News
Malaysian scientist Swee Lay Thein shares US$3m Breakthrough Prize for pioneering gene-editing research
9+ hour, 16+ min ago (254+ words) LOS ANGELES, April 19 " Malaysian physician-scientist Dr Swee Lay Thein and American researcher Dr Stuart H. Orkin have won the US$3 million (RM11. 86 million) Breakthrough Prize in Life Sciences for research that helped create the first approved CRISPR gene-editing therapy. According to a…...
Storm raises $56 M series C for phase II RNA inhibitor trial in sarcoma
3+ day, 17+ hour ago (56+ words) RNA epigenetics specialist Storm Therapeutics Ltd. has closed a $56 million series C to fund phase II development of its lead RNA enzyme inhibitor, STC-15, in treating myo- and liposarcomas, and announced that the first patient has been treated in the…...
$3 million prize goes to duo whose research led to first sickle cell CRISPR therapy
3+ hour, 53+ min ago (802+ words) Two scientists whose work ushered in the first approved therapy using the gene-editing tool CRISPR have won the $3 million Breakthrough Prize in Life Sciences. The prize winners " Dr. Swee Lay Thein, of the National Heart, Lung and Blood Institute (NHLBI),…...
Duality of Gene Splicing
8+ hour, 40+ min ago (599+ words) Samuel Stanbro (Fall 2024) For several decades, there has been ongoing research and development in the field of gene splicing, sometimes also referred to as gene therapy " Duality of Gene Splicing Samuel Stanbro (Fall 2024) For several decades, there has been ongoing…...
Philly scientists win 2026 Breakthrough Prize for developing gene therapy for blindness
18+ hour, 41+ min ago (1356+ words) Luxturna was the first FDA-approved gene therapy for a disease caused by mutations in a specific gene. It helped establish Philadelphia as a biotech hub. Three Philadelphia scientists won a $3 million Breakthrough Prize for developing the first FDA-approved gene therapy…...
Magnetic muon measurements and gene-therapy advances win US$3 million Breakthrough prizes
1+ day, 6+ hour ago (728+ words) David Hertzog and Katherine High were among the winners of this year's Breakthrough prize. Credit: Dennis R. Wise & Colin Lenton Last year, the particle-physics and accelerator laboratory Fermilab announced the final results of its measurements of the muon's magnetic moment, which…...
CRISPR Gene Editing: Breakthrough Hope for Down Syndrome | Science-Environment
20+ hour, 29+ min ago (602+ words) In a groundbreaking development researchers have engineered a modified version of the CRISPR gene-editing tool This innovation shows potential to silence the extra chromosome responsible for Down syndrome as indicated by early laboratory experiments Down syndrome is a genetic condition…...
Medicine's next leap: Delivering gene therapies exactly where they're needed
2+ day, 58+ min ago (112+ words) "It's kind of like we just discovered that cells use a new media like phones or Tik Tok for communication and not only face-to-face conversation," says'Dr. Derrick Gibbings, Professor and Associate Vice-President, Research Support and Infrastructure at the University of…...
Jason S. Tedrow adapts new technologies to scale up drug manufacturing
1+ week, 2+ day ago (493+ words) As Sanofi's head of process chemistry, he mixes traditional chemistry with flow chemistry and new biocatalysts to reduce costs and improve efficiency by Neil Savage, special to C&EN A version of this story appeared in Volume 100, Issue 12 A yearly…...
Expert: FDA, industry still wary of enrolling patients in early cell and gene therapy trials
1+ week, 2+ day ago (946+ words) A cutting-edge reference for mastering regulatory intelligence in the age of AI. This 28-chapter guide combines expert insights with real-world stories and practical frameworks. The new member-exclusive RAPS Journal of Regulatory Affairs, a bimonthly, peer-reviewed publication building on the success…...