Super car-t cell therapy! Prgn-3005 is qualified for orphan drug: with safety switch, no need for in vitro expansion, only 2 days for patients

January 8, 2020 / BIOON / -- precision is a biopharmaceutical company dedicated to developing innovative gene and cell therapy to improve patients' lives Recently, the company announced that the U.S Food and Drug Administration (FDA) has granted prgn-3006 orphan drug qualification (odd), which is a pioneering therapy developed by precision's non viral ultracar-t treatment platform for the treatment of relapsed or refractory acute myeloid leukemia (AML) (clinical trial identifier: nct03927261) In the third quarter of 2019, precigen announced that it had completed the first cohort of patients enrolled in the clinical trial, and expected to release the first data in the second half of 2020 Orphan drug (Orphan Drug) is a kind of medicine used for prevention, treatment and diagnosis of rare diseases Rare diseases are a general term for diseases with very low incidence rate, also known as orphan diseases In the United States, rare diseases refer to the types of diseases with a population of less than 200000 Incentives for drug research and development of rare diseases include various incentives for clinical development, such as tax credits related to clinical trial costs, FDA user fee relief, FDA assistance in clinical trial design, and a 7-year market monopoly period for the approved indications after the drug is launched Dr Helen sabzevari, President and CEO of precision, said: "FDA has granted prgn-3006 orphan drug qualification, highlighting the key medical needs for new therapies for AML patients AML is a progressive, debilitating and often fatal disease with limited treatment options As the first regulatory qualification of our proprietary ultracar-t treatment platform, this orphan drug qualification will help to promote the development of prgn-3006, and provide important incentives and support to provide the drug to patients with this disease as soon as possible " Prgn-3006 is developed by using the revolutionary ultracar-t treatment platform of precigen company, which eliminates in vitro amplification, shortens manufacturing time, and provides the ability to carry out car-t treatment for patients in cancer center only one day after non viral gene transfer Prgn-3006 ultracar-t is a multi gene car-t cell therapy It uses the advanced non viral gene delivery system of precigen company to jointly express a chimeric antigen receptor, membrane-bound interleukin-15 (mbil15) and a killing switch It has better accuracy and control in targeting recurrent or refractory AML and high risk MDS The preclinical data of prgn-3006 ultracar-t in the treatment of AML and MDS by a day after gene transfer with non viral and polygenic auto car-t cells have been published at the annual meeting of the American Society of Hematology (ash) in 2019 Precigen's ultracar-t platform has the potential to subvert the current pattern of car-t cell therapy by shortening production time, reducing production related costs, using advanced methods to accurately target tumors and control the immune system to increase patient access The platform has brought several key advances: 1) non viral gene transfer uses multi gene vectors to express multi effect genes and improves tumor growth Targeted accuracy and control ability eliminate the need for viruses; 2) the persistence and expected phenotype of ultracar-t infusion help to solve T cell failure, which is a common problem in car-t therapy; 3) control T cells by combining kill switch technology, which can potentially improve safety; 4) use the proprietary non viral gene transfer process to rapidly manufacture ultracar-t cells Cell, eliminating the need for proliferation in vitro, thus reducing the waiting time of patients after gene transfer from several weeks to one day Source: precision Receives FDA orphan drug design for prgn-3006 ultracar-t Gamma in Patients with Acute Myeloid Leukemia (AML)