AveXis

Jaguar Gene Therapy Launches with Mission to Accelerate Breakthroughs in Gene Therapy for Patients Suffering from Severe Genetic Diseases

Retrieved on: 
Thursday, February 25, 2021

Jaguar Gene Therapy launched today after being in stealth mode for the past year and is uniquely positioned to accelerate gene therapy development.

Key Points: 
  • Jaguar Gene Therapy launched today after being in stealth mode for the past year and is uniquely positioned to accelerate gene therapy development.
  • Led by the former AveXis leadership and resourced by Deerfield Management , Jaguar leverages a proven management team with the expertise to accelerate the development, manufacturing and commercialization of novel gene therapy treatments for patients suffering from severe genetic diseases.
  • Jaguar is uniquely positioned to accelerate breakthroughs in gene therapy for patients suffering from severe genetic diseases, said Sean Nolan, Executive Chairman of Jaguar Gene Therapy.
  • Jaguar Gene Therapy, LLC is accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases.

REGENXBIO Announces Agreement to Monetize Portion of Zolgensma® Royalties for $200 Million

Retrieved on: 
Tuesday, December 22, 2020

If either cap is met, the royalty rights would revert to REGENXBIO.

Key Points: 
  • If either cap is met, the royalty rights would revert to REGENXBIO.
  • Zolgensma is currently approved for the treatment of Spinal Muscular Atrophy (SMA) in the United States, Japan, Europe, Brazil and Canada.
  • Morgan Stanley & Co. LLC served as sole structuring agent and Covington & Burling LLP served as counsel to REGENXBIO.
  • REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.

Health Canada approves Zolgensma®, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA)1

Retrieved on: 
Wednesday, December 16, 2020

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3.

Key Points: 
  • In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3.
  • Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1.
  • Advances and challenges in developing a therapy for spinal muscular atrophy.
  • Time Is Motor Neuron: Therapeutic Window and Its Correlation with Pathogenetic Mechanisms in Spinal Muscular Atrophy.

BioLife Solutions Awarded Three New Patents for Cell and Gene Therapy Bioproduction Tools

Retrieved on: 
Monday, March 2, 2020

BOTHELL, Wash., March 2, 2020 /PRNewswire/ -- BioLife Solutions , Inc. (NASDAQ: BLFS), a leading developer and supplier of best-in-class bioproduction tools for cell and gene therapies, today announced three new patents for bioproduction tools related to the 2019 acquisitions of Astero Bio and SAVSU Technologies.

Key Points: 
  • BOTHELL, Wash., March 2, 2020 /PRNewswire/ -- BioLife Solutions , Inc. (NASDAQ: BLFS), a leading developer and supplier of best-in-class bioproduction tools for cell and gene therapies, today announced three new patents for bioproduction tools related to the 2019 acquisitions of Astero Bio and SAVSU Technologies.
  • The new patents awarded to BioLife include:
    This patent covers the key technologies used in the -80C dry ice evo smart shipping container, currently in use by Novartis to transport the AveXis ZOLGENSMA gene therapy.
  • We look forward to providing a continuous stream of innovative solutions to the high growth cell and gene therapy market."
  • BioLife Solutions is a leading supplier of cell and gene therapy bioproduction tools.

Adverum Biotechnologies Appoints Angela Thedinga as Chief Technology Officer

Retrieved on: 
Tuesday, February 25, 2020

REDWOOD CITY, Calif., Feb. 25, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that Angela Thedinga has been promoted to chief technology officer.

Key Points: 
  • REDWOOD CITY, Calif., Feb. 25, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that Angela Thedinga has been promoted to chief technology officer.
  • I am pleased to congratulate Angela on her role as CTO, who since joining the company has demonstrated executive leadership across the organization and strategic insight, said Leone Patterson, president and chief executive officer of Adverum Biotechnologies.
  • Before joining Adverum, Ms. Thedinga served as vice president of program management and chief of staff at AveXis, Inc., a gene therapy company acquired by Novartis in 2018.
  • Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases.

Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028

Retrieved on: 
Monday, September 30, 2019

Pipeline products with the potential to help address the root cause of Spinal Muscular Atrophy (SMA) by producing the missing protein in Spinal Muscular Atrophy (SMA) patients are the ones that have been met with enthusiasm among key opinion leaders (KOLs).

Key Points: 
  • Pipeline products with the potential to help address the root cause of Spinal Muscular Atrophy (SMA) by producing the missing protein in Spinal Muscular Atrophy (SMA) patients are the ones that have been met with enthusiasm among key opinion leaders (KOLs).
  • Therapies that are being developed to help improve muscle functions in Spinal Muscular Atrophy (SMA) patients also are expected to play a pivotal role in patient care.
  • Until Zolgensma's recent FDA approval, Biogen's Spinraza was the only approved therapy for Spinal Muscular Atrophy (SMA) patients.While Spinraza has definitely reaped the benefits of first-to-market therapy for Spinal Muscular Atrophy (SMA), its need for a chronic dosage coupled with an intrathecal route of administration represents a logistical challenge for patients.
  • The latest report, "Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028" provides an overview of Spinal Muscular Atrophy (SMA), including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.

MDA Celebrates FDA Approval of Zolgensma for Treatment of Spinal Muscular Atrophy in Pediatric Patients

Retrieved on: 
Friday, May 24, 2019

Zolgensma is a one-time intravenous (into the vein)infusion for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survivalmotor neuron 1 (SMN1) gene, including those who are pre-symptomatic at diagnosis.

Key Points: 
  • Zolgensma is a one-time intravenous (into the vein)infusion for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survivalmotor neuron 1 (SMN1) gene, including those who are pre-symptomatic at diagnosis.
  • Zolgensma is only the second gene therapy approved by the FDA to treat any disease, placing the field of neuromuscular disease at the forefront of genetic medicine.
  • "Zolgensma is poised to be another life-altering therapy for the SMA community," says MDA President and CEO Lynn O'Connor Vos.
  • AveXis is currently testing Zolgensma in clinical trials for patients with SMA type 2 as well as in pre-symptomatic SMA patients.

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

Retrieved on: 
Friday, May 24, 2019

Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.

Key Points: 
  • Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.
  • We now have data four years out from the trial, and we see the durability of this gene therapy."
  • Zolgensma will be made available in the US and will be marketed by AveXis, a Novartis company.
  • This includes answering questions about Zolgensma, verifying reimbursement assistance and coordinating financial assistance programs for eligible patients.

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

Retrieved on: 
Monday, April 1, 2019

The addition of the six-building Longmont campus consists of nearly 700,000 square-feet of space for biologic drug manufacturing, offices, laboratories, warehousing and utilities.

Key Points: 
  • The addition of the six-building Longmont campus consists of nearly 700,000 square-feet of space for biologic drug manufacturing, offices, laboratories, warehousing and utilities.
  • Initial start-up activities in Longmont will include preparing the facility for scaling, manufacturing and testing of gene therapies and hiring staff.
  • We are eager to add the talented team in Longmont to AveXis, and we hope that they will choose to join us as we build world-leading manufacturing capabilities in gene therapy."
  • Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) is an investigational gene therapy currently in development as a one-time infusion for SMA Type 1.

Neurogene Appoints Industry Leaders Dr. Sukumar Nagendran and Dr. Serge Stankovic to Board of Directors

Retrieved on: 
Wednesday, February 27, 2019

Neurogene, Inc ., a company founded with a mission to bring life-changing medicines to patients and families affected by rare neurological diseases, today announced the appointment of two biotech industry leaders to its board of directors: Dr. Sukumar Nagendran and Dr. Srdjan (Serge) Stankovic.

Key Points: 
  • Neurogene, Inc ., a company founded with a mission to bring life-changing medicines to patients and families affected by rare neurological diseases, today announced the appointment of two biotech industry leaders to its board of directors: Dr. Sukumar Nagendran and Dr. Srdjan (Serge) Stankovic.
  • Dr. Nagendran brings more than 30 years of experience as an accomplished physician, drug developer and biotech leader, including extensive work in gene therapy clinical development.
  • Prior to his time at AveXis, Dr. Nagendran held senior roles at Reata Pharmaceuticals, Daiichi Sankyo, Pfizer and Novartis in medical affairs and clinical development.
  • Prior to moving into the biotech industry, Dr. Nagendran practiced internal medicine, with a focus on diabetes and cardiovascular disease.