On November 9, 2021 MAIA Biotechnology, Inc., a targeted therapy, immuno-oncology company focused on developing potential first-in-class oncology drugs ("MAIA"), reported that the Company will present a scientific poster at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting (SITC) (Free SITC Whitepaper) which will be held November 10-14, 2021, in Washington, DC (Press release, MAIA Biotechnology, NOV 9, 2021, View Source [SID1234594946]).

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Poster Title:

Telomerase-Driven Telomeric DNA Modification in Cancer Cells Leads to Efficient Induction of cGAS-mediated Innate and Adoptive Immune Responses

Abstract ID:

697

Category:

Immune-stimulants and immune modulators

Date:

Friday, Nov. 12, 2021

Time:

7:00 am – 8:30 pm

Location:

Poster Hall, Walter E. Washington Convention Center

Accepted abstracts can be found in the Journal for ImmunoTherapy of Cancer (JITC).

On November 9, 2021 EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, reported that it has entered into a research collaboration with the University of Wisconsin–Madison as part of the company’s effort of translating proprietary LEAPER RNA editing technology into in vivo therapies (Press release, EdiGene, NOV 9, 2021, View Source [SID1234594945]).

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Under the partnership, EdiGene’s U.S.-based R&D Center will work with the David Gamm Laboratory at the University of Wisconsin-Madison to evaluate the pharmacological property of LEAPER RNA base editing candidates targeting specific genetic diseases.

"EdiGene’s U.S. R&D Center has built up its capabilities with an exceptional team of talented scientists who are advancing the company’s pipeline both within our own labs and through external partnerships," said Bo Zhang, Ph.D., Head of EdiGene’s U.S. subsidiary. "Dr. Gamm’s knowledge and insights are a tremendous asset in this effort."

"This collaboration allows us to leverage our expertise and experience to help translate EdiGene’s RNA base editing technology into transformative medicines that can benefit patients with genetic diseases who today have limited or no therapeutic options," said David Gamm, MD, Ph.D., Principal Investigator of the research, Professor of Ophthalmology and Visual Sciences, and Director of the McPherson Eye Research Institute at the University of Wisconsin–Madison.

LEAPER (Leveraging endogenous ADAR for programmable editing of RNA) employs short engineered ADAR-recruiting RNAs (arRNAs) to recruit native ADAR enzymes to change specific adenosine to inosine. LEAPER is a robust, precise, and efficient RNA editing technology uniquely suited for in vivo therapies with broad therapeutic applicability. It is developed by Professor Wensheng Wei’s lab at Peking University. Professor Wensheng Wei is the Scientific Founder of EdiGene.

"Earlier this year, we announced the first patient enrolled in multicenter Phase I clinical study of our investigational ex vivo gene-editing hematopoietic stem cell therapy ET-01. This collaboration furthers our efforts in RNA editing and in vivo gene-editing therapies," said Dong Wei, Ph.D., CEO of EdiGene. "Along with the expansion of our U.S. R&D Center, such effort will help accelerate the translation of our proprietary gene-editing technology for patients in need worldwide."

About the David Gamm Laboratory

The Gamm laboratory was established at the University of Wisconsin-Madison in 2003 to advance the use of human pluripotent stem cells (hPSCs) in the study and treatment of retinal degenerative diseases. To facilitate these efforts, the Gamm lab developed the first hPSC-based 3D retinal organoid culture method, which has since yielded key insights into mechanisms of early human retinal development. Moreover, their studies have established the authenticity of hPSC-derived retinal progeny, including photoreceptor cells (rods and cones), retinal pigmented epithelium (RPE) cells, and neural retinal tissue. Lastly, they were pioneers in the use of patient-specific and gene-modified iPSCs to model retinal disorders and to test therapeutic strategies, and have advanced efforts to adapt this technology for human use.

On November 9, 2021 Transgene (Paris:TNG), a biotech company that designs and develops virus-based immunotherapeutics against cancer, and BioInvent International AB ("BioInvent") (Nasdaq Stockholm: BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, reported preclinical data supporting the mode of action of BT-001, their novel dual mechanism-of-action oncolytic Vaccinia virus. The data demonstrate high intratumoral expression of an immune checkpoint-inhibiting antibody and robust anti-tumoral activity in several tumor models (Press release, Transgene, NOV 9, 2021, View Source [SID1234594944]).

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BT-001, developed by BioInvent and Transgene, is a clinical phase oncolytic virus engineered to deliver an anti-CTLA-4 antibody and human GM-CSF in a tumor-specific vehicle (the VVcopTK-RR- virus backbone) for the treatment of solid tumors.

The companies’ poster at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (SITC 2021) shows that BT-001 selectively replicates in tumor cells. The murine surrogate of BT-001 delivered sustained and high intratumoral levels of antibody accompanied by low systemic exposure. These differential expression levels were associated with high depletion of intratumoral regulatory T cells (Treg) but the absence of systemic Treg depletion. Similar effects in humans would allow BT-001 to deliver powerful antitumor immunity.

Patient inclusion into the ongoing Phase I/IIa clinical study of BT-001 (NCT04725331) is progressing well. The multicenter trial, authorized in Europe and in the U.S., is assessing BT-001 as single agent and in combination with the PD-1 checkpoint inhibitor pembrolizumab for the treatment of solid tumors. Initial Phase I data are expected in the first half of 2022.

Other data highlighted in the SITC (Free SITC Whitepaper) poster show improved survival in several syngeneic tumor models following treatment with a murine version of BT-001. There is also evidence of a positive synergistic effect between the murine ‘BT-001’ oncolytic virus expressing the CTLA-4 antibody and a systemic PD-1 checkpoint inhibitor.

"These impressive data, demonstrating the multiple mechanisms of action and anti-cancer properties of BT-001, played a key role in our decision to take this unique oncolytic virus into the clinic. We are pleased to be able to share them with our scientific and clinical peers at SITC (Free SITC Whitepaper)" said Martin Welschof, CEO of BioInvent and Hedi Ben Brahim, Chairman and CEO of Transgene.

SITC 2021 will take place on November 10–14, 2021, at the Walter E. Washington Convention Center in Washington, D.C. and virtually. The poster, entitled "Vectorized Treg-depleting aCTLA-4 elicits antigen cross-presentation and CD8+ T cell immunity to reject "cold" tumors", will be presented on the Virtual ePoster Hall and presented in the Poster Hall (Hall E) on Saturday, November 13, 2021.

Authors: Monika Semmrich, Jean-Baptiste Marchand, Matilda Rehn, Laetitia Fend, Christelle Remy, Petra Holmkvist, Nathalie Silvestre, Carolin Svensson, Patricia Kleinpeter, Jules Deforges, Fred Junghus, Linda Mårtensson, Johann Foloppe, Ingrid Teige, Eric Quéméneur and Björn Frendéus.

Abstract and poster number: 746

About BT-001
BT-001 is an oncolytic virus generated using Transgene’s Invir.IO platform and its patented large-capacity VVcopTK-RR- oncolytic virus, which has been engineered to encode both a Treg-depleting human recombinant anti-CTLA-4 antibody generated by BioInvent’s proprietary n-CoDeR/F.I.R.S.T platforms, and the human GM-CSF cytokine. By selectively targeting the tumor microenvironment, BT-001 is expected to elicit a much stronger and more effective antitumoral response. As a consequence, by reducing systemic exposure, the safety and tolerability profile of the anti-CTLA-4 antibody will be greatly improved.

BT-001 is being co-developed as part of a 50/50 collaboration on oncolytic viruses between Transgene and BioInvent. To know more on BT-001, watch our video here.

On November 9, 2021 Palleon Pharmaceuticals, a company pioneering the field of glyco-immunology to treat cancer and inflammatory diseases, reported data on a novel mechanism of action of the company’s EAGLE sialoglycan degradation therapeutic platform (Press release, Palleon Pharmaceuticals, NOV 9, 2021, View Source [SID1234594943]). Preclinical studies, presented in a poster at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), show that EAGLE therapeutic candidates’ desialylation of T cells enhances T cell anti-tumor immunity, and that lead candidate E-602 (Bi-Sialidase) is efficacious and safe in animal models.

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"The exciting package of data we are presenting at SITC (Free SITC Whitepaper) demonstrates for the first time that Palleon’s EAGLE platform could offer a novel immunomodulatory approach to enhancing T-cell immunity for cancer treatment," said Li Peng, Ph.D., Chief Scientific Officer of Palleon and the poster’s principal author. "These findings will bolster our upcoming IND filing for E-602 and continue to deepen our understanding of the therapeutic potential of the EAGLE platform."

Upregulation of sialoglycans on tumors has been observed for decades and correlates with poor clinical outcomes across many tumor types. Recent findings made possible by advances in the tools used to study glycobiology have shown that these sialoglycans are immunosuppressive. In addition to tumor cells, most immune cells present substantially more abundant sialoglycans than non-hematological healthy cells, which may also contribute to immunosuppression. Palleon utilized various assays to study the effect of E-602 on naïve, exhausted, and effector T cells. These studies found that desialylation by E-602 enhanced naïve T cell priming/activation, restored exhausted-like T cell functions, and enhanced effector T cell function.

Further studies evaluated the single-agent antitumor activity of E-602 in multiple syngeneic mouse tumor models, and its safety profile in rat and non-human primate models. These studies found that E-602 demonstrated single-agent antitumor activity and a wide safety margin.

On November 9, 2021 Applied DNA Sciences, Inc. (NASDAQ: APDN) (Applied DNA or the "Company"), a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing and nucleic acid-based technologies, reported the publication of a study in Molecular Therapy: Methods and Clinical Development on a methodology for the manufacture of novel types of CAR constructs that employ the Company’s LinearDNA as part of a manufacturing process for the efficient generation of CD19-specific CAR T-cells (CAR19 T-cells) based on co-electroporation of a LinearDNA transposon and mRNA encoding of piggyBac transposase (Press release, Applied DNA Sciences, NOV 9, 2021, View Source [SID1234594942]). PCR-produced LinearDNA is manufactured by LineaRx, the Company’s majority-owned subsidiary, to serve as a pure, fast, and flexible alternative to plasmid DNA (pDNA) for biotherapeutic applications.

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The study, titled "Enzymatically produced piggyBac transposon vectors for efficient non-viral manufacturing of CD19-specific CAR T cells", details the utility of LinearDNA in the cost-effective production of preclinical CAR T cells. Its authors, members of the Institute of Hematology and Blood Transfusion (ÚHKT) in Prague, Czechia, and the Faculty of Natural Sciences at Charles University, also in Prague, propose that the combination of LinearDNA and a transposon/transposase system offers therapy developers an effective research tool for making experimental CAR T cells rapidly and efficiently without the need for complicated virus production or the use of pDNA.

Pavel Otáhal, contributing author and Head of the Gene Immunotherapy Research Department at ÚHKT, stated, "Our study compares the manufacture of CAR19 T-cells via PCR-made transposon DNA (LinearDNA) with mRNA encoding of the transposase against a conventional plasmid approach. We found CAR T efficacy of the LinearDNA system versus the plasmid system to be identical. Further, we found no mutations in the coding sequence of LinearDNA and with >99% purity that obliviated the need for purification typical of a plasmid approach. As an institution dedicated to diagnosing and treating serious blood diseases and with the ability to pursue investigational medicines from development to manufacture and clinical trial for patients who have exhausted all approved treatment options, having a cost-effective and rapid production chain is integral to ÚHKT’s mission. We find LinearDNA to be an excellent platform for CAR T-cell therapy development."

Dr. James A. Hayward, president and CEO of Applied DNA, said, "The clinical successes of CAR T-cell therapy against blood cancers have been impressive, though limited by the complex production of viral vectors that are currently needed for T-cell genetic transformation and the use of pDNA. These manufacturing complexities have likewise hindered research on CAR T-cell therapies. As described in the publication, the use of LinearDNA, coupled with non-viral transfection systems, we believe, overcomes many of the existing manufacturing complexities associated with pDNA and viral vectors, thereby offering therapy developers a rapid and cost-effective tool for manufacturing preclinical CAR T cells. The authors’ findings as it relates to LinearDNA coincide with the industry’s growing interest in alternatives to pDNA for CAR T-cell therapies with approximately 50% of recent CRO orders coming from CAR T cell developers."

The detailed study write-up can be found at: Molecular Therapy: Methods and Clinical Development, the leading journal for research in the areas of gene transfer, vector development and design, stem cell manipulation, development of gene-, peptide-, protein-, oligonucleotide-, and cell-based therapeutics to correct genetic and acquired diseases, vaccine development, preclinical target validation, safety/efficacy studies, and clinical trials.

About LineaRx, Inc., and LinearDNA

LineaRx seeks to commercialize the biotherapeutic value of Applied DNA’s deep expertise and experience in the design, manufacture, and chemical modification of DNA by large-scale polymerase chain reaction ("PCR") via its LinearDNA Platform.

The LinearDNA Platform is a proprietary large-scale PCR-based manufacturing platform that allows for the large-scale production of specific high-fidelity DNA sequences. Unlike plasmid-derived DNA, LinearDNA is free of adventitious DNA sequences and can be chemically modified to optimize the DNA for specific applications. The LinearDNA platform is currently being used by customers to manufacture DNA as components of in vitro diagnostic tests and for preclinical nucleic acid-based drug development in the fields of adoptive cell therapies (CAR T and TCR therapies), DNA vaccines (anti-viral and cancer), RNA therapies, clustered regularly interspaced short palindromic repeats-based (CRISPR) therapies and gene therapies, as well as the Company’s COVID-19 veterinary vaccine candidate.