04, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report financial and operating results

Apr 25, 2017 AveXis Presents Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology

– 10 of 12 patients on proposed therapeutic dose sat unassisted; patient videos demonstrate motor milestone achievement not seen in natural history of disease – – 86% of patients without feeding support and 70% of patients without respiratory support prior to dosing continue without any support –

Apr 18, 2017 AveXis to Host Webcast of Preclinical and Clinical Studies of AVXS-101 as Presented at the Annual Meeting of the American Academy of Neurology

– Includes Results from Phase 1 Trial of AVXS-101 in SMA Type 1 – – Conference call and webcast Tuesday, April 25 at 4:30 p.m. EDT – CHICAGO , April 18, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare

Mar 16, 2017 AveXis Reports Topline Results from Phase 1 Trial of AVXS-101 in SMA Type 1 and Fourth Quarter and Full Year 2016 Financial and Operating Results

– No new treatment-related safety or tolerability findings – – No new events reported and 15 of 15 patients event-free at 13.6 months of age; majority of patients receiving proposed therapeutic dose sit unassisted – – Conference call and webcast March 16 at 4:30 p.m.

Mar 09, 2017 AveXis to Report Topline Phase 1 Trial Results in SMA Type 1 and Fourth Quarter and Full Year 2016 Financial and Operating Results on March 16, 2017

-- Conference call and webcast on March 16 at 4:30 p.m. EDT -- CHICAGO , March 09, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce

Mar 02, 2017 AveXis to Present Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at AAN Annual Meeting

-- Key preclinical and clinical data of AVXS-101 in SMA to be presented -- CHICAGO , March 02, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today

Feb 15, 2017 AveXis Appoints Rick Modi as Senior Vice President and Chief Business Officer

CHICAGO , Feb. 15, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the appointment of Rick Modi to the executive management team as

Feb 06, 2017 AveXis Announces Single-Arm Design for European Pivotal Study of AVXS-101 in SMA Type 1 Patients

Company provides update following receipt of Scientific Advice response from the EMA CHICAGO , Feb. 06, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic

Jan 31, 2017 AveXis Gene Therapy AVXS-101 Granted Access into EMA PRIME Program for Spinal Muscular Atrophy Type 1

PRIME intended to optimize development and speed evaluation of AVXS-101 through enhanced interactions and early dialogue with EMA CHICAGO , Jan. 31, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and

Jan 03, 2017 AveXis to Present at the 35th Annual J.P. Morgan Healthcare Conference

CHICAGO , Jan. 03, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced Sean Nolan , President and Chief Executive Officer, will present a

Dec 21, 2016 AveXis to Use Intended Commercial GMP Product in SMA Type 2 Study

– SMA Type 2 study to initiate in Q2 2017 – – Streamlines development of Type 2 clinical data set – – Conference call and webcast December 21 , at 4:30 p.m. EST – CHICAGO , Dec. 21, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for

Nov 10, 2016 AveXis Reports Third Quarter 2016 Financial and Operating Results

– AVXS-101 demonstrated continued motor function improvement in ongoing study; majority of patients on proposed therapeutic dose achieved key developmental milestones – – Confirmed single-arm design for pivotal study of SMA Type 1 in U.S. – – Conference call of November 1, 2016 , was in lieu of Q3

Nov 08, 2016 AveXis to Present at Jefferies 2016 London Healthcare Conference

CHICAGO , Nov. 08, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases , today announced that Sean Nolan , president and chief executive officer, will

Nov 01, 2016 AveXis Announces Single-Arm Design for U.S. Pivotal Study of AVXS-101 in SMA Type 1 Patients

– Company provides update following receipt of FDA minutes from Type B meeting – – Conference call and webcast November 1 at 4:30 p.m. EDT – CHICAGO , Nov. 01, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering

Oct 31, 2016 AveXis to Report Third Quarter 2016 Financial and Operating Results

Conference call and webcast on November 10 at 4:30 p.m. EST CHICAGO , Oct. 31, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce

Oct 10, 2016 AveXis to Host Webcast Update of Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Data, Including Developmental Milestones, as of September 15, 2016 as presented by Jerry Mendell , M.D. at the International Annual Congress of the World Muscle Society -- -- Webcast Today, October 10, 2016 at 8:30 a.m. EDT – CHICAGO , Oct. 10, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc.

Oct 08, 2016 AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 as Presented at the International Annual Congress of the World Muscle Society

-- Majority of patients on the proposed therapeutic dose achieved key developmental milestones including sitting unassisted; two patients walking independently -- -- Conference call and webcast October 10 at 8:30 a.m. EDT -- CHICAGO --(BUSINESS WIRE)--Oct. 8, 2016-- AveXis, Inc.

Sep 08, 2016 AveXis Announces Pricing of Public Offering of Common Stock

CHICAGO , Sept. 08, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the pricing of an underwritten public offering of 4,250,000 shares of its common stock, 3,960,145 shares of which are being offered by AveXis and 289,855 shares of which are being offered by PBM Capital

Sep 06, 2016 AveXis Announces Proposed Public Offering of 4,000,000 Shares of Common Stock

CHICAGO , Sept. 06, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced that it intends to offer and sell, subject to market conditions, 3,566,474 shares of its common stock in an underwritten public offering. Additionally, PBM Capital Investments, LLC , an existing stockholder of

Aug 11, 2016 AveXis Reports Second Quarter 2016 Financial and Operating Results and Interim Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Interim data through July 1 showed no “events” -- -- Continued motor function improvement with 25% of patients in the proposed therapeutic dose group in the normal range -- -- FDA requested Type B Meeting to discuss SMA Type 1 clinical development pathway -- -- Conference call and webcast August

Aug 02, 2016 AveXis to Report Second Quarter 2016 Financial and Operating Results and Interim Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

Conference call and webcast on August 11 at 4:30 p.m. EDT CHICAGO , Aug. 02, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce financial

Jul 20, 2016 AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1

CHICAGO , July 20, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. , (Nasdaq:AVXS) a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration ( FDA ) has granted

Jul 11, 2016 AveXis Appoints Michael B. Johannesen as Senior Vice President, General Counsel and Chief Compliance Officer

CHICAGO , July 11, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (Nasdaq:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the appointment of Michael B.

Jun 06, 2016 AveXis to Present at Goldman Sachs Annual Global Healthcare Conference and Jefferies 2016 Healthcare Conference

CHICAGO , June 06, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that members of the management team will present at two

May 16, 2016 AveXis Presents Pulmonary Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Richard Shell, MD, Presented Data as of April 1, 2016 at the American Thoracic Society 2016 Conference -- CHICAGO , May 16, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing novel treatments for patients suffering from rare and life-threatening

May 12, 2016 AveXis Reports First Quarter 2016 Financial and Operating Results

Encouraging interim data for lead clinical program, AVXS-101, for Spinal Muscular Atrophy Type 1 Conference call May 12 at 4:30 p.m. EDT CHICAGO , May 12, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from

May 06, 2016 AveXis Reports Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Jerry Mendell , MD, Presented Data as of April 1, 2016 at the American Society of Gene & Cell Therapy 19 th Annual Meeting -- -- Company to Host Webcast Today at 4:30 p.m. Eastern Daylight Time -- CHICAGO , May 06, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc.

May 05, 2016 AveXis to Report First Quarter 2016 Financial Results on Thursday, May 12

CHICAGO , May 05, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce financial results for the first quarter ended March 31, 2016 on

Apr 25, 2016 AveXis to Report Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Jerry Mendell , M.D., to Present Data as of April 1, 2016 at the American Society of Gene & Cell Therapy 19 th Annual Meeting -- -- Company to Host Webcast on Friday, May 6, 2016 at 4:30 p.m. Eastern Daylight Time -- CHICAGO , April 25, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc.

Mar 16, 2016 AveXis Reports Fourth Quarter and Full Year 2015 Financial and Operating Results

-- Initial public offering raised $98.2 million in net proceeds -- -- Encouraging interim data for lead clinical program, AVXS-101, for Spinal Muscular Atrophy Type 1 – -- Conference call March 16 at 4:30 p.m. EDT -- CHICAGO , March 16, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc.

Mar 08, 2016 AveXis to Report Full Year and Fourth Quarter 2015 Financial Results on Wednesday, March 16

CHICAGO , March 08, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce financial results for the fourth quarter and full year ended

Mar 07, 2016 AveXis Announces Appointments of Daniel G. Welch, Terrence C. Kearney and Frank Verwiel, M.D. to Board of Directors

CHICAGO , March 07, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (Nasdaq:AVXS), a leading clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced three appointments to its Board of Directors: Daniel G.

Feb 10, 2016 AveXis Announces Pricing of Initial Public Offering

CHICAGO , Feb. 10, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (Nasdaq:AVXS), a clinical stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the pricing of its initial public offering of 4,750,000 shares of

Jan 04, 2016 AveXis Announces Completed Enrollment of Phase 1 Clinical Trial for AVXS-101 in Spinal Muscular Atrophy Type 1

Chicago, Ill. – January 4, 2016 – AveXis, Inc., a clinical-stage gene therapy company developing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced patient enrollment has completed for the Phase 1 clinical trial of AVXS-101 for the

Oct 05, 2015 Data from Ongoing Study of AVXS-101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress

Brighton, United Kingdom. – October 5, 2015 – AveXis, Inc., a clinical stage gene therapy company developing treatments for rare and life-threatening neurological genetic diseases, presented data from its ongoing Phase 1/2 trial of AVXS-101, the company’s initial product candidate for the treatment

Sep 16, 2015 AveXis Strengthens Executive Leadership Team with Key Appointments

– Thomas J. Dee, Senior Vice President, Chief Financial Officer – – James J. L’Italien, Ph.D., Senior Vice President, Chief Regulatory and Quality Officer – – Andrew Knudten, Senior Vice President, Manufacturing and Supply Chain – – Sukumar Nagendran, M.D., Senior Vice President and Chief Medical

Sep 08, 2015 AveXis Secures $65 Million in Financing

Company builds investor syndicate comprising leading mutual and biotech funds to advance its spinal muscular atrophy gene therapy program Chicago, Ill. – September 8, 2015 – AveXis, Inc., a leading gene therapy company developing treatments for rare and life-threatening neurological genetic

Jun 23, 2015 AveXis Gains Orphan Drug Designation from the European Medicines Agency for Gene Therapy Treatment in Spinal Muscular Atrophy

Dallas – June 23, 2015 – AveXis, Inc., a leading gene therapy company developing treatments for rare and life-threatening genetic diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug designation to its gene transfer therapeutic currently in clinical development

Jun 18, 2015 AveXis Appoints Sean P. Nolan as Chief Executive Officer

DALLAS, June 18, 2015 — AveXis, Inc., a leading gene therapy company developing treatments for rare and life-threatening genetic diseases, today announced the appointment of Sean P. Nolan as Chief Executive Officer. He will oversee all functions at AveXis, including clinical, regulatory, finance,

Apr 30, 2015 AveXis Announces Management Succession Plan

April 30, 2015, Dallas, Texas: AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases, today announced that John Carbona has resigned from his position as CEO. Mr. Carbona will remain a Director of AveXis.

Mar 19, 2015 AveXis EU Ltd. Granted SME Status by the European Medicines Agency

Dallas – March 19, 2015 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced that the European Medicines Agency (EMA) has granted AveXis’ wholly owned

Feb 25, 2015 AveXis Announces the Formation of its European Subsidiary, AveXis EU, Ltd.

Dallas – February 25, 2015 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced the formation of its EU-based wholly-owned subsidiary, AveXis EU, Ltd.

Jan 06, 2015 AveXis Closes its Series C Financing Round with Deerfield Management and Roche Venture Fund

Dallas – January 6, 2015 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced it has completed a $10 million financing round led by Deerfield Management