About CAR-T Therapies
Redefining cancer care
The growing field of immuno-oncology has emerged as a breakthrough in cancer treatment, the revolutionary science that harnesses a patient’s own immune system to attack disease. Our mission is to develop next-generation cell and gene therapeutics to treat patients with urgent medical needs, initially with development of CAR-T product candidates.
Our proprietary gene engineering platform technologies are instrumental in creating potential therapeutics predominantly composed of a specific T cell subset, stem cell memory T cells, or TSCM, which we believe can address the limitations of early-generation CAR-T therapies, including:
- A safer treatment that drives more gradual tumor killing with less toxicity
- A potentially longer lasting treatment with better duration of response
- The ability to move quickly to clinic
- Cost savings in manufacturing
- Key to success in solid tumors
What is CAR-T?
CAR-T therapy is a form of cell therapy that uses immune cells to attack cancer. The advent of CAR-T therapies has revolutionized treatment of some blood-based cancers by demonstrating profound initial response rates and, in some cases, the ability to cure. Despite these response rates, several key limitations have curtailed broad adoption of early-generation CAR-T products. Limitations include safety concerns and an inability to treat solid-tumor cancers. We believe we can overcome these limitations to create safer and more effective medicines for cancer and potentially other diseases.
Which diseases will Poseida seek to treat?
Initially, Poseida’s product candidates are focused on multiple myeloma and castrate-resistant prostate cancer, or CRPC. Our most advanced program, P-BCMA-101, is an autologous CAR-T therapy being developed to treat patients with relapsed/refractory multiple myeloma. We also plan to address breast, ovarian cancer, and other solid tumor indications with P-MUC1C-ALLO1, our first pan solid tumor product candidate that holds the potential to target several different cancers.
CEO Eric Ostertag, M.D., Ph.D., on How Stem Cell Memory T-Cells Can Lower Toxicity in Treating Cancer Patients
Siddiq Abdul-Alim, Ph.D., Describes The Mothership of All T-Cells
Julia Coronella, Ph.D., Recognizes the Impact of piggyBac® on Stem Cells
How is Poseida’s CAR-T approach different?
Our proprietary gene engineering technologies are used to create potential therapies predominantly composed of a specific T cell subset, stem cell memory, or TSCM, which we believe can address the limitations of other CAR-T therapies, including duration of response, the ability to treat solid tumors, and safety concerns. This is possible because Poseida does not rely on viral vectors to manufacture therapies, instead we use our novel non-viral gene engineering technologies that create high TSCM products and have multiple other attributes that result in a potentially safer treatment with equivalent or better outcomes.
- Strong correlation with best responses in the clinic
- More gradual tumor killing with less toxicity
- Potentially better duration of response and potential for re-response
- Key to CAR-T success in solid tumors
How are CAR-T therapies made?
Today, CAR-T therapies are customized to each patient by removing a specific set of cells from the individual’s blood, modifying the cells in a lab to intensify the immune system’s response to cancer, and re-injecting the cells into the patient. These are known as autologous therapies.
Poseida is also developing allogeneic therapies, which are created using the cells of a universal donor and produced in large quantities, so it is available for on-demand use. Poseida plans to develop allogeneic versions of all of our hematological and solid tumor product candidates.
About Gene Therapy
Seeking answers for rare genetic diseases
We have several gene therapies in development addressing rare and life-threatening diseases, including two liver-directed gene therapy product candidates in orphan genetic diseases – Ornithine Transcarbamylase (OTC) deficiency and Methylmalonic Acidemia (MMA). The potential for our science in gene therapy is far reaching, and we are excited to be researching multiple approaches in a wide array of cell types and tissues for non-liver-directed gene therapies. In fact, we have recently announced a new potential product candidate, P-FVIII-101, currently in development for the in vivo treatment of hemophilia A and delivered via Poseida’s proprietary nanoparticle technology.
Our gene therapy candidates utilize piggyBac in combination with AAV delivery (adeno-associated virus), and our innovative nanoparticle technology to overcome the limitations of traditional gene therapies that rely strictly on viral delivery. PiggyBac’s ability to deliver large capacity genetic cargo and permanently integrate into DNA enables us to extend our technologies into diseases beyond the reach of transient viral-based delivery methods. Our potential to enable durable gene expression, even in tissues with rapidly dividing cells allows us to pursue a wide spectrum of genetic diseases, including many indications within the pediatric population.
We believe that our approach will result in integration and long-term stable expression at potentially much lower doses than AAV technology alone, resulting in much greater tolerability and a better safety profile for patients. Our eventual goal is to completely replace AAV with our nanoparticle technology in all applications and rapidly develop safer and more effective gene therapies that address critical unmet needs.
P-BCMA-101 for Relapsed/Refractory Multiple Myeloma
Poseida is conducting an ongoing Phase 1 clinical trial for our lead drug candidate P-BCMA-101, developed for patients with relapsed/refractory multiple myeloma. The study is still enrolling.
P-PSMA-101 CAR-T Cells in the Treatment of Subjects with Metastatic Castration-Resistant Prostate Cancer (mCRPC)
Poseida is conducting a Phase 1 clinical trial for our drug candidate P-PSMA-101, developed for patients with prostate cancer. The study is enrolling.