PackGene Biotech

June 25, 2026 — Sangamo Therapeutics has filed for Chapter 11 protection in the U.S. Bankruptcy Court for the District of Delaware, marking a major restructuring step for one of the longstanding pioneers in genomic medicine. The company reported $100 million to $500 million in both assets and liabilities and entered the process with two stalking horse asset purchase agreements already in place.

June 24, 2026 — REGENXBIO plans to file a Biologics License Application in the third quarter of 2026 for RGX-202, its investigational AAV8 gene therapy for Duchenne muscular dystrophy, under the FDA’s accelerated approval pathway. RGX-202 is designed to deliver a microdystrophin gene using an AAV8 vector, with the goal of partially restoring dystrophin function in muscle.

June 23 2026 — A Korean research consortium is aiming to begin clinical studies by 2029 for a domestically developed gene therapy for inherited retinal diseases, a group of rare genetic disorders that can cause progressive vision loss and blindness. The effort is part of the Gene and Cell Therapy Research Center project led by the Korea Research Institute of Bioscience and Biotechnology, or KRIBB, under the Korean government’s Global TOP Strategic Research Group initiative.

June 23026 — Serapha Bio has launched with $230 million in funding commitments to advance SERP-01, an investigational in vivo base editing therapy for alpha-1 antitrypsin deficiency, or AATD. The company is being formed through a reverse merger with Boundless Bio and will trade under the ticker AATD after the transaction closes.

June 22, 2026 — A research team led by the University of California, Riverside has developed an AAV9-based gene therapy that restored production of the missing brain protein FMRP, corrected abnormalities in brain circuitry, and improved behavior in a mouse model of Fragile X syndrome. The study was published in Molecular Therapy Nucleic Acids. Fragile X syndrome is the most common single-gene cause of autism spectrum disorder.

June 22 2026 — Genprex announced that the Canadian Intellectual Property Office has issued a Notice of Allowance for a patent covering the use of REQORSA® Gene Therapy, also known as quaratusugene ozeplasmid, in combination with PD-1 or PD-L1 antibodies for the treatment of both non-small cell lung cancer, or NSCLC, and small cell lung cancer, or SCLC. The newly allowed Canadian patent expands Genprex’s intellectual property portfolio for REQORSA-based immunotherapy combinations.

June 22, 2026 — Riaan Research Initiative announced that Riaan Singh Digeorge, a six-year-old child from New York City, has become the first patient to receive an experimental AAV9 gene therapy for Cockayne syndrome, a devastating ultra-rare genetic disorder associated with premature aging and early death. The treatment was administered at NewYork-Presbyterian Komansky Children’s Hospital on April 21, 2026, through a neurosurgical procedure performed by Dr. Mark Souweidane.

June 22, 2026 — CARsgen Therapeutics announced that China’s National Medical Products Administration has approved satricabtagene autoleucel, or satri-cel, for the treatment of patients with Claudin18.2-positive, HER2-negative advanced gastric or gastroesophageal junction adenocarcinoma who have failed at least two prior lines of therapy. The approval marks a major milestone for the cell therapy field.

June 22, 2026 — REGENXBIO announced that it has reached alignment with the U.S. FDA on next steps for potential accelerated approval of NAVSUNLI™, also known as clemidsogene lanparvovec-sngl or RGX-121, an investigational one-time gene therapy for Mucopolysaccharidosis II, or MPS II, also known as Hunter syndrome. The update follows REGENXBIO’s appeal of the FDA’s February 2026 Complete Response Letter for NAVSUNLI.

June 16, 2026 — Pooled data from two open-label dose-escalation studies suggest that AAVrh.10hFXN, an investigational AAV-based gene therapy encoding normal human frataxin, may improve key cardiac measures in patients with Friedreich’s ataxia cardiomyopathy. The results were published in JAMA Cardiology.