Public release of ASH’15 (American Society of Hematology) abstracts today was a bomb! Stocks of some gene/ cell therapy companies were heavily impacted today by release of ASH abstracts. Here is my pick of the most interesting clinical-focused presentations. Try to get more info and live tweet from ASH’15, which will take place a month from now in Orlando.
The first case of infusion allo- TALENs-modified CD19 CAR T-cells in child with B-ALL
This n=1 case with short-term follow-up sent stock of French biotech company Cellectis to the sky in the last 4 days (+58%). It was picked by Nature and multiple other media outlets.
Update from Northstar trial – gene/ cell therapy of beta-thalassemia major
This update caused big stock plunge (-21% today) of “biotech favorite” company Bluebird bio – read coverage here and here and here.
Well, investors must be very naive and immature to expect 100% cures at this point.
CAR- modified cells
Overview of CAR T-cell clinical trials updates from ASH’15 here.
First-in-man use of CD123-CAR T-cells in AML
Preliminary results of first-in-human anti-CD22 CAR T-cell therapy trial
CRISPR/Cas9 gene editing of TCR, HLA-I and PD1 for generation of universal CAR T-cells
Preclinical development of CAR19/IL15-modifieid cord blood-derived NK cells
Results of Ph1 trial of CD30-CAR T-cells in lymphoma
Lineage switch as a relapse mechanism in ALL treated with CD19-CAR T-cells
Termination of CD123-CAR-T Cells to mitigate toxicity in AML model
Development of TALENs-edited TCR-deficient universal CD123-CART-cells
Other immunocellular therapy trials
MSCs in severe neutropenic patients with septic shock – results of RUMCESS trial
14-years persistence of suicide gene-modified donor T-cells
Results of Ph1 trial assessing NK cells expanded from cord blood
Persistence of expanded cord blood-derived NK cells in patients with multiple myeloma
Third party CMV-specific cytotoxic T-cells in post HSC transplant patients with CMV infections
Hematopoietic cells expansion/ enhancement
Update on trial, assessing Notch-expanded non HLA-matched off-the-shelf cord blood product
Comparison of immune reconstitution between expanded and unmanipulated cord blood units
Ex vivo immune modulation of HPC(A) products by small molecules
Short pulse of HSC with PGE2 leads to robust enhancement of engraftment
Fucosylated cytotoxic T-cells demonstrate enhanced killing
Validation of Expamer reagent for activation and production of therapeutic CAR T-cells
Validation of automatic enumeration of HPC as alternative to CD34 flow cytometry
Manufacturing an enhanced CAR T-cell product by culture modifications
Optimization of CAR NK cell product development
Myeloid cell in PBMC inhibit expansion of CAR T-cells
Validation of CliniMACS Prodigy for automated transduction of T-cells with CARs
Successful CART-19 tech transfer from academia to industry
Please share your favorite abstracts, I may missed a lot of exciting things!