AVROBIO Inc. (NASDAQ: AVRO) stock gained by at last close while the AVRO stock price surged by in the after-hours trading session. The mission is to make individualized gene therapy a reality for everyone on the planet. With a standard dose of gene therapy engineered to stimulate permanent expression of therapeutic protein even in tough tissues and organs like the brain, muscle, and bone, they hope to prevent, stop, or cure disease throughout the body.
AVRO stock’ Update
At the 14th International Congress of Inborn Errors of Metabolism (ICIEM), which happened on Nov. 21-23, 2021, in Sydney, Australia, AVROBIO organized a panel of experts managing the patient outcomes in Phase 2 FAB-GT clinical study of AVR-RD-01, innovative one-time gene therapy for Fabry disease. Revised study results from the first lentiviral gene therapy clinical studies for Fabry disease and Gaucher disease type 1, as well as 10- to 12-month safety data from gene therapy-treated animals with mucopolysaccharidosis type II (MPSII), or Hunter syndrome, were among the other topics discussed.
The clinicians talked about the patient outcomes during apheresis (the procedure of collecting the participant’s stem cells), conditioning (the process of clearing space in the patient’s bone marrow to obtain their stem cells back following the introduction of the therapeutic transgene), infusion of the genetically modified stem cells, and after getting the gene therapy.
Dr. Nicholls commented that,
Being a member of this first-in-the-world medical trial to introduce this investigational gene therapy to the Fabry disease community in Australia has been thrilling and fulfilling. While the current therapy has made a significant impact in patients’ lives, there are restrictions to the influence of enzyme replacement therapy (ERT) on Fabry disease symptoms in the heart, kidneys, and central nervous system (CNS). They know, for example, that even with a combination of ERT and other renal preventive therapies, renal deterioration will occur. Of course, they hope this isn’t the case. For these individuals and their families, better therapeutic alternatives are required.