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CARISMA Therapeutics Announces First Patient Dosed in Landmark Clinical Study Evaluating Engineered Macrophages in Humans

CARISMA Therapeutics Inc., a clinical stage biopharmaceutical company spun out of the University of Pennsylvania (Penn) and focused on discovering and developing innovative immunotherapies, announced that the first patient has been dosed in the Phase 1 multi-center clinical trial for CT-0508, a human epidermal growth factor receptor 2 (HER2) targeted chimeric antigen receptor macrophage (CAR-M).

This clinical trial represents a pivotal milestone in gene-based therapy development, as it is the first time CAR-engineered macrophages are being used in a clinical study. The study focuses on patients with recurrent or metastatic HER2-overexpressing solid tumors whose cancers do not have any approved HER2-targeted therapies or who do not respond to treatment. Preclinical findings for CT-0508 indicated that CAR-M therapy may have the potential to overcome challenges that T-cell therapies have encountered in the solid tumor setting.

“Through our novel CAR-M platform, CARISMA plans to develop new treatment options that aim to address the unmet needs of patients who are battling hard-to-treat cancers,” said Debora Barton, MD, Chief Medical Officer at CARISMA Therapeutics. “After much investigation in the lab, we are looking forward to evaluating CT-0508 in a clinical setting to assess this first CAR-engineered macrophage product and also provide vital learnings for a potential new way of treating cancer.”

CT-0508 was originally developed by Saar Gill, MD, PhD, Scientific Co-founder of CARISMA Therapeutics, and an Assistant Professor of Hematology-Oncology in the Perelman School of Medicine at the University of Pennsylvania and the Center for Cellular Immunotherapies at Penn’s Abramson Cancer Center, and Michael Klichinsky, PharmD, PhD, Scientific Co-founder, and Senior Vice President of Discovery at CARISMA Therapeutics.

“The preclinical findings suggest that CAR-Ms access solid tumors, survive in a hostile tumor environment, specifically phagocytose antigen-expressing cancer cells, and trigger an adaptive, long-lasting immune response,” Gill said. “This is the first time this kind of technology is being explored in humans, and we are excited to collect important data from this trial that will help to validate the platform as a potential new therapeutic approach for these patients.”

The clinical trial is open for enrollment and details can be found at ClinicalTrials.gov. CARISMA will continue to treat and evaluate patients at two clinical trial sites, including the University of Pennsylvania and the University of North Carolina Lineberger Comprehensive Cancer Center in Chapel Hill.

(Source: PR Newswire, 2021)


HepaTx, Namocell, and Takara Bio USA complete second phase of collaboration on single cell analysis for cell therapy to treat late-stage liver diseases

HepaTx Corporation, Namocell Inc., and Takara Bio USA, Inc. (TBUSA) announced successful completion of the second phase of their collaboration on single cell analysis of hepatocyte-like cells (iHeps) differentiated from adipose tissue-derived stromal cells (ASCs) using Namocell’s Single Cell Dispensers and Takara Bio’s SMART-seq® kits to characterize HepaTx’s novel cell therapies for liver disease. The results of the second phase demonstrated a 95% success rate in single cell isolation and library construction. Single cell RNA-seq showed distinct clusters of cells at various differentiation stages. The high reproducibility of results within sample groups will support development of novel cell therapies and validate production processes.

“Namocell’s single cell isolation platforms and Takara Bio’s single cell analysis products enabled us to take a deep look at the biology underlying our cell therapy platform and make key advances in development. Namocell’s gentle cell dispensing technology enabled us to obtain reliable and consistent phenotype information on our cells. Takara Bio’s reagents and sequencing technology yielded extremely consistent sample quality and very high information content. The results from this collaboration will be valuable for our development programs,” said Dr. Eric Schuur, CEO of HepaTx.

“We are excited to see the second phase results, and glad that our Single Cell Dispensers enabled HepaTx to advance their innovative stem cell-based therapy that could revolutionize the treatment of late-stage liver diseases by replacing liver transplants. Namocell’s Single Cell Dispensers simplify plate-based single cell RNA-seq, which not only produces more genes per cell, but also gives researchers the flexibility to decide how many samples to analyze and how many cells to be included in each sample, while keeping the analysis cost-efficient. This is ideal when you need to analyze a large amount of different samples but only a few cells per sample,” said Dr. Junyu Lin, CEO of Namocell.

“We are excited to be working with Namocell and HepaTx and contribute to the development of an innovative clinical solution. Leveraging our SMART-seq technology with Namocell’s Single Cell Dispenser is an ideal match for performing plate based single cell RNA-seq, enabling researchers to perform full length sequence analysis on single cells providing a deeper view into gene expression and cellular differentiation,” said Dr. Suvarna Gandlur, Associate Director of Next Generation Sequencing at Takara Bio USA.

(Source: PRNewswire, 2021)


Precigen Announces First Patient Dosed for PRGN-2012 AdenoVerse™ Immunotherapy in Patients with Recurrent Respiratory Papillomatosis (RRP)

Precigen, Inc., a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced that the first patient has been dosed in the Phase I study of PRGN-2012, a first-in-class, investigational off-the-shelf (OTS) AdenoVerse immunotherapy in adult patients with recurrent respiratory papillomatosis (RRP) (clinical trial identifier: NCT04724980). The US Food and Drug Administration recently granted Orphan Drug Designation for PRGN-2012 in RRP in March 2021.

PRGN-2012 is an innovative therapeutic vaccine with optimized antigen design that uses Precigen’s gorilla adenovector technology, part of Precigen’s proprietary AdenoVerse platform, to elicit immune responses directed against cells infected with HPV 6 or HPV 11. Gorilla adenovectors have numerous advantages, including the ability for repeat administration, the inability to replicate in vivo, which may improve safety, and the ability to deliver large payload capacity. In preclinical models, PRGN-2012 has demonstrated strong and specific immune response against HPV 6 and HPV 11.

PRGN-2012 is under development through a Cooperative Research and Development Agreement (CRADA) with the Center for Cancer Research (CCR) at the National Cancer Institute (NCI), which is part of the National Institutes of Health (NIH). This CRADA has allowed Precigen to rapidly and cost-effectively advance PRGN-2012 to the clinic. The Phase I clinical study of PRGN-2012 is led by Scott M. Norberg, DO, Assistant Research Physician, Genitourinary Malignancies Branch, of CCR NCI, and Clint T. Allen, MD, Principal Investigator, Section on Translational Tumor Immunology, of the National Institute on Deafness and Other Communication Disorders (NIDCD), which is also part of the NIH.

The Phase I study follows 3+3 dose escalation of PRGN-2012 as an adjuvant immunotherapy following standard-of-care surgical removal of visible papillomatosis disease. Patients receive up to four injections of PRGN-2012. The primary objective of the study is to determine safety and tolerability and recommended Phase II dose (RP2D) of PRGN-2012. The study will enroll 3 to 6 subjects at each dose level, and 12 patients will be treated at the maximum tolerated dose.

“Dosing the first patient with PRGN-2012, the first in the infectious disease setting, represents a significant milestone for the OTS AdenoVerse platform. RRP patients need new adjuvant treatment options to reduce the number of devastating repeat surgical procedures commonly associated with this disease,” said Helen Sabzevari, PhD, President and CEO of Precigen. “We look forward to producing clinical data to build upon the encouraging signals we have seen in preclinical studies in which PRGN-2012 was shown to induce robust HPV 6 and HPV 11-specific T-cell response in RRP patient samples in vitro.”

(Source: PRNewswire, 2021)