PackGene Biotech

Sep 8, 2026 — A multidisciplinary consortium led by investigators at the Mass General Brigham Gene and Cell Therapy Institute and Massachusetts General Hospital has been awarded up to $39.2 million by the Advanced Research Projects Agency for Health (ARPA-H) to advance on-demand, distributed manufacturing of genetic medicines. It is one of five teams selected under ARPA-H’s Genetic Medicines and Individualized Manufacturing for Everyone (GIVE) program.

Sep 8, 2026 — Bristol Myers Squibb has reported positive Phase 2 results for arlo-cel, an investigational GPRC5D-directed chimeric antigen receptor (CAR) T-cell therapy, in patients with relapsed or refractory multiple myeloma. According to the company, the therapy improved response rates in heavily pretreated patients who had received at least four prior lines of therapy.

Sep 8, 2026 — PeproMene Bio has announced the publication in The Lancet of Phase 1 results for PMB-CT01, a first-in-class BAFF-R-targeted chimeric antigen receptor (CAR) T-cell therapy, in patients with relapsed or refractory B-cell non-Hodgkin lymphoma. Seven of nine treated patients, or approximately 78%, achieved a complete response, including four of six who had progressed after conventional CD19-directed CAR T-cell therapy (“CAR after CAR”).

Sep 8, 2026 — BrainChild Bio, a cell therapy developer focused on CAR T-cell treatments for children with rare brain cancers, has raised $116 million in Series A financing. Spun out of Seattle Children’s Hospital, the company is developing CAR T therapies for direct administration into the central nervous system, an approach that remains relatively uncommon in brain cancer.

Sep 8, 2026 — A clinical study published in Cell Stem Cell reports that a base-editing therapy achieved durable clinical remission in patients with sickle cell disease (SCD) and beta-thalassemia from a range of genetic backgrounds. The work, from CorrectSequence Therapeutics and collaborators, evaluated CS-101/CS-206, therapies developed with the company’s transformer Base Editor (tBE). Base editing enables precise, single-letter changes to DNA without creating double-strand breaks.

Sep 7, 2026 — Rznomics, a South Korea-based developer of RNA-based gene therapies, has reported a breakthrough in the high-efficiency production of circular RNA (circRNA), published in Nucleic Acids Research on September 4, 2026. The study reports that optimized target-site selection and P1 engineering improved self-circularization efficiency by up to approximately sevenfold versus the original STS design.

Sep 4, 2026 — Ionis Pharmaceuticals has received U.S. Food and Drug Administration (FDA) approval for zilganersen, to be marketed as Zanvastro, for Alexander disease, a rare neurodegenerative disorder — the first disease-modifying therapy approved in this indication. Zanvastro is an antisense oligonucleotide designed to reduce production of glial fibrillary acidic protein (GFAP), whose abnormal accumulation in astrocytes drives Alexander disease.

Sep 3, 2026 — Typewriter Therapeutics has emerged from stealth with $56 million in Series A financing to advance its non-viral, in vivo gene therapy platform toward the clinic. The round was led by RA Capital Management and AN Venture Partners. The company’s platform uses target-primed reverse transcription (TPRT) to insert therapeutic genetic sequences directly in vivo without viral vectors.

Sep 2, 2026 — uniQure has submitted an application to the U.S. Food and Drug Administration (FDA) for AMT-130, its investigational AAV gene therapy for Huntington’s disease, in what could become the first approved treatment targeting the underlying cause of the disease. The company has requested priority review and has also completed a submission to U.K. regulators.

Aug 25, 2026 — Researchers at Boston Children’s Hospital have reported a first-in-human pilot trial of a new approach that streamlines stem cell collection for sickle cell disease gene therapy, potentially removing a major barrier to treatment. Autologous gene therapy for sickle cell disease requires collecting large numbers of a patient’s hematopoietic stem cells — a lengthy process that can involve multiple hospital visits and deter patients from pursuing treatment.