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Wednesday, February 14, 2024 5:38:59 PM
1. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
FDA Approved the FIRST EVER Gene Editing Therapy in Dec 2023
So before that Gene Editing was considered RUO [Research Use Only]. In Revenue terms < $1B across the board [various research institutions]
2. CRISPR/Cas9 was the technology that was approved in '1' above.
3. ABBIE uses a RNA targeted integration of a desired DNA Seq WTF does that mean? It means that in CRISPR the DNA's double strand is cut and a NEW SEQ is inserted [The risk here being Nature's innate ability to repair DNA...BEFORE the intended Gene Editing & fuck up, ex:- Somebody could walk out of the clinic with a cancer...when they went in for say...Sickle Cell therapy, and die in < '1' year].
4. Best way to explain ABBIE is that if you have a '3' legged table, that is wobbly. You DON'T Cut all THREE LEGS to Re-size them!
You might want to add a Shim to the bottom of one of the legs!
CRISPR by itself cuts BOTH strands of a DNA SEQ [DNA is a Double Helix Strand] and then uses Cas9 for SPECIFICITY.
ABBIE still has the specificity of Cas9, HOWEVER, it uses a guide-RNA to construct the repair [Compare this too the Shim I alluded to above]
Hope this helps....It's NOT WHETHER ABBIE was NOT considered useful by larger companies, it's just that ONLY in DEC 2023 Gene-Therapy was approved....Barron needs to let Aguilar loose...to go find Partnerships & he needs to do all the CDMO, Rx, making Coffee...etc.
The PPS will 'F' kng take off. With just ONE PARTNER!
I agree with some of the stuff that SLC raised. Barron needs to DELIVER ! We're in OT now. It's NOW or NEVER !
Throw me to the WOLVES.......and I shall return LEADING THE PACK!
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