Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform ...

As gene therapy evolves to target additional indications—including more common indications—the choice of delivery platform is bound to become more interesting. At present, the most common delivery ...

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...

Out of the many challenges scientists face in bringing genomic medicine to patients, delivery is near the top of the list. Due to these barriers in getting the genetic payload to the correct target ...

Adeno-associated virus (AAV) vectors have become widely recognized as a fundamental tool in gene therapy, primarily due to their potential as a delivery system for the treatment of genetic disorders.

What Are AAV Vector-Based Therapies? Gene therapies are groundbreaking approaches utilizing viral and non-viral vectors. These vectors introduce therapeutic genes into patient cells to treat diseases, ...

Adeno-associated virus (AAV)–mediated gene therapy has emerged as a promising treatment for hemophilia B. Data on safety and durability from 13 years of follow-up in a cohort of patients who had been ...

SGT-003 gene therapy showed significant microdystrophin expression and muscle integrity improvements in DMD patients, with no serious adverse events reported. The novel AAV vector demonstrated ...

At a median 13 years after a single infusion of AAV-mediated gene therapy, factor IX expression remained stable in 10 patients with severe hemophilia B. The median annualized bleeding rate decreased ...

Dublin, Sept. 29, 2025 (GLOBE NEWSWIRE) -- The "Adeno-Associated Viral (AAV) Vector Market Industry Trends and Global Forecasts to 2035: Distribution by Type of Therapy, Therapeutic Area, Route of ...

HOUSTON, May 12, 2025 /PRNewswire/ -- PackGene Biotech, Inc. announced today six presentations at the American Society of Gene & Cell Therapy (ASGCT) 28 th annual meeting in New Orleans, May 13-17, ...