The American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), and World Federation of Hemophilia (WFH) have developed joint clinical practice guidelines on the diagnosis and management of von Willebrand Disease (VWD), the world s most common inherited bleeding disorder. The guidelines were published today in
Beam Therapeutics: A Gene Editing Stock
Beam Therapeutics Inc. (NASDAQ:BEAM), founded in 2017, is a biotech firm working on CRISPR 2.0, which is an advanced base editing technology that targets a single misspelling in the DNA code, ensuring greater accuracy. The technology basically allows scientists to permanently make specific edits to single bases in DNA and RNA, without the need of cutting the strands.
The technology could theoretically treat hundreds of genetic disorders caused by single-letter misspellings, commonly called point mutations, though the company has yet to carry out human trials for proving its efficacy in real life. Beam has several research programs in place for different rare diseases. However, it has not received regulatory approval for any clinical trial so far.
Chimeric Antigen Receptor (CAR) -modified Cytokine-Induced Killer Cell (CAR-CIK) Technology Featured on ASH
News Highlights: Chimeric Antigen Receptor (CAR) -modified Cytokine-Induced Killer Cell (CAR-CIK) Technology Featured on ASH
DURHAM, NC, December 16, 2020 (GLOBE NEWSWIRE) – CoImmune, Inc. announced today that its CAR-CIK technology was on display at the American Society of Hematology (ASH) annual meeting with an interim update on the phase 1/2 dose escalation clinical trial in B-cell acute lymphoblastic leukemia (B- ALL). The study is being conducted in Ospedale San Gerardo, Monza, Italy by Principal Investigator Andrea Biondi, MD and at Papa Giovanni XXIII, Bergamo, Italy by Principal Investigator Alessandro Rambaldi, MD. The presentation was on December 7 during the immunotherapy session and was given by Dr. Chiara Magnani from the Tettamanti Research Center, Monza, Italy.
Following is a transcript of their remarks:
Susan O Brien, MD: Hi, everyone, and welcome to this roundtable where we re going to discuss CLL presentations at ASH 2020 this year. I m Dr. Susan O Brien from the Chao Family Comprehensive Cancer Center at the University of California in Irvine, and I m joined by two esteemed colleagues, Dr. Jennifer Brown from Dana-Farber and Dr. Anthony Mato from Memorial Sloan Kettering.
We have two presentations today on the TRANSCEND clinical trial, and that is an interesting CAR-T trial of liso-cel which targets CD19, like many of the other CAR-Ts, but is a little bit different in that it has a defined component of CD4 and CD8 cells, which I think is the only CAR-T that s actually doing that. One of the presentations involved just the CAR-T alone and the other was the CAR-T given in combination with ibrutinib. Jennifer, maybe you could speak to that and the rationale for actually giving it with ibrutinib in the sense that many of the patients had
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