Get the latest articles from Pharmaceutical Online delivered to your inbox. The U.S. Food and Drug Administration, has granted AVXS-101 orphan drug and breakthrough therapy designations for all types of SMA The FDA also provide fast track designation for the SMA Type 1 indication. “Our primary focus is to bring gene therapies to patients suffering from devastating rare neurological diseases—such as SMA, genetic amyotrophic lateral sclerosis and Rett syndrome—and continued investment in establishing our manufacturing infrastructure is a critical component to accomplishing this goal,” Andrew Knudten, AveXis chief technical officer, said in a statement. “Our primary focus is to bring gene therapies to patients suffering from devastating rare neurological diseases – such as SMA, genetic amyotrophic lateral sclerosis, and Rett syndrome – and continued investment in establishing our manufacturing infrastructure is a critical component to accomplishing this goal,” he said. All rights reserved. Catalent said the additional capacity, along with access to its adeno-associated virus (AAV) gene therapy development, manufacturing, and process characterization expertise, will enable AveXis to ensure a stable and consistent supply of Zolgensma as AveXis prepares to launch the gene therapy. Learn how your comment data is processed. Samulski joined Pfizer in Sanford, N.C., as vice president of gene therapy after its purchase of Bamboo Therapeutics but is transitioning back to UNC and to AskBio as the company’s scientific founder and chief science officer, Mikhail said. AveXis will use the new plant to make its first product candidate, AVXS-101, a gene therapy to treat three types of spinal muscular atrophy (SMA). Catalent and AveXis have signed a long-term strategic agreement whose value was not disclosed. Also, the additional manufacturing staff will provide AveXis the “flexibility to enter into multiple external partnerships as the development and manufacturing partner of choice in gene therapy,” Lennon said. AveXis, the gene therapy biotech Novartis snapped up for $8.7 billion, will build a new manufacturing plant in North Carolina to produce its specialized therapies. JDIG projects result in positive net tax revenue to the state treasury, even after taking into consideration the grant’s reimbursement payments to a given company. RELATED: Manufacturing costs loom large for personalized CAR-T cancer meds and their need for speed. The one-time treatment, which is priced at $475,000, is made from a patient’s own T cells, which are collected and then re-engineered at specialized manufacturing facilities, and then infused back into the patient to attack cancer. Paragon Gene Therapy will also provide process development for clinical supply of additional viral therapies in AveXis pipeline, Catalent added. Novartis Gene Therapies is pursuing registration in close to AveXis, Inc. Roy Cooper. AveXis purchased rights to its gene therapy technology in 2015 for an undisclosed sum from Asklepios BioPharmaceutical, a Chapel Hill gene therapy platform company commonly called AskBio. All rights reserved. The Utility Account helps rural communities finance necessary infrastructure upgrades to attract future business. Until now, AveXis had addressed manufacturing of the gene therapy by lining up four U.S. production sites, including AstraZeneca’s former advanced biologics therapy manufacturing campus in Longmont, CO, which AveXis agreed to acquire in April. with patients in Initial start-up activities in Longmont will include preparing the facility for scaling, manufacturing … Novartis has a history of pharmaceutical manufacturing in North Carolina. "The new facility also will give the company complete control over manufacturing, he added." Life science professionals at NCBiotech also provided technical due diligence and other support for the project. In addition to the N.C. site, which is expanding, and the planned Colorado site, AveXis also has manufacturing facilities in Illinois and San Diego. While most public attention on Zolgensma has focused on its list price of $2.1 million—Novartis has said it is developing discounted patient-access programs with insurers—AveXis has sought to ensure it can manufacture the gene therapy. © 2020 Questex LLC. In 2006 the company’s generic drug unit, Sandoz, took over operation of a manufacturing facility built in 1997 by Novopharm in Wilson. “This collaboration allows us to leverage our new state-of-art GMP commercial manufacturing facility and AAV development and scale-up expertise to support AveXis from the early development stage to this critical launch,” added Pete Buzy, Catalent’s president, gene therapy. Write CSS OR LESS and hit save. Bamboo was sold to Pfizer in 2016 for $150M in a deal that could be worth as much as $645M if certain milestones are met. “The transformation of gene-based technologies into economic impact in North Carolina continues to be a focus of the Biotechnology Center, not only for human health but also for animal health and agriculture,” noted Bill Bullock, senior vice president of statewide operations & economic development with the North Carolina Biotechnology Center.
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