LA Times Piece Glosses Over Gritty Realities Beta Thalassemia Gene Therapy: Bluebird Bio vs. San Rocco Therapeutics

SRT gene therapy vector, since 2012, two out of three patients treated with myelosuppression and the SRT investigational product produced in 2009-2019 have no adverse effects reported and a decrease in transfusions by 43%.

But there is more. While Hiltzik briefly touches on adverse effects associated with the Bluebird gene therapy product now approved, he doesn’t delve into any details. Actually, Zynteglo is associated with adverse events, including patients with Clonal Dominance (a precursor to cancer), Myelodysplastic Syndrome, and Leukemia. By December 2021, Fierce Pharma’s Angus Liu wrote about yet again another FDA hold against the gene therapy as the company acknowledged in a press release. Why didn’t Hiltzik discuss these incidents?

Hiltzik didn’t cover any of the legal skirmishes involving the gene therapy’s intellectual property status. SRT secured the IP from MSK. Now, at least some experts at MSK and elsewhere suggest that lentiviral vectors are safer with an insulator. See the piece in Cell on this topic. Interestingly, as reported recently by TrialSite, while San Rocco Therapeutics (SRT) secured a settlement with MSK and the rights to the lentivector gene therapy for beta-thalassemia, the academic medical center continues to refuse access to the vector, despite its obligation as part of a legal settlement. Is it because MSK added an insulator (thanks to public funds)which improves the investigational product? Are they holding back for other reasons?

Why wouldn’t they follow through on what was settled? Who are they aligned with behind the scenes? Why would the Los Angeles Times totally bypass such a related story?

According to a recent discussion with San Rocco Therapeutic CEO Patrick Girondi: “Bluebird misappropriated SRT technology to attempt to fix their faulty vector which uses a mutant gene. We arecurrently in a lawsuit for patent infringement—as a result of Bluebird buying the wrong product, the misappropriation, sabotage, and cost investors billions. The stock fell from over 200 with a $12b market capitalization to under a million. But there is a bigger victim in all of this—the entire country. People lose faith in our system, and we all suffer greatly.”

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