On April 4, 2022 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported that the FDA has granted Fast Track designation for PRGN-3006 UltraCAR-T in patients with relapsed or refractory (r/r) AML (clinical trial identifier: NCT03927261) (Press release, Precigen, APR 4, 2022, View Source [SID1234611410]). PRGN-3006 was previously granted FDA Orphan Drug Designation.

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PRGN-3006 UltraCAR-T is a multigenic autologous chimeric antigen receptor (CAR)-T cell treatment utilizing Precigen’s non-viral Sleeping Beauty system to simultaneously express a CAR specifically targeting CD33, which is over expressed on AML blasts; membrane bound IL-15 for enhanced in vivo expansion and persistence; and a kill switch to conditionally eliminate CAR-T cells for an improved safety profile.

Precigen’s UltraCAR-T platform is designed to overcome limitations of currently available CAR-T therapies by utilizing an advanced overnight non-viral gene delivery manufacturing process at a medical center’s cGMP facility without the need for ex vivo expansion. Current CAR-T cell therapies are limited due to, inter alia, the prolonged interval between apheresis to product infusion and an exhausted phenotype of T cells resulting from lengthy ex vivo expansion. UltraCAR-T cells for the PRGN-3006 study are manufactured overnight using Precigen’s proprietary UltraPorator system.

"We are very pleased to receive the FDA’s Fast Track designation, which facilitates development and expedites the review process of drugs that address serious conditions and high unmet medical needs," said Helen Sabzevari, PhD, President and CEO of Precigen. "AML is a rapidly progressing disease with a very poor prognosis. The Fast Track designation will help facilitate the timely development of this program and we look forward to working more closely with the FDA to potentially bring this new and highly differentiated overnight UltraCAR-T therapy to patients."

About AML
AML is a cancer that starts in the bone marrow, but most often moves into the blood.1 Though considered rare, AML is among the most common types of leukemia in adults.2 In 2019, it was estimated that 21,450 new cases of AML would be diagnosed in the US.2 AML is uncommon before the age of 45 and the average age of diagnosis is about 68.2 The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25 percent overall, and less than a 5 percent 5‐year survival rate for patients older than 65.3 Amongst elderly AML patients (≥ 65 years of age), median survival is short, ranging from 3.5 months for patients 65 to 74 years of age to 1.4 months for patients ≥ 85 years of age.3