The gene therapy development company, AveXis, Incorporation, just announced that they will be expanding their clinical trial development program for the potential gene therapy treatment: AVXS-101, reported Globe News Wire.
Finding answers to those suffering with SMA has always been a focus for AveXis, and they hope with the program growth they will be able to achieve more with greater efficiency.
With so much potential stemming form AVXS-101, Chief Medical Officer Dr. Sukumar of AveXis is anticipating this year to make huge strides for patients and their families that face the struggles of SMA. Patients of SMA are debilitated by the disease, which causes them to lose their motor neurons, leading to muscle atrophy and even paralysis. The disease is caused by a defective SMN1 gene; this gene leads to the function of motor neurons. SMA affects about 1 in every 10,000 births and is known to be a leading genetic related cause to infant deaths. To read more about this rare disease, click here.
The potential new treatment, AVXS-101, has been designed to target the cause of the disease and prevent further muscular deterioration from occurring. It is also developed to address motor neurons specifically.
AveXis is a successful company known for its clinical trials for gene therapy development, specifically for rare diseases and neurological genetic disorders. While they have focused on SMA over the years, with their expansion they hope to find gene therapies for Rett syndrome, RTT, as well as amyotrophic lateral sclerosis (ALS), all diseases that are caused from mutated genes.
While there is promise for AVXS-101’s future for SMA patients, AveXis was sure to share that more needs to be done before assuring this treatment as successful. They will have to follow patients post trials to observe any potential side effects or consequences brought about from the therapy.
People from the SMA community should hold hope and push out positive vibes for the researchers at AveXis, but they should make sure to keep their excitement at bay as they look into the therapy deeper this year.
