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Msg  24172 of 24377  at  6/22/2021 1:48:29 PM  by


The following message was updated on 6/22/2021 2:03:47 PM.

Two very worn but shining pennies, fwiw -- "two cents" as a repeated concept.

From a 2018 overview of gene engineering progress offered by dnmun that includes a discussion of CAR-T as the first successful application of GE in cancer. LINK is provided:


As we are stuck in two buck chunk, it is worthwhile for some, me included, to reinforce the long thesis. The following excerpt is stated as "gospel" in the link when the article turns attention to CAR therapies within gene engineering:

>> Universal third-party CAR-T cells that can be used off the shelf would allow more rapid and cheaper treatment compared to autologous patient-specific T cells. <<

My point here: The gospel still holds true in competitor CAR-T trials I have reviewed ... UNTIL the news comes out on estimates of Eden BioCell and/or Precigen's cost of autologous CAR-T. Foundations crack under the corner offices to over 600 CAR-T trials around the world. Two-day in design, entire application at bedside, and just maybe ... no need for lymphodepletion. Yowza.

The cracks are directly tied to another excerpt about CAR-T future "challenges" found in the link:

>> Many challenges remain, including addressing genotoxicity from integrating gene delivery vectors or off-target genome editing, improving gene transfer or editing efficiency to levels necessary for effective treatment of many diseases, addressing immune responses to repeated in vivo administration of vectors, and reaching a societal consensus regarding contentious issues such as the ethics of germline editing and payment for expensive curative therapies. <<

Autologous Sleeping Beauty-vectored CAR-T is HLA-specific to the patient. Off-target has never been mentioned as it relates to SB. Survival of SB cell count transfer beyond 70% is proven and FDA-accepted. Dr. Cooper provides the "1,000-fold" expansion in daily cell count expansion with SB. They draw blood after the infusion and can count the number of cells present in the blood sample. Immune responses to a patient's own vector are mute. Costs in a four or five-digit dollar figure will break the chair legs under the sitting CEOs. As to unmentioned efficacy which this paper does not question, I defer to Dr. June's recent statement and Dr. Cooper's earlier report of four year 100% responses -- CAR-T does work. Sleeping Beauty in the application is not deviating with the basic engineering design of the CAR cell engineering platform ... only the vector.

I guess that when E-B and PGEN do provide initial CAR-T data ... Helen, Huang and Postma will have initial consistent with 80%-range ORR and minor AE concerns, but careful and sound data on cost for us to read ... and watch the game being changed before our very eyes. In addition to two-day vein-to-vein, both PGEN and EB have trial arms excluding lymphodepletion. All the best.

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