Accelerated Biosciences’ Immune-Privileged Human Trophoblast Stem Cells (hTSCs) Offer Breakthrough Opportunities in Cancer-Targeting Therapeutics and Regenerative Medicine Treatments

On May 11, 2021 Accelerated Biosciences, a regenerative medicine innovator, reported that new data that further demonstrates statistically significant cytolysis with induced pluripotent stem cell (iPSC)-derived natural killer (NK) cells programmed from its ethically sourced human trophoblast stem cells (hTSCs) (Press release, Accelerated Biosciences, MAY 11, 2021, View Source [SID1234579715]). Pluristyx, a Seattle-based firm supporting drug development, regenerative medicine, and cell and gene therapies, further confirmed Accelerated Biosciences’ hTSC line offers before-unrealized opportunities in cell-specific therapeutics. Along with this recent data on successful iPSC differentiation, Accelerated Biosciences has already demonstrated efficient differentiation of its pluripotent stem cells with remarkable doubling times and growth characteristics to programmed NK, cartilage, bone, fat, neuron, pancreas, liver, and secretome cells.

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"This new data validates our findings," explains Yuta Lee, President and Founder of Accelerated Biosciences. "We know the properties of our trophoblast stem cells have been long-sought by the medical science community because of the potential to speed and amplify the development of life-saving therapeutics; they’re immune privileged, chromosomally stable (not tumorigenic), pathogen free, pluripotent, easy to scale and manufacturer, and of special interest, they are ethically sourced from the chorionic villi (pre-placental tissue) of non-viable and often life-threatening tubal ectopic pregnancies." Mr. Lee’s father, Professor Jau-Nan Lee, MB, MD, PhD, an obstetrics and gynecologic physician and researcher in Taiwan, first isolated hTSC in 2003. Mr. Lee created Accelerated Biosciences to elevate the visibility of this pluripotent human trophoblast stem cell platform to those engaged in developing allogeneic cell therapeutics and has been instrumental in the filing and prosecution of intellectual property to protect the company’s hTSC platform – to date holding 34 patents.

Benjamin Fryer, PhD, Co-founder and CEO of Pluristyx, worked closely with Accelerated Biosciences to prepare much of its key hTSC data. Dr. Fryer, a trophoblast expert who was previously a research scientist at Janssen Research & Development of Johnson & Johnson, now serves on Accelerated Biosciences’ Scientific Advisory Board. "Initially I was skeptical these cells were what they said they were. If we hadn’t grown and characterized them in our lab, I might have remained skeptical. These are indeed trophoblast stem cells," explained Dr. Fryer. "The potential of these cells is enormous. One of the industry’s largest challenges is that it’s almost impossible to scale primary cells. These cells are scalable. With these cells you can make the amount required for millions of patients and they’re sourced compliant to regulatory requirements. We’ve made IPS cells (induced pluripotent stem cells) and NK (natural killer) cells from them, which is the next wave of cells for cell therapies. For therapeutic developers, because these cells are not sourced from a person or viable embryo, these cells deliver the trifecta of legal, ethical, and IP advantages."

"As the biotechnology industry works toward developing therapies that target only diseased cells without harming healthy cells and tissues, cell-based therapies draw increasing interest," explains industry expert, Martina Molsbergen, CEO of C14 Consulting, who has partnered with Accelerated Biosciences in a business development role. "With all the promise that cell therapies hold, the biotechnology industry also remains concerned that the therapeutics are derived in a socially and ethically responsible manner. Accelerated Biosciences has discovered and is now offering what scientists see as the holy grail of stem cell sources."

Prominent biosciences experts have been drawn to Accelerated Biosciences’ cell breakthrough. Protein chemist and molecular biologist Igor Fisch, PhD, former President and CEO of Selexis, Geneva, Switzerland, recognizes the impact that Accelerated Biosciences’ hTSCs will have on human health: "Not only are these cells ‘politically correct’, but they can also differentiate. Because they are sourced from pre-placenta material, they’re immune privileged, which means that are not seen as foreign by the human body. With these cells, we can create a cell bank – a single source for a wide range of patients."

Peter Hudson, FTSE, BSc Hons, PhD, Chief Scientist and a senior advisor to Avipep P/L in Melbourne, Australia, and an adjunct professor at the University of Queensland, led a large oncology consortium to complete the first Phase 1 clinical trial of a novel engineered antibody targeting prostate and ovarian cancer. Hudson’s interest in Accelerated Biosciences’ hTSCs has evolved into a role on its Scientific Advisory Board. "Trophoblast stem cells are likely to be the next wave of cancer-targeting therapeutics," explains Dr. Hudson. "The ability to ethically source trophoblast stem cells and program them to target only diseased, cancerous cells is very powerful technology."

Why are scientists so interested in stem cell-based therapies?

The human body constantly produces specialized cells from its own stem cells (undifferentiated cells) to renew and repair itself. Current therapies harness this power in autologous cell therapies in which the patient’s own cells are removed, differentiated into disease-fighting cells, and reinserted.

What makes the human trophoblast stem cell so important to medical science?

The human trophoblast stem cell (hTSC) comes from placental tissue and has special properties that make it extremely desirable to therapeutic developers. The hTSC is such an early stem cell that it has much more capacity for growth than a stem cell taken from an adult, for example. This means that one cell can become millions. The hTSC also carries with it the same immune-privilege that a growing embryo has inside its mother: it’s not seen as foreign although it’s genetically different than its mother. Unlike other foreign materials, the hTSC is not rejected by the human body, which means that it can be used with many different patients (allogeneic cell therapy). With these benefits, the scientific community holds a high regard for hTSCs, but it also faces socio-ethical concerns about how those stem cells are typically sourced.

Accelerated Biosciences sidesteps hTSC sourcing concerns in a profoundly elegant way. Dr. Jau-Nan Lee, an OB-GYN in Taiwan, found inspiration in what was considered medical waste. When surgical intervention was necessary to remove an ectopic pregnancy that would otherwise risk the woman’s life, the non-viable embryo and pre-placental tissue lodged in the fallopian tube was removed, sent to pathology, and discarded. Gaining permission from institutional colleagues and sampling the pre-placental tissue, Dr. Lee isolated hTSC that offered all the benefits of hTSC – pluripotency, immune privilege, and scalability – without pathogens and without ethical compromises.

Molecular Targeting Technologies, Inc. and University of Antwerp Begin First-in-Human Study of TDURA Diagnostic for Early Detection of Response to Colon Cancer Therapy

On May 11, 2021 Molecular Targeting Technologies, Inc. (MTTI) and University of Antwerp reported the approval of a Clinical Trial Application by the European Federal Agency for Medicines and Health Products (FAMHP) (equivalent to a US IND) (Press release, Molecular Targeting Technologies, MAY 11, 2021, View Source [SID1234579714]). The clinical study will evaluate the safety, dosimetry and treatment response of TDURA (99mTc-Duramycin), in patients with advanced colorectal cancer (CRC)* .

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Colorectal cancer is the third most commonly diagnosed malignancy in the world and the second leading cause of cancer death in the United States. While a range of novel active agents has improved the prognosis of patients with colorectal cancer, 50% of advanced colorectal cancer patients die from metastatic disease.

Monitoring treatment efficacy early, within days, can significantly improve patient outcomes. Current diagnostic techniques can take weeks to months to gauge tumor killing drug efficacy. In some cases, treatment may only be effective in 40% of patients, leading to rapidly advancing cancer and higher costs while regrouping to change therapies.

Duramycin, a naturally occurring peptide that binds to phosphatidylethanolamine (PE), has been radiolabeled and used for early imaging of tumor death in animal models.

Professor Sigrid Stroobants, MD, Chair of Nuclear Medicine, U of Antwerp commented "Objective and accurate evaluation of tumor response to therapy is one of the biggest challenges in oncology. Early assessment of therapeutic ineffectiveness can avoid treatment related toxicity and could lead to improved survival through earlier treatment intensification, stopping the ineffective therapy, or starting second-line therapy."

Chris Pak, MTTI President & CEO said, "The most commonly used methods to evaluate the effectiveness of a treatment are morphological and volumetric which cannot see the effects of therapy early. If TDURA optimizes patient treatment, the benefits to the patients and savings to health care systems will be substantial."

Lucence Launches US Validation Study for LiquidHALLMARK® Liquid Biopsy Test for Biomarker Detection in Lung Cancer

On May 11, 2021 Precision oncology company Lucence is currently conducting the first US validation study of its technology, reported that examining the use of the company’s amplicon-based LiquidHALLMARK liquid biopsy assay versus tissue biopsy in detecting guideline-recommended biomarkers in lung cancer (Press release, Lucence, MAY 11, 2021, View Source [SID1234579713]).

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LIQUIK, Liquid Biopsy for Detection of Actionable Genomic Biomarkers in Patients With Advanced Non-Small Cell Lung Cancer, aims to better understand the concordance of LiquidHALLMARK with conventional tissue-based profiling. The prospective study will enroll 200 treatment-naive newly diagnosed metastatic non-squamous non-small cell lung cancer (NSCLC) patients. LIQUIK will compare LiquidHALLMARK with tissue next-generation sequencing (NGS) for mutation profile results in NSCLC patients who have at least one of nine clinically relevant genes— EGFR, ALK, RET, ROS1, NTRK fusions, MET, BRAF, ERBB2 and KRAS—detected by tissue biopsy. LIQUIK has enrolled its first patient and will continue enrollment across 7 study sites nationwide.

"The launch of our first prospective multicenter study in the United States brings us one step closer to advancing precision cancer care for the benefit of patients everywhere," said Dr. Min-Han Tan, founding CEO of Lucence. "Building evidence to support the clinical utility and sensitivity of our test will enable us to make LiquidHALLMARK’s high resolution, target-rich insights more widely available to patients across the country."

Liquid biopsy holds promise for a range of applications across cancer including non-invasive screening, biomarker detection, treatment monitoring, and testing for minimal residual disease. For non-small cell lung cancer, liquid biopsy is a NCCN guideline-recommended option for testing in cases where a tissue biopsy is not medically feasible or when there is insufficient material for molecular analysis.

LiquidHALLMARK is a comprehensive, amplicon-based NGS assay for ultrasensitive biomarker detection. Powered by AmpliMark, a proprietary sequencing technology, LiquidHALLMARK examines plasma circulating tumor DNA mutations in 80 genes, including fusions in 10 genes. LiquidHALLMARK provides >99% sensitivity at a detection limit of 0.1% variant allele frequency, and targets single nucleotide variants (including cis-trans), insertions and deletions, copy number variations, microsatellite instability, fusions, and viruses. LiquidHALLMARK targets have been identified in 15 cancers.

AmpliMark is the foundational technology in Lucence’s liquid biopsy tests. AmpliMark uses a unique molecular barcode and error-correction technology designed to improve liquid biopsy test sensitivity for single nucleotide variants and fusion genes.

Late last year Lucence’s Palo Alto laboratory received certification from the U.S. Department of Health and Human Services’ Centers for Medicare & Medicaid Services under the Clinical Laboratory Improvement Amendments (CLIA) of 1988. LiquidHALLMARK is currently available to US oncologists as a laboratory developed test.

BostonGene Announces Cancer Research Collaboration with the Abramson Cancer Center of the University of Pennsylvania

On May 11, 2021 BostonGene Corporation, a biomedical software company committed to defining optimal precision medicine-based therapies for cancer patients, reported a Master Clinical Research Collaboration Agreement with the Abramson Cancer Center (ACC) of the University of Pennsylvania to support multiple research projects at the cancer center (Press release, BostonGene, MAY 11, 2021, View Source [SID1234579712]).

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The first research project to arise from this collaboration aims to support clinical research focusing on personalized cancer vaccines, a new approach of active immunotherapy which utilizes the patient’s own immune system to identify tumor specific neoantigens. BostonGene’s advanced computational algorithms will identify cancer specific neoantigens and profile the immune activation status of the tumor by performing advanced multi-omics analysis, including the interpretation and visualization of cancer patient’s genomic, transcriptomic and imaging datasets. The analysis includes the identification of targetable molecular alterations, evaluation of gene expression and gene signatures, characterization of cellular components in the tumor microenvironment, estimation of tumor heterogeneity, prediction of neoantigens and tumor clonality.

"BostonGene’s strategy is to revolutionize medicine in the quest to identify better, personalized treatment options with successful outcomes," said Andrew Feinberg, President and CEO at BostonGene. "We are pleased to support the Abramson Cancer Center by providing sophisticated analytics and integration of scientific and clinical knowledge in an effort to improve the standard of care and redefine the treatment selection approach for cancer patients."

The ACC, a global leader in basic, translational, clinical, and biomedical research for the advancement of cancer care, is a matrix cancer center embedded within the University of Pennsylvania and the University of Pennsylvania Health System. The ACC, a National Cancer Institute-Designated Comprehensive Cancer Center, is comprised of cancer specialists committed to offering cancer patients the newest and most innovative therapeutic advances.

"We look forward to using BostonGene’s technology to help in our work to better understand the mechanisms of cancer neoantigen recognition and to the discovery of new immunotherapy treatment options," said Gerald Linette, MD, PhD, a professor of Medicine in the Perelman School of Medicine at the University of Pennsylvania and Clinical Director of the Parker Institute for Cancer Immunotherapy at Penn. "We are excited about this collaboration, and with our combined expertise have an opportunity to make a profound impact on how cancer patients are treated in the future."

AUM Biosciences enters in a Strategic Collaboration with Handok and CMG Pharm to develop ‘CHC2014’, New Pan-TRK Inhibitor for Cancer Treatment

On May 11, 2021 AUM Biosciences ("AUM"), a global, clinical stage biotechnology company focused on discovering, acquiring and developing next generation targeted oncology therapeutics reported it has entered into a strategic collaboration agreement with Handok Inc [002390: KOSPI] ("Handok") and CMG Pharmaceutical Co. Ltd [KOSDAQ: 058820] ("CMG") for the worldwide (ex-Korea) development, manufacturing and commercialisation rights of a highly specific, safe and efficacious Pan-TRK inhibitor (Press release, AUM BioSciences, MAY 11, 2021, View Source [SID1234579711]). Financial terms of the agreement were not disclosed.

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CHC2014 is completing a Phase I in Korea and has shown promising data to justify advancement to a tumor agnostic registrational program. CHC2014 has shown superior in-vivo anti-tumor activity compared to available treatment options. It has shown strong efficacy on solvent front and gatekeeper mutations addressing a unique solution to the current challenge in TRK Inhibitor market.

"We are glad to enter in a partnership with AUM to advance the global development of innovative targeted cancer therapy CHC2014, which is being co-developed by Handok-CMG-NOV and was successfully studied in phase 1 trial in Korea. After successful development of CHC2014 by AUM management with global capability, we expect that CHC2014 can help improving the lives of patients with rare cancers" commented by Young-Jin Kim, Chairman and CEO of Handok, Inc.

"CHC2014, once developed as a new pan-TRK inhibitor, will provide another solution to cancer patients with TRK-fusion", and "We expect AUM Biosciences specializing for target-specific anti-cancer small molecules to successful global development and commercialization of CHC2014 for the patients", said Mr. Joohyung Lee, CEO of CMG Pharmaceutical.

AUM "portfolio-model" strategy has made significant progress since its establishment through strategic collaborations and partnerships with leading academic institutions and pharmaceutical and biotech companies globally.

"We thank Handok and CMG for its trust in AUM and its continued support in further developing the CHC2014 asset worldwide. We are very encouraged by the clear scientific and clinical differentiation that CHC2014 presents as compared to the other TRK inhibitors" commented Vishal Doshi, CEO, AUM Biosciences. "We are delighted to onboard Handok and CMG as a strategic investor in AUM"