John Carbona, CEO of AveXis, along with Dr. Brian Kaspar have been invited to present the company’s latest developments at the 26th Annual Piper Jaffray Healthcare Conference, which will take place on Tuesday, December 2. AveXis is a biopharmaceutical company currently developing gene therapy-based treatments for severe genetic and orphan diseases such as spinal muscular atrophy (SMA) in order to improve the lives of patients living with these debilitating and often fatal conditions.
This year, the Piper Jaffray Healthcare Conference will include more than 250 companies in the field of biotechnology, specialty pharmaceuticals, medical technology, medical diagnostics, life science tools, and healthcare services sectors. In addition, the conference will feature panel discussions with industry experts, physicians, and academicians regarding the complexity of regulatory, reimbursement, and legal issues, as well as discussions about the current state of the largest and most expensive diseases.
AveXis uses its clinical-stage gene therapy platform to focus on the creation of innovative treatments for SMA, an autosomal-recessive genetic disease that kills more children than any other genetic disease worldwide. The disease is caused by a genetic defect in the SMN1 gene, which is responsible for the survival of motor neurons as well as provoking the loss of motor neuron and progressive muscle weakness.
ChariSMA, AveXis’ lead therapeutic project, is currently being tested in collaboration with Nationwide Children’s Hospital in Columbus, Ohio, which launched patient enrollment last April in order to evaluate the safety and efficacy of the intravenous therapy. ChariSMA is an intravenous therapy that delivers a genetically-modified virus comprised of the Survival Motor Neuron replacement gene (SMN) through AveXis’s ReGenX Biosciences technology platform. The company expects to be able to improve both survival rate and motor function of the dosed SMA patients in the study with this new therapeutic approach. AveXis announced last week that it has finished the dosage of their phase 1 non-randomized clinical trial, which is the first human gene treatment in the world for pediatric SMA.
In addition, the U.S. Food and Drug Administration recently granted ChariSMA orphan drug designation, which allows for accelerated development of novel therapies for the treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States.