Looking at $BEAM science:
Taking a look at the preclinical data for their programs so far.
Taking a look at the preclinical data for their programs so far.
1/ They have two main programs around correcting Sickle Cell disease. Their first program BEAM-101 is doing a simple gene knockout on the gene that suppresses Fetal Hemoglobin expression causing the reactivation of the Fetal Hb gene.
2/ Their second program uses base editing to change the defective base in the Sickling Hemoglobin to a Makassar that works normally. 
3/ BEAM-101:
ex-vivo editing of Fetal Hb using electroporation
Showed 90% editing in CD34 cells
Showed greater than a 60% increase in Fetal Hb
Showed a decrease in Sickling Hb to less then 20%
This is well below the level necessary to prevent sickling
They only have mouse data.
ex-vivo editing of Fetal Hb using electroporation
Showed 90% editing in CD34 cells
Showed greater than a 60% increase in Fetal Hb
Showed a decrease in Sickling Hb to less then 20%
This is well below the level necessary to prevent sickling
They only have mouse data.
4/ There exists 2 potential off target risks of base editing. They are guide dependent and guide independent off target deaminations. They ran extensive tests and showed no signs of any potential off target deaminations.
5/ BEAM-102:
ex-vivo editing of the Beta Hb using electroporation
This approach attempts to fix the defective Beta globin gene why changing the A to a G which makes it a usable variant of Beta Globin called the Makassar Variant.
ex-vivo editing of the Beta Hb using electroporation
This approach attempts to fix the defective Beta globin gene why changing the A to a G which makes it a usable variant of Beta Globin called the Makassar Variant.
6/ Showed 80% correction of HB to the Makassar variant
70% of the produced RBC colonies showed 70% bi-allelic editing
20% of the produced RBC colonies showed 20% mono-allelic editing
70% of the produced RBC colonies showed 70% bi-allelic editing
20% of the produced RBC colonies showed 20% mono-allelic editing
7/ CAR-T:
They have an early CAR-T program focused on anti-CD7 for T cell ALL. I give them credit for going after a leukemia that isn't being targeted by everyone else. T cell ALL is the rarer form of ALL as most are B cell related.
They have an early CAR-T program focused on anti-CD7 for T cell ALL. I give them credit for going after a leukemia that isn't being targeted by everyone else. T cell ALL is the rarer form of ALL as most are B cell related.
8/ BEAM-201:
Universal CD7 targeted CAR-T
Created using electroporation
Included knockout of TRAC, CD52, CD7 and PD1 while inserting the anti CD7 CAR.
Universal CD7 targeted CAR-T
Created using electroporation
Included knockout of TRAC, CD52, CD7 and PD1 while inserting the anti CD7 CAR.
9/ Showed 96% to 99% on target editing of all 4 knockouts
No known chromosomal rearrangements were seen
85% of cells expressed the CAR after insertion
Estimated that 77% of had all 5 genetic modifications
In-vivo demonstrated CD7 dependent cytokine expression
No known chromosomal rearrangements were seen
85% of cells expressed the CAR after insertion
Estimated that 77% of had all 5 genetic modifications
In-vivo demonstrated CD7 dependent cytokine expression
10/ Showed in-vivo clearing of CD7 models
Next Steps will be in-vivo delivery using Lipid Nanoparticles.
Next Steps will be in-vivo delivery using Lipid Nanoparticles.
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