On August 25, 2020 CARISMA Therapeutics Inc., a biopharmaceutical company focused on discovering and developing innovative immunotherapies, reported it has entered into a scientific research and licensing agreement with Nathaniel R. Landau, PhD and NYU Langone Health through which CARISMA will attain exclusive rights to develop and commercialize their Vpx lentiviral vector globally for all indications (Press release, Carisma Therapeutics, AUG 25, 2020, View Source [SID1234564020]). The Vpx lentiviral and CARISMA’s Ad5f35 vectors are the only two vectors known to be effective in engineering macrophages, a pivotal aspect of CARISMA’s approach.

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The Vpx lentiviral vector, which is proven to be highly effective at transducing human monocytes, macrophages and dendritic cells, will broaden the utility of the CARISMA-engineered monocyte and macrophage platforms by enabling integration of transgenes for long-term, durable expression.

"CARISMA is committed to pursuing science and partnerships that will build upon and enhance our macrophage technology," said Steven Kelly, President and Chief Executive Officer at CARISMA Therapeutics. "This exciting collaboration with NYU Langone Health and Dr. Landau advances CARISMA’s position as the leader in the engineered macrophage space and equips us with additional tools to further unlock the power and potential of the macrophage."

The announcement follows the recent U.S. Food and Drug Administration clearance of an investigational new drug (IND) application for CARISMA’s lead product candidate, CT-0508, an anti-human epidermal growth factor receptor 2 (HER2) targeted chimeric antigen receptor macrophage (CAR-M). Under this IND, CARISMA intends to initiate its Phase 1, first-in-human, multi-center study in patients with recurrent or metastatic HER2 overexpressing solid tumors after failure of approved HER2 targeted agents later this year.

"We are excited to collaborate with CARISMA on this project. We believe that that the Company’s commitment to immunotherapy and its achievements to date in macrophage engineering will bring important advances in the treatment of cancer," said Dr. Landau, a Professor in the Department of Microbiology at the NYU Grossman School of Medicine and the inventor of the Vpx lentiviral vector. "The collaboration will advance the field of cancer immunotherapy and bring new understanding of how to engineer macrophages for therapeutic purposes."

On August 25, 2020 M2GEN reported the launch of its oncology-focused Bioinformatics Support Services (Press release, M2Gen, AUG 25, 2020, View Source [SID1234564019]). Leveraging the results and knowledge gained from the ORIEN Avatar Program, an extensive, unique and ever-growing oncology clinico-genomics dataset, the M2GEN Bioinformatics team provides bioinformatics support services adhering to best-in-class standards.

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M2GEN offers the following bioinformatics services: Paired Tumor and Germline Whole Exome Sequencing (WES) analysis with Panel of Normals (PoN) filtering, Tumor-Only Whole Exome Sequencing (WES) analysis with Virtual Normal (VN) somatic mutation classification, and Tumor-Only RNA Sequencing (RNA-Seq) analysis. Pipeline analysis is WES and RNA-Seq capture kit agnostic, and uses the most up to date human genome reference (GRCh38/hg38) and gene build (Gencode V32). All bioinformatics services are performed in HIPAA compliant and HI Trust certified cloud solutions.

M2GEN Bioinformatics Support Services utilize one of the largest Panel of Normals (PoN) available for high-quality somatic mutation calling. Over 20,000 germline samples from cancer patients inform the segregation of somatic and germline variants, and identify recurrent sequencing artifacts. In addition, the Avatar germline sequencing data is leveraged as a Virtual Normal (VN) cohort during Tumor-Only analysis, allowing for superior somatic mutation classification when patients’ matched normal biospecimens are unavailable.

M2GEN’s Bioinformatics Support Services are led by an experienced team of bioinformatics and computational biology experts trained at world-renowned scientific institutions such as Baylor College of Medicine Human Genome Sequencing Center, Memorial Sloan Kettering Cancer Center, European Molecular Biology Laboratory, European Bioinformatics Institute, Wellcome Trust Sanger Institute and Columbia University.

"M2GEN now provides next generation sequencing analysis pipelines to customers in academia and biopharma industry. Our end-to-end bioinformatics solutions and pipelines are tuned for large-scale oncology multi-omics studies that allow our customers to dedicate more time to results interpretation and discovery," said Oliver Hampton PhD, Vice President of Bioinformatics & Biostatistics at M2GEN.

"M2GEN continues to expand its portfolio of data solutions, services, and technologies to be the best partner possible for its clients as we transform cancer care together and make precision medicine a reality," said Helge Bastian, PhD, President & CEO of M2GEN. "M2GEN’s bioinformatics services enable researchers in academic and pharmaceutical research worldwide to gain the most meaningful molecular insights in oncology. These molecular insights will accelerate the development of novel and more effective patient specific therapies, turning cancer into a manageable condition, or better still a curable disease."

On August 25, 2020 Perrigo Company plc (NYSE; TASE: PRGO), a leading global provider of Quality, Affordable Self-Care Products, reported that CEO and President, Murray S. Kessler and CFO, Ray Silcock, will host virtual one-on-one meetings at the Barclay’s Global Consumer Staples Conference on Wednesday, September 9, 2020 (Press release, Perrigo Company, AUG 25, 2020, View Source [SID1234564018]).

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Also, Mr. Kessler will present at the 18th Annual Morgan Stanley Global Healthcare Conference at 8:45 am EST on Thursday, September 17, 2020. Interested parties can access the presentation webcast at View Source

On August 25, 2020 The MDS Foundation reported that a large international study led by researchers at Memorial Sloan Kettering finds that having two mutated copies of the TP53 gene, as opposed to a single mutated copy, is associated with worse outcomes in myelodysplastic syndrome and acute myeloid leukemia (Press release, MDS Foundation, AUG 25, 2020, View Source [SID1234564017]). The findings have immediate clinical relevance for risk assessment and treatment of people with myelodysplastic syndrome.

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Considered the "guardian of the genome," TP53 is the most commonly mutated gene in cancer. TP53’s normal function is to detect DNA damage and prevent cells from passing this damage on to daughter cells. When TP53 is mutated, the protein made from this gene (called p53) can no longer perform this protective function, which can result in cancer. Across many cancer types, mutations in TP53 are associated with much worse outcomes, like disease recurrence and shorter survival.

As with all genes, there are two copies of TP53 in our cells. One copy we get from our mothers, the other we get from our fathers. Until now, it was unclear whether a mutation in one copy of TP53 TP53 was enough to cause worse outcomes, or if mutations in both copies were necessary. A new study led by researchers at Memorial Sloan Kettering definitively answers this question for a blood cancer called myelodysplastic syndrome (MDS), a precursor to acute myeloid leukemia.

"Our study is the first to assess the impact of having one versus two dysfunctional copies of TP53 on cancer outcomes," says molecular geneticist Dr. Elli Papaemmanuil, a member of the Epidemiology and Biostatistics Department at MSK and the lead scientist on the study, whose results were published August 3 in the journal Nature Medicine. "From our results, it’s clear that you need to lose function of both copies to see evidence of genome instability and a high-risk clinical phenotype in MDS."

"The consequences for cancer diagnosis and treatment are immediate and profound," she says.

A LARGE, MULTICENTER STUDY

The study analyzed genetic and clinical data from 4,444 patients with MDS who were being treated at hospitals all over the world. Researchers from 25 centers in 12 countries were involved in the study, which was conducted under the aegis in collaboration with investigators in the International Working Group for Prognosis in MDS (IWG-PM) whose goal is to develop new international guidelines for the treatment of this disease. Findings were independently validated using data from the Japanese MDS working group led by Dr. Seishi Ogawa’s group at Kyoto University.

"Currently, the existing guidelines do not consider genomic data, like TP53 and other acquired mutations, when assessing a person’s prognosis or determining appropriate treatment for this disease," says Dr. Peter Greenberg, Director of Stanford University’s MDS Center, Chair of the National Comprehensive Cancer Network Practice Guidelines Panel for MDS, and a participant in the study. "Studies are ongoing reflecting this need for change."

Tracey Iraca, MDS Foundation Executive Director stated, "This study is important in updating the IPSS-R to include molecule information in light of the more personalized treatments now being explored for MDS patients."

Using new computational methods and the database and collaborative input of the IWG-PM, the investigators found that about one-third of MDS patients had only one mutated copy of TP53. These patients had similar outcomes as patients who did not have a TP53 mutation — that is, good response to treatment, low rates of disease progression, and better survival. Two-thirds of patients, on the other hand, had two mutated copies of TP53. These patients had much worse outcomes — including treatment-resistant disease, rapid disease progression, and low overall survival. In fact, the researchers found that TP53 mutation status — either 0/1 or 2 mutated copies of the gene — was the most important variable when predicting outcomes.

"Our findings are of immediate clinical relevance to MDS patients," Dr. Papaemmanuil says. "Going forward, all MDS patients should have their TP53 status assessed at diagnosis."

As for why it takes two "hits" to TP53 to see an effect on cancer outcomes, Dr. Elsa Bernard, a postdoctoral scientist in the Papaemmanuil lab and the study’s first author, speculates that having one normal copy is enough to provide adequate protection against DNA damage. This would explain why having only one mutated copy was not associated with genome instability or any worse survival over having two normal copies.

Given the frequency of TP53 mutations in cancer, these results argue for examining the impact of one versus two mutations in other cancers as well. They also reveal the need for clinical trials designed specifically with these molecular differences in mind.

"With the increasing adoption of molecular profiling at the time of cancer diagnosis, we need large evidence-based studies to inform how to translate these molecular findings into optimal treatment strategies," Dr. Papaemmanuil says.

On August 25, 2020 Oxford Finance LLC ("Oxford"), a specialty finance firm that provides senior debt to life sciences and healthcare services companies worldwide, reported that on July 15, 2020, it closed a $10 million senior secured term loan with Inhibrx, Inc. (or "the Company") (Nasdaq: INBX), a clinical-stage biotechnology company focused on developing a broad pipeline of novel biologic therapeutic candidates (Press release, Oxford Finance, AUG 25, 2020, View Source [SID1234564016]). The funds, which were secured prior to the Company’s recent initial public offering, will provide general growth capital as Inhibrx aims to evolve into a commercial-stage biotechnology company.

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Inhibrx combines a deep understanding of target biology with innovative protein engineering, proprietary discovery technologies, and an integrative approach to research and development to design highly differentiated therapeutic candidates. The Company has a pipeline of four programs in Phase 1 clinical trials targeting cancer and respiratory diseases.

"This closing represents Oxford’s second opportunity with Inhibrx," said Christopher A. Herr, senior managing director at Oxford. "The Company is steadily advancing in its clinical pipeline, with its broad-based suite of platform technologies the Company can generate antibodies across several therapeutic areas, including oncology and respiratory illnesses. We are thrilled to continue supporting their promising growth and development."