The Director of the San Raffaele-Telethon Institute for Gene Therapy Luigi Naldini reflects on what a recent Nature study reveals and about the future of in vivo gene transfer “Our study provides an ...
A mouse study shows CAR-T cells can be engineered in vivo with precise gene editing, a potential breakthrough that could cut ...
Kelonia Therapeutics, Inc., a clinical-stage biotechnology company pioneering in vivo gene delivery, today shared first-in-human data from the ongoing inMMyCAR(TM) study, a Phase 1 clinical trial ...
The researchers say that, to their knowledge, this is the first demonstration of programmable, site-specific integration of a ...
A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has identified a unique window shortly after birth in which circulating blood stem cells can ...
Gene therapy using lentiviral vectors (LVs) has proven effective in ex vivo applications, where a patient’s stem cells are extracted, genetically modified, and then reinfused to achieve therapeutic ...
SynGenSys, a biotechnology company designing synthetic gene promoter systems to address critical bottlenecks in biopharma manufacturing and enhance cell and gene therapy development, today announced ...
CTNS-RD-04, an ex vivo gene therapy for cystinosis, showed reduced white-cell cystine levels and an acceptable safety profile ...
Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently ...
Recordati Joins Boehringer Ingelheim Venture Fund in Financing Round, with Participation from Delos and Blue Bay Capital New Investment, Combined with Recent $8.4 Million ARPA-H Award, Brings Funds ...
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