AveXis, a Novartis company, is the world’s leading gene therapy company, redefining the possibilities for patients and families affected by life-threatening genetic diseases through our innovative gene therapy platform.
Founded in 2013 and headquartered in Bannockburn, IL, the goal of AveXis’ cutting-edge science is to address the underlying, genetic root cause of diseases. AveXis pioneered foundational research, establishing AAV9 as an ideal vector for gene transfer in diseases affecting the central nervous system, laying the groundwork to build a best-in-class, transformational gene therapy pipeline.
AveXis received its first U.S. Food and Drug Administration approval in May 2019 for the treatment of spinal muscular atrophy (SMA). AveXis is also developing therapies for other genetic diseases, including Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) SOD1 and Friedreich’s ataxia
We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and other severe genetic diseases.
We are advancing a focused pipeline of innovative gene therapies. We are currently conducting a pivotal phase 3 trial in our lead indication, hemophilia B, and have initiated a phase 1/2 trial in Huntington’s disease. Our pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities.
Voyager Therapeutics, Inc develops gene therapies for people living with severe neurological diseases. These include Parkinson’s disease, Huntington’s disease, amyotrophic lateral sclerosis (ALS), Friedrich’s ataxia, Alzheimer’s disease and other genetically defined neurological disorders. Voyager is a leader in adeno-associated virus (AAV) vector engineering, manufacturing and delivery to the nervous system. Our work begins with a commitment to patients, their families and care providers. We apply our expertise in research, development and manufacturing in unlocking the potential of gene therapies to advance the treatment options for fatal or life-limiting neurological disorders. Founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience, Voyager is headquartered in Cambridge, Massachusetts. For more information on Voyager, please visit the Company’s website at www.voyagertherapeutics.com.