3+ day, 4+ hour ago   (282+ words) Sandhoff disease, like Tay-Sachs disease, is a fatal neurodegenerative lysosomal storage disease caused by the absence of ß-hexosaminidase (Hex) which leads to the accumulation of GM2 ganglioside in lysosomes. The disease, for which there are no available therapies, results in the…...

4+ day, 10+ hour ago   (475+ words) Genetic Engineering and Biotechnology News Biotech company Moonwalk Biosciences announced that it has raised $70 million from an oversubscribed series B financing round. The company plans to use the funds to continue developing its adipose-targeted RNAi therapies for obesity and cardiometabolic…...

1+ week, 8+ hour ago   (259+ words) NewBiologix will use its Xcell™ stable manufacturing platform to generate and characterize a research cell bank for Synastra’s DMD gene therapy candidate. The agreement also provides an option to transition the program to a commercial license supporting future clinical and…...

1+ week, 2+ day ago   (338+ words) This eBook brings together expert perspectives on several of the most pressing analytical topics facing today’s cell and gene therapy developers. The post The Analytical Blueprint forCell & Gene Therapy Success appeared first on GEN - Genetic Engineering and Biotechnology News. Cell…...

1+ week, 3+ day ago   (464+ words) By David H. Crean, PhD That is the environment biotech executives are navigating right now. The strategic responses being formulated quietly, deliberately, at the level of pipeline sequencing, capital allocation, and partnership architecture will shape the competitive landscape of biopharma for…...

1+ week, 4+ day ago   (301+ words) Under the agreement, Andelyn will transition Genprex's adeno-associated virus (AAV)-based diabetes gene therapy candidate with process optimization, scale-up, and IND-enabling and clinical-stage manufacturing to support clinical trials. The post Andelyn to Support Manufacturing Scaleup for Genprex’s Diabetes Gene Therapy…...

1+ week, 6+ day ago   (632+ words) Investors appear to question whether Rasonque™ is too expensive to achieve the millions of short-term dollars, and billions of longer-term dollars, in sales projected by analysts Revolution Medicines (Nasdaq: RVMD) appeared to have the makings of another strong stock surge…...

2+ week, 3+ day ago   (337+ words) The biomanufacturing workforce of the future is hampered by a lack of familiarity with the technology today. Consequently, when those students choose careers, biotech isn’t top of mind and, even when it is, they may not have developed the deep…...

3+ week, 2+ day ago   (257+ words) Researchers from the International Institute of Molecular and Cell Biology in Warsaw (IIMCB) say they have created the first systematic catalog of the substrate receptors of cullin–RING ligases and analyzed how their genetic variants may translate into disease symptoms....

3+ week, 3+ day ago   (588+ words) By Gareth John Macdonald Inefficiencies that limit the global supply of viral vectors are negatively impacting the gene therapy industry, according to researchers, who say there is an urgent need for better downstream purification materials. The call came from Stefano…...