PIPELINE

Cell Type Matters

CAR-T for Oncology

CAR-T therapy is a form of anti-cancer therapy in which T cells are genetically modified to attack tumors, an approach that has revolutionized how cancer is treated. Currently, all commercially available CAR-T therapies are autologous, where the T cells are derived from each patient. Poseida believes that the future of CAR-T and our ability to offer new treatment options lies in allogeneic approaches, where the T cells are derived from healthy donors.

Our clinical and preclinical candidates are designed as off-the-shelf treatments for cancer. We modify healthy donor derived T cells using Cas-CLOVER gene editing to disrupt expression of T cell receptor beta and human leukocyte antigens (HLA). Specifically, T cell receptor beta gene deletion eliminates or reduces unwanted immune reactions, such as graft-versus-host-disease (GvHD). Beta-2 microglobulin gene deletion improves persistence by eliminating CAR-T rejection by the patient’s immune system via mismatched HLA proteins. The large cargo capacity of our piggyBac system allows us to develop allogeneic CAR-Ts able to target multiple antigens for greater tumor recognition capacity.

DISCOVERY

PRECLINICAL

IND-ENABLING

PHASE 1

PHASE 2

Heme Malignancies

P-BCMA-ALLO1

The Roche company logo.

PHASE 1

This allogeneic CAR-T candidate targeting B cell maturation antigen, or BCMA, is in development to treat relapsed/refractory multiple myeloma. This allogeneic program offers an improved VH-based binder which targets BCMA. This program is being developed in partnership with Roche and has shown early evidence of encouraging safety and efficacy.

P-CD19CD20-ALLO1

The Roche company logo.

PHASE 1

This allogeneic dual CAR-T candidate targets both CD19 and CD20 antigens to treat B-cell malignancies. Dual targets address the limitations of single antigen loss and tumor variability. This program is being developed in partnership with Roche.

P-BCMACD19-ALLO1

The Roche company logo.

Option

Option

PRECLINICAL

This allogeneic dual CAR-T candidate targets both BCMA and CD19 to treat relapsed/refractory multiple myeloma.

P-CD70-ALLO1

The Roche company logo.

Option

Option

PRECLINICAL

This allogeneic CAR-T candidate targets CD70 for the treatment of acute myeloid leukemia.

Solid Tumor

P-MUC1C-ALLO1

PHASE 1

This allogeneic CAR-T candidate is in development for multiple solid tumor indications including breast, colorectal, lung, ovarian, pancreatic and renal carcinomas, as well as other cancers expressing a cancer-specific form of the Mucin 1 protein called MUC1-C.

P-PSMA-ALLO1

PRECLINICAL

This allogeneic CAR-T candidate targets prostate-specific membrane antigen, or PSMA, to treat prostate cancer. This allogeneic program offers improved VH-based binding technology targeting PSMA, which can potentially enhance anti-tumor efficacy.

Gene Therapy

We are developing several liver-directed gene therapies to address rare and life-threatening diseases that are often diagnosed in early infancy. Our initial focus utilizes piggyBac in combination with AAV and non-viral nanoparticles or fully non-viral nanoparticle delivery. Our long-term aim is to move to fully non-viral therapies.

DISCOVERY

PRECLINICAL

IND-ENABLING

Liver Directed

P-FVIII-101

PRECLINICAL

This liver-directed gene therapy combines piggyBac with our nanoparticle delivery technology for the treatment of Hemophilia A. Hemophilia A is a bleeding disorder caused by a deficiency in Factor VIII production. Our preclinical data have demonstrated the potential to correct Factor VIII deficiency to near-normal levels in both juvenile and adult mouse models using our fully non-viral system.

P-OTC-101

PRECLINICAL

This liver-directed gene therapy combines piggyBac with AAV, also called our Hybrid System, for the treatment of OTC deficiency (OTCD). OTCD is an often-fatal urea cycle disease caused by congenital mutations in the OTC gene. In a neonatal mouse model of severe OTCD, we observed an approximately 100-fold increase in OTC levels with a single treatment showing correction of the underlying cause of OTCD.

P-PAH-101

PRECLINICAL

P-PAH-101 is a liver-directed gene therapy to treat Phenylketonuria (PKU). PKU is an inherited genetic disorder caused by mutations in the PAH (phenylalanine hydroxylase) gene resulting in buildup of phenylalanine in the body. If left untreated, PKU can affect a person’s cognitive development. P-PAH-101 utilizes piggyBac technology combined with a hybrid adeno-associated virus (AAV) and nanoparticle delivery system. Our preclinical data has demonstrated the potential to resolve phenylalanine to normal levels following a single treatment in juvenile and adult mice.

Our Partnerships

Poseida welcomes partners and collaborators to help us translate our innovative technologies into cell and gene therapies with the capacity to cure. We know the challenges we face cannot be solved by one person – or even one organization. We strive to partner with leaders in their fields who share our commitment towards developing better and safer treatments for those with limited or no therapeutic options.

With Roche, our strategic collaboration focuses on allogeneic CAR-T therapies directed at hematologic malignancies. The partnership includes multiple existing Poseida programs along with the development of novel off-the-shelf cell therapies against targets in multiple myeloma, B-cell lymphoma, and other hematologic cancers.

Partner With Us

If you are interested in collaborating with Poseida to usher in a new class of innovative solutions for cell and gene therapies, please contact us. We are actively seeking potential collaborators in these areas:

  • Allogeneic CAR-T or TCR-T therapies for solid tumors
  • In vivo liver or other tissue-directed gene therapies
  • Application of our technology in therapeutic areas beyond CAR-T and gene therapy
  • Novel technologies that further enable our approaches to cell and gene therapy