On February 22, 2023 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, reported that the Company plans to highlight its clinical and preclinical pipeline progress during a virtual R&D Day to be held today at 10:00am ET / 7:00am PT (Press release, Poseida Therapeutics, FEB 22, 2023, View Source [SID1234627547]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"R&D Day is our annual showcase for the innovative and exciting science we are advancing at Poseida that continues to drive our leadership in the field of cell and gene therapies," said Mark Gergen, Chief Executive Officer of Poseida Therapeutics. "Today, we will announce our second liver-directed preclinical gene therapy program partnered with Takeda: P-PAH-101 for the in vivo treatment of Phenylketonuria, or PKU. We are excited to share the progress we have made with our site-specific Super piggyBac platform to enable highly targeted site-specific editing and insertion, one of the most sought-after characteristics of genetic engineering. Finally, in our cell therapy portfolio, we continue to differentiate ourselves, expanding our capabilities for our allogeneic T cell platform to deploy TCRs in combination with CARs in solid tumors. We are thankful for the continued dedication of our scientists, partners and collaborators as we work together to unlock the potential of our technologies to treat patients with cancer and rare genetic diseases."

The Company’s third-annual R&D Day will feature its executive leadership and scientists for a morning of presentations and fireside chats with special guest speakers exploring the future of cell and gene therapy. The program will highlight the Company’s proprietary genetic engineering platform technologies, differentiated allogeneic CAR-T programs, and novel approaches to gene therapy as well as ongoing collaborations with Roche and Takeda.

External speakers will include:

George M. Church, Ph.D., a pioneer in the fields of genetics and synthetic biology and Chair of the Company’s Gene Therapy Scientific Advisory Board;

Madhu Natarajan, Ph.D., Head of the Rare Diseases Drug Discovery Unit at Takeda;

Christine Brown, Ph.D., Professor, City of Hope, a CAR-T cell expert and member of the Company’s Immuno-Oncology Scientific Advisory Board.

Key R&D Day Topics and Highlights

In Vivo Gene Therapy Programs

The Company will present advancements in hybrid technology highlighting the potential for single treatment cures across multiple diseases.

P-OTC-101 is the Company’s liver-directed gene therapy program for the in vivo treatment of urea cycle disease caused by a deficiency in the ornithine transcarbamylase (OTC) enzyme, a defect that impairs the body’s ability to detoxify ammonia, a byproduct of protein metabolism. The Company will show data highlighting disease correction and evaluation in non-human primates, with the Company’s lead lipid nanoparticle formulation that has demonstrated favorable tolerability.

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

Emerging Technologies in Gene Therapy

The Company will highlight its continuing focus on innovation in its emerging platform technologies at today’s event.

Site-specific Super piggyBac DNA Delivery, first unveiled at the Company’s R&D Day in February 2022, has continued to advance. The Company has made significant enhancements to efficiency and site-specific transposition, with up to 60% of haploid genomes modified.

The Company has made key enhancements to its non-viral gene delivery system resulting in nearly 10-fold improvements in DNA expression in the past 12 months on a pathway towards realizing the full potential of non-viral gene delivery.

Allogeneic Cell Therapy Programs

The Company will recap early clinical data presented at the European Society for Medical Oncology Immuno-Oncology Annual Congress in December 2022 (ESMO I-O) on both of its Phase 1 allogeneic cell therapy programs: P-MUC1C-ALLO1, a wholly-owned CAR-T product candidate targeting solid tumors derived from epithelial cells, including breast and ovarian cancers, and P-BCMA-ALLO1, a CAR-T product candidate partnered with Roche targeting relapsed/refractory multiple myeloma. The Company plans to present additional updates on both trials at a medical conference in 2023.

The Company will present preclinical data on additional emerging allogeneic CAR-T programs including P-CD19CD20-ALLO1, P-CD70-ALLO1 and P-ckit-ALLO1.

Emerging Technologies in Cell Therapy

The Company will share early preclinical data highlighting progress made towards developing dual-targeting CAR-TCR-T therapies capable of recognizing extracellular and intracellular solid tumor antigens for potential improved clinical outcomes.

R&D Day Webcast Information

Registration for this virtual event and access to the live webcast will be available on the Investors & Media section of the Company’s website, www.poseida.com. A replay of the webcast will be available for 90 days following the presentation.