P-FVIII-101 / Roche - LARVOL DELTA

Novel Cyclohexane Based Ionizable Lipids (CHILs) for Non-viral Liver Delivery of Complex Nucleic Acid Genetic Medicine Payloads (ASGCT 2025) - "Data demonstrating the evolution of this novel class of CHILs from conception through the identification and integration of HBC365 into P-KLKB1-101 and P-FVIII-101 programs will be covered in this presentation. Disease Focus of Abstract:None"

Cardiovascular • Complement-mediated Rare Disorders • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Hereditary Angioedema • Rare Diseases

P-FVIII-101 is a Non-viral Genetic Medicine with the Potential for Controlled Modulation of FVIII Transgene Expression to Match Patient Needs (ASGCT 2025) - "The standard of care for patients with severe hemophilia A (HemA) has dramatically improved in recent decades with the advent of prophylactic bispecific antibodies such as emicizumab...Delivery of small molecule rimiducid induces activation of iCasp9 in transduced cells triggering their death...Future preclinical studies will further explore the tunability of these systems to deliver tailored therapeutic levels that provide a durable solution for FVIII restoration in HemA patients. Disease Focus of Abstract:Hemophilia"

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Pain • Pediatrics • Rare Diseases

Sustained FVIII Expression with a Tolerable, Titratable, Fully Non-Viral Gene Therapy for Hemophilia A (ASGCT 2024) - "For potential individualized correction in HemA patients we also demonstrate the ability to achieve near full correction (96%) in HemA mice through repeat dosing.Overcoming historical challenges in DNA delivery, our non-viral platform efficiently delivers DNA and mRNA, combining the super piggyBac (SPB) transposase mRNA with plasmid human FVIII (hFVIII) transposon DNA (P-FVIII-101)...Adult Sprague-Dawley rats and C57Bl6 mice were administered the same LNPs. Delivery to rats resulted in 50-75% hFVIII Ag expression, which translates to anticipated therapeutic correction.This validation of Poseida's non-viral platform components—including novel ionizable lipids, targeting ligands, and excipients—highlights the capabilities of the piggyBac DNA insertion system and our non-viral approach for stable FVIII transgene expression, with potential for re-dosing, providing proof-of-principle for an effective and durable therapy for Hemophilia A."

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hepatology • Rare Diseases • IL6

Poseida Therapeutics Provides Updates and Financial Results for the First Quarter of 2023 (PRNewswire) - "P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate targeting solid tumors derived from epithelial cells, including breast and ovarian cancers...The Company currently expects to present further clinical updates for the program at a medical meeting in 2023; P-BCMA-ALLO1 is an allogeneic CAR-T product candidate being developed to target relapsed/refractory multiple myeloma...The Company currently expects to present further clinical updates for the program at a medical meeting in 2023; P-CD19CD20-ALLO1 is a preclinical allogeneic CAR-T product...The Company expects to file an IND for P-CD19CD20-ALLO1 in mid-2023; The Company is advancing its P-FVIII-101 preclinical program partnered with Takeda, which is in development for the in vivo treatment of Hemophilia A....The Company is presenting preclinical data from this program at the upcoming American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting being held in Los Angeles on May 16-20, 2023."

IND • P1 data • Preclinical • Genetic Disorders • Hematological Malignancies • Hemophilia • Multiple Myeloma • Oncology

Poseida Therapeutics Hosts Second Annual Virtual R&D Day Highlighting Novel Pipeline Assets and Latest Technology Innovations (PRNewswire) - "Poseida Therapeutics, Inc...today announced that the Company plans to highlight its clinical and preclinical pipeline progress during a virtual R&D Day to be held today beginning at 10:00am ET / 7:00am PT....Today the Company will share data showing potentially therapeutic levels of expression of Factor VIII can be achieved using a fully nanoparticle system to deliver treatment in juvenile animal models, demonstrating the potential to achieve single treatment cures even in the underserved juvenile patient population."

Preclinical • Genetic Disorders • Hemophilia

Poseida Therapeutics Announces Research Collaboration with Takeda for Novel Non-Viral In Vivo Gene Therapies (PRNewswire) - "Poseida Therapeutics, Inc...today announced that it has entered into a research collaboration and exclusive license agreement with Takeda Pharmaceutical Company Limited ('Takeda')...for the research and development of up to eight gene therapies. The collaboration will focus on developing non-viral in vivo gene therapy programs, including Poseida's Hemophilia A program. Under the terms...parties will collaborate to initially develop up to six in vivo gene therapy programs....Takeda also has an option to add two additional programs to the collaboration and is obligated to provide funding for all collaboration program R&D costs. Poseida will receive an upfront payment of $45 million and preclinical milestones that together could potentially exceed $125 million in the aggregate, if milestones for six programs are achieved. Poseida is also eligible to receive future clinical development, regulatory, and commercial milestone payments..."

Licensing / partnership • Genetic Disorders • Hemophilia