On January 31, 2017 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new immunotherapies, reported improved production times utilizing its non-viral platform to engineer chimeric antigen receptor (CAR)-modified T cells in an ongoing Phase 1 study of second-generation Sleeping Beauty CD19-specific CAR+ T cells (Press release, Ziopharm, JAN 31, 2017, View Source [SID1234517599]). Plans to progress the Company’s point-of-care (POC) approach with administration of CAR-T cell therapy in less than 2 days are also underway helping expand access to innovative T-cell therapies.

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"Through the application of Intrexon’s industrial engineering components and principles, we have enhanced our non-viral approach to T-cell therapies. Shortening the manufacturing process and avoiding the complexity of viral-based approaches with Sleeping Beauty represents a significant advance which dramatically reduces barriers to broadening delivery of CAR-expressing T cells to a wide range of institutions and importantly cancer patients in need," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM.

In contrast to the multi-step methods to produce virus-modified CAR- and TCR-expressing T cells, the non-viral Sleeping Beauty system offers the ability to rapidly generate genetically modified products through a simplified process. The improvements include avoiding activation and propagation of T cells as well as reduced time in culture.

In the clinical setting, data published in the Journal of Clinical Investigation in August 2016 from two Phase 1 trials, infusing first-generation Sleeping Beauty CD19-specific CAR T cells produced in 4 weeks, demonstrated positive long-term survival data for patients with acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL). In the ongoing Phase 1 study utilizing second-generation Sleeping Beauty CD19-specific CAR+ T cells for lymphoid malignancies, compressed production times with younger T cells are being deployed:

A patient with multiple-relapsed B-cell ALL received Sleeping Beauty-modified CD19-specific CAR+ T cells with the manufacturing time reduced to 3 weeks and achieved a complete remission with normalization of PET/CT tumor imaging.
A patient with triple-hit NHL treated in January 2017 was the first to receive Sleeping Beauty-modified CD19-specific CAR+ T cells with the manufacturing time reduced to 2 weeks.
In the pre-clinical setting, the time to administration of third generation Sleeping Beauty CAR+ T cells co-expressing a membrane-bound version of IL-15 (mbIL15) has been reduced to less than two days. This shortened process delivers genetically modified T cells with superior proliferative potential. Data presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2016, supported by an earlier publication in the Proceedings of the National Academy of Sciences, revealed promising results:

Third generation Sleeping Beauty CAR+ T cells demonstrated that a single low-dose of T cells co-expressing a CD19-specific CAR and mbIL15 resulted in sustained in vivo persistence that produced potent anti-tumor effects and superior leukemia-free survival.
These clinical and pre-clinical data support the Company’s POC plans to rapidly infuse Sleeping Beauty CAR+ T cells in a Phase I trial to be launched later this year. With the intent to administer clinical-grade Sleeping Beauty CAR+ T cells in less than 48 hours, this non-viral CAR-T approach has the potential to outpace viral-based methods.

"Together with ZIOPHARM, we are realizing the promise of engineered T cells and are excited to advance the POC approach. Our ability to generate T cells that co-express immunoreceptors and cytokines such as membrane-bound IL-15 through non-viral gene transfer, plus integration of our RheoSwitch Therapeutic System enabling conditional control of these and other essential components, represents what we believe will be the best-in-class platform in CAR-T and TCR-based cellular therapy," stated Geno Germano, President of Intrexon Corporation (NYSE:XON).plete remission with normalization of PET/CT tumor imaging.
A patient with triple-hit NHL treated in January 2017 was the first to receive Sleeping Beauty-modified CD19-specific CAR+ T cells with the manufacturing time reduced to 2 weeks.
In the pre-clinical setting, the time to administration of third generation Sleeping Beauty CAR+ T cells co-expressing a membrane-bound version of IL-15 (mbIL15) has been reduced to less than two days. This shortened process delivers genetically modified T cells with superior proliferative potential. Data presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2016, supported by an earlier publication in the Proceedings of the National Academy of Sciences, revealed promising results:

Third generation Sleeping Beauty CAR+ T cells demonstrated that a single low-dose of T cells co-expressing a CD19-specific CAR and mbIL15 resulted in sustained in vivo persistence that produced potent anti-tumor effects and superior leukemia-free survival.
These clinical and pre-clinical data support the Company’s POC plans to rapidly infuse Sleeping Beauty CAR+ T cells in a Phase I trial to be launched later this year. With the intent to administer clinical-grade Sleeping Beauty CAR+ T cells in less than 48 hours, this non-viral CAR-T approach has the potential to outpace viral-based methods.

"Together with ZIOPHARM, we are realizing the promise of engineered T cells and are excited to advance the POC approach. Our ability to generate T cells that co-express immunoreceptors and cytokines such as membrane-bound IL-15 through non-viral gene transfer, plus integration of our RheoSwitch Therapeutic System enabling conditional control of these and other essential components, represents what we believe will be the best-in-class platform in CAR-T and TCR-based cellular therapy," stated Geno Germano, President of Intrexon Corporation (NYSE:XON).. Plans to progress the Company’s point-of-care (POC) approach with administration of CAR-T cell therapy in less than 2 days are also underway helping expand access to innovative T-cell therapies.

"Through the application of Intrexon’s industrial engineering components and principles, we have enhanced our non-viral approach to T-cell therapies. Shortening the manufacturing process and avoiding the complexity of viral-based approaches with Sleeping Beauty represents a significant advance which dramatically reduces barriers to broadening delivery of CAR-expressing T cells to a wide range of institutions and importantly cancer patients in need," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM.

In contrast to the multi-step methods to produce virus-modified CAR- and TCR-expressing T cells, the non-viral Sleeping Beauty system offers the ability to rapidly generate genetically modified products through a simplified process. The improvements include avoiding activation and propagation of T cells as well as reduced time in culture.

In the clinical setting, data published in the Journal of Clinical Investigation in August 2016 from two Phase 1 trials, infusing first-generation Sleeping Beauty CD19-specific CAR T cells produced in 4 weeks, demonstrated positive long-term survival data for patients with acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL). In the ongoing Phase 1 study utilizing second-generation Sleeping Beauty CD19-specific CAR+ T cells for lymphoid malignancies, compressed production times with younger T cells are being deployed:

A patient with multiple-relapsed B-cell ALL received Sleeping Beauty-modified CD19-specific CAR+ T cells with the manufacturing time reduced to 3 weeks and achieved a complete remission with normalization of PET/CT tumor imaging.
A patient with triple-hit NHL treated in January 2017 was the first to receive Sleeping Beauty-modified CD19-specific CAR+ T cells with the manufacturing time reduced to 2 weeks.
In the pre-clinical setting, the time to administration of third generation Sleeping Beauty CAR+ T cells co-expressing a membrane-bound version of IL-15 (mbIL15) has been reduced to less than two days. This shortened process delivers genetically modified T cells with superior proliferative potential. Data presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2016, supported by an earlier publication in the Proceedings of the National Academy of Sciences, revealed promising results:

Third generation Sleeping Beauty CAR+ T cells demonstrated that a single low-dose of T cells co-expressing a CD19-specific CAR and mbIL15 resulted in sustained in vivo persistence that produced potent anti-tumor effects and superior leukemia-free survival.
These clinical and pre-clinical data support the Company’s POC plans to rapidly infuse Sleeping Beauty CAR+ T cells in a Phase I trial to be launched later this year. With the intent to administer clinical-grade Sleeping Beauty CAR+ T cells in less than 48 hours, this non-viral CAR-T approach has the potential to outpace viral-based methods.

"Together with ZIOPHARM, we are realizing the promise of engineered T cells and are excited to advance the POC approach. Our ability to generate T cells that co-express immunoreceptors and cytokines such as membrane-bound IL-15 through non-viral gene transfer, plus integration of our RheoSwitch Therapeutic System enabling conditional control of these and other essential components, represents what we believe will be the best-in-class platform in CAR-T and TCR-based cellular therapy," stated Geno Germano, President of Intrexon Corporation (NYSE:XON).