Poseida Therapeutics Provides Update on Key Programs and Developments During R&D Day
"Over the past year, we have made tremendous progress as we continue to validate our novel technology platforms and advance our broad and deep clinical and preclinical programs," commented
Specific highlights will include an early look at the ongoing P-PSMA-101 clinical trial; demonstration of the potential for single treatment cures with a completely non-viral nanoparticle-based gene therapy system; an extensive study of our Cas-CLOVER™ Site-Specific Gene Editing System demonstrating best-in-class gene editing specificity; and a look at our CAR-NK, and induced pluripotent stem cell, or iPSC, capabilities.
Key Program Highlights
Autologous CAR-T Update
P-BCMA-101 is an autologous CAR-T product candidate in an ongoing Phase 1 dose expansion trial and Phase 2 trial in development for the treatment of relapsed/refractory multiple myeloma to treat patients with multiple myeloma. Today's discussion will include data demonstrating the importance of T stem cell memory in CAR-T.
P-PSMA-101 is a solid tumor autologous CAR-T product candidate in an ongoing Phase 1 dose escalation trial in development to treat patients with metastatic castrate resistant prostate cancer, or mCRPC. Today's presentation will include a case study of a patient with mCRPC treated with P-PSMA-101 at a dose of 0.25 x 10e6 cells/kg (~20 x 10e6 total cells) who showed a marked decrease in PSA expression levels of more than 50% in the first three weeks post treatment and is continuing on trial. The patient was reported to have Grade 1 CRS in the second week which was treated to resolution. The Company intends to provide an additional update on this program later in 2021.
Allogeneic CAR-T Update (including Cas-CLOVER off-target analysis)
P-BCMA-ALLO1 is the Company's first allogeneic CAR-T product candidate in development for the treatment of relapsed/refractory multiple myeloma. The Company will present updated preclinical data and ongoing IND enabling work, with an expected filing in the first half of 2021. Data utilizing Cas-CLOVER™, the Company's high-precision gene editing technology to eliminate knock out TCR and B2M to address alloreactivity in P-BCMA-ALLO1, will also be presented.
P-MUC1C-ALLO1 is the Company's allogeneic CAR-T product candidate currently in preclinical development, with the potential to treat a wide range of solid tumors, including breast and ovarian cancers. The Company will share updated preclinical data demonstrating complete tumor elimination in triple negative breast and ovarian cancer models. P-MUC1C-ALLO1 will be the first clinical product to be manufactured at
piggyBac® Delivery in vivo for Liver Directed Gene Therapies
P-OTC-101 is the Company's first liver-directed gene therapy program for in vivo treatment of urea cycle disease caused by congenital mutations in the OTC gene, a condition characterized by high unmet medical need. Preclinical data from ongoing IND enabling studies will be presented by
Anti-cKit CAR-T: Safer non-genotoxic conditioning regimens are potentially possible with the Company's anti-cKit CAR-T program for hematopoietic stem cell, or HSC, conditioning, which may reduce transplant morbidity and mortality, resulting in better outcomes and a greatly expanded number of potential indications. Data include results from preclinical experiments demonstrating the ability of anti-cKit CAR-T cells to deplete human stem cell grafts in NSG mice and to prolong survival in a mouse model of AML.
Genetically Modified HSCs: HSCs can be modified via the piggyBac DNA Delivery System and/or the Cas-CLOVER Site-Specific Gene Editing System. Today's presentation will show data confirming that genetically modified HSCs engraft in the bone marrow and demonstrate long-term persistence. CAR-HSC has the potential to be a highly effective CAR-T approach, as it theoretically could provide an inexhaustible supply of effector cells to eradicate tumor and can be differentiated to generate high yields of CAR-T, CAR-NK and CAR-myeloid cells.
iPSCs: Cas-CLOVER is also efficient for creating knockouts and knock-ins in induced pluripotent stem cells, or iPSCs, with very low toxicity. Data will be presented showing the greater efficiencies of Cas-CLOVER as compared to an industry standard editing platform for therapeutic knock-in using plasmid DNA.
Genetically Modified NK Cells: The Company will also present data on efficient genetic modification of NK Cells using piggyBac and Cas-CLOVER platform technologies. The Cas-CLOVER gene editing system can be used to efficiently edit NK cells, or CAR-NK cells, while piggyBac can be used to effectively deliver large therapeutic transgenes to activated or
R&D Day Webcast Information
A live webcast of the Company's R&D Day event will be available on the Investors & Media section of
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding the clinical data presented, the potential benefits of
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