A Review of AAV Gene Therapy
To date, seven AAV gene therapies have received global market approval, as summarized below
Review: Highlighting Five Critical FDA-approved Therapies of 2023
Recent reports indicate that CBER granted approval to nine groundbreaking therapies within the first half of the year. This number surpasses the total approvals for any full year over the past two decades. Notable upcoming approvals for the latter half of 2023 include Iovance's Lifileucel, BrainStorm Cell Therapeutics' NurOwn, and a duo of gene therapies targeting sickle cell disease.
Breaking news! The first DMD gene therapy, SRP-9001, has been approved for marketing and is priced at $3.2 million
On June 22nd, the US Food and Drug Administration (FDA) approved the world's first one-time gene therapy SRP-9001 (Elevidys) to treat Duchenne muscular dystrophy (DMD), which is applicable to patients aged 4-5 who can walk independently.
Countdown | Global Progress in DMD Gene Therapy Drug Launches and Research
Duchenne muscular dystrophy (DMD) is a rare, fatal genetic disease caused by a mutation in the gene that encodes for dystrophin, a protein that protects muscles from damage. This results in a loss or functional deficiency of dystrophin, leading to a series of muscle disorders.
Good News: Weiguang Gene and Decode Genetics have reached a strategic cooperation to jointly promote gene therapy drug R&D
Recently, Weiguang Gene (Suzhou) Co., Ltd. (Weiguang Gene), an alliance member, and Decode Genetics Technology Co., Ltd. (Decode Genetics), a subsidiary of Sanyou Group, have reached an agreement to establish a strategic partnership and jointly develop new gene therapy drugs.