On December 7, 2023 Mission Bio, a leader in single-cell multiomic solutions for precision medicine, reported new data generated by clinical researchers from leading biopharma companies and top academic institutions will be showcased at the 65th ASH (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Mission Bio, DEC 7, 2023, View Source [SID1234638272]). This announcement follows on the heels of the successful launch of the company’s scMRD Assay, to which the latest data on 48 samples from the Clínica Universidad de Navarra will be featured in a poster presentation at the conference. The widespread adoption of Tapestri for hematological research and therapeutic development signifies the importance of single-cell multi-omic data and indicates a potential new gold standard in precision medicine.

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At the American Society of Hematology (ASH) (Free ASH Whitepaper) conference, being held in San Diego on December 9-12, world-leading scientists will present results demonstrating Tapestri’s capabilities to uncover critical insights into clonal dynamics, therapeutic response mechanisms, and clones driving disease persistence and relapse, reflecting the platform’s unique capabilities and position in the market.

Included among the presentations is the first data demonstrating the potential actionability of Mission Bio’s Tapestri Single-Cell MRD (scMRD) AML Multiomics Assay, which will be presented from the lab of Felipe Prosper, MD, PhD, Co-director of the Hematology and Cell Therapy Unit at the Clínica Universidad de Navarra, highlighting its feasibility for assessing MRD in AML patients.

"Understanding the genetic heterogeneity at the single-cell level is crucial to the discovery and development of safer and more effective treatments for blood cancers," said Todd Druley, MD, PhD, Chief Medical Officer of Mission Bio. "We’re excited to see so many of our customers leverage Tapestri, validating the scientific merit of our technology, demonstrating its practical application in a clinical setting, and substantiating the impact of single-cell data to potentially transform care by guiding more personalized treatments in AML, multiple myeloma (MM), and other blood cancers."

Among the ASH (Free ASH Whitepaper) presentations, researchers from Dr. Prosper’s lab will present results demonstrating the concordance between Mission Bio’s scMRD assay and the gold-standard techniques for MRD, such as PCR and multiparameter flow cytometry (MFC), in AML patient samples. The data suggest the scMRD Multiomics Assay’s potential role in advancing targeted therapeutic approaches and its influence on treatment paradigms in the future.

In the presentation, "Minimal Residual Disease Detection By Single Cell DNA Sequencing Technology: A Feasible Approach for Clinical Application and Identification of the Landscape of MRD Clones," the researchers showed that Mission Bio’s scMRD AML Multiomics Assay:

Identified rare leukemic clones that were missed by conventional assays.
Distinguished clonality of clinically actionable mutations, compared to MFC and PCR data, which only designated MRD status.
Detected MRD-positive patients, which were deemed negative by MFC and PCR,
Differentiated between clonal hematopoiesis (CH) clones and leukemic clones.
"Patients suffering from relapsed AML have poor overall survival, necessitating more effective methods for MRD monitoring and detection to better understand the genomic landscape of clonal evolution," said Dr. Prosper. "This impressive data is a significant step towards demonstrating the feasibility of Mission Bio’s assay to detect and monitor MRD in AML patients, to ultimately enable more effective therapeutic strategies, including combination and targeted therapies, based on insights from single-cell DNA and protein multiomics for improved patient outcomes in the future."

These results follow Mission Bio’s successful commercial launch of the scMRD AML Multiomics Assay, designed to offer deep genotypic and immunophenotypic insights into MRD signatures within individual cells.

"Multiple Myeloma therapy, similarly to other blood cancers like AML, remains a challenge and huge unmet medical need because of the poor prognosis and high relapse rate. Tapestri’s ability to combine SNVs, CNVs, and surface protein expression at a single-cell level will be crucial to myeloma surveillance and understanding clonal evolution, and our ability to fully realize precision medicine," said Cedric Dos Santos, PhD, Director AML/MDS/MM, Translational Medicine, at Genentech.

Data from other blood cancer studies will be presented by leading researchers in over 24 presentations leveraging Mission Bio’s Tapestri Platform, showcasing its broad application in hematology. These studies from institutions including Genentech, Merck, MD Anderson Cancer Center, Broad Institute of MIT and Harvard, and St. Jude Children’s Research Hospital embody Mission Bio’s commitment to advancing hematological research. For a complete list of ASH (Free ASH Whitepaper) presentations using Tapestri, please visit our website.

On December 7, 2023 BostonGene, a leading provider of AI-driven molecular and immune profiling solutions, reported the selection of four abstracts for oral presentation, four abstracts as poster presentations, and two abstracts for online publication at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 9-12, 2023, in San Diego, California and virtually (Press release, BostonGene, DEC 7, 2023, View Source [SID1234638271]). BostonGene will exhibit in booth #2350.

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"We look forward to showcasing compelling data at ASH (Free ASH Whitepaper), which will underscore the pivotal role of molecular and immune profiling alongside advanced analytics in propelling precision medicine for individuals battling cancer," said Nathan Fowler, MD, Chief Medical Officer at BostonGene.

Details of the presentations are below:

Oral presentations:

Abstract: 601
Title: Clinical Outcomes of Patients with Relapsed/Refractory Follicular Lymphoma Treated with Tisagenlecleucel: Phase 2 Elara 3-Year Follow-up
Date & time: Sunday, December 10, 2023 | 4:30 PM
Location: Room 6CF – San Diego Convention Center
Presenter: Stephen J Schuster, MD, Abramson Cancer Center, University of Pennsylvania

This presentation highlights results from the Phase 2 ELARA trial that show efficacy, safety, pharmacokinetic, and exploratory biomarker analyses in relapsed/refractory follicular lymphoma patients treated with tisagenlecleucel after a median follow-up of more than 3 years. Higher baseline levels of CD8+ naive T cells are associated with improved long-term clinical outcomes.

Research conducted in collaboration with The University of Texas MD Anderson Cancer Center, Abramson Cancer Center, University of Pennsylvania, The University of Melbourne, Hospital Universitario, Oslo University Hospital, Royal Brisbane Hospital, University of Michigan, City of Hope National Medical Center, Moffitt Cancer Center, Hospices Civils de Lyon and Claude Bernard Lyon 1 University, Kyushu University Hospital, Tohoku University Graduate School of Medicine, University of Amsterdam, University of California San Francisco, University of Chicago, Royal Prince Alfred Hospital, Universidad de Sevilla, Oregon Health and Science University, University of Cologne, West German Cancer Center, University of Duisburg-Essen, IRCCS San Raffaele Scientific Institute, Hokkaido University, King’s College London, University of Kansas Medical Center, Ordensklinikum Linz Barmherzige Schwestern-Elisabethinen, Universitair Ziekenhuis Gent, University Hospital of Ulm, University of Bologna, Cambridge University, Novartis Pharmaceuticals Corporation, Novartis Pharma AG, Hôpital Saint-Louis, Ludwig-Maximilians-University Hospital

Abstract: 851
Title: A CD5 Gene Signature Identifies Diffuse Large B-Cell Lymphomas Sensitive to Bruton’s Tyrosine Kinase Inhibition
Date & time: Monday, December 11, 2023: 3:45 PM
Location: Room 6DE – San Diego Convention Center
Presenter: Alan Cooper, University of Chicago

This study highlights the discovery of a novel transcriptomic-based biomarker of BTK inhibitors (BTK-i) response in DLBCL patients. The biomarker, a CD5 gene signature, expands upon existing genetic DLBCL classifications by identifying DLBCL patients that can benefit from BTK-i, warranting further prospective validation in BTK-i clinical trials.

Research conducted in collaboration with the University of Chicago, City of Hope National Medical Center, The University of British Colombia, Janssen Research & Development, Centre for Lymphoid Cancer

Abstract: 905
Title: Machine Learning (ML)-Enabled Automation for High-Throughput Data Processing in Flow Cytometry
Date & time: Monday, December 11, 2023 | 3:45 PM
Location: Room 6CF – San Diego Convention Center
Presenter: Anna Kamysheva, MSc, BostonGene

This presentation describes BostonGene’s development of an ML-based algorithm for automated cell-type labeling that enables the detection of rare and/or new cell populations with high speed and accuracy. This algorithm is applicable in clinical settings, particularly for detecting hematological abnormalities and cancers.

Abstract: 983
Title: Addition of Acalabrutinib to Lenalidomide and Rituximab Induces High Complete Response Rates in Patients with Previously Untreated Follicular Lymphoma: Results of a Phase II Study
Date & time: Monday, December 11, 2023 | 5:30 PM
Location: Grand Hall C – Manchester Grand Hyatt San Diego
Presenter: Paolo Strati, MD, MD Anderson

The study demonstrates the safety and efficacy of lenalidomide and rituximab treatment in combination with acalabrutinib in previously untreated FL patients and highlights the use of bulk RNA sequencing with deconvolution to analyze the biological effects of this treatment combination on peripheral blood immune cells.

Research conducted in collaboration with The University of Texas MD Anderson Cancer Center

Poster presentations:

Abstract: 1676
Title: Preliminary Analysis of an Ongoing Phase II Study of Response-Adapted Therapy with Copanlisib and Rituximab for Untreated Follicular Lymphoma
Date & time: Saturday, December 9, 2023 | 5:30 PM – 7:30 PM
Location: Halls G-H – San Diego Convention Center
Presenter: Rahul Lakhotia, MBBS, National Cancer Institute, National Institutes of Health

This study describes the preliminary analysis (including safety and efficacy) of an ongoing "window of opportunity" study of copanlisib, followed by response-adapted copanlisib and rituximab in treatment-naïve patients with follicular lymphoma (FL). Gene expression profiling was used to identify high-risk patients and find a predictive signature of response to PI3K inhibitor.

Research conducted in collaboration with the National Cancer Institute, National Institutes of Health and Mayo Clinic

Abstract: 3644
Title: A Novel Comprehensive Tumor-Informed Plasma cfDNA Assay to Monitor Minimal Residual Disease for Hematological and Solid Malignancies
Date & time: Sunday, December 10, 2023 |6:00 PM – 8:00 PM
Location: Halls G-H – San Diego Convention Center
Presenter: Anastasiya Yudina, MSc, BostonGene

This presentation describes our cell-free DNA (cfDNA) testing platform for minimal residual disease (MRD) monitoring. It encompasses all major clinically reliable genetic hotspots events and can detect clinically relevant mutations in plasma samples before clinical manifestation.

Abstract: 3001
Title: Genomic and Transcriptomic Profiles of Blastoid and Pleomorphic Mantle Cell Lymphoma Are Distinct from Classic Histology Mantle Cell Lymphoma
Date & time: Sunday, December 10, 2023 |6:00 PM – 8:00 PM
Location: Halls G-H – San Diego Convention Center
Presenter: Preetesh Jain, MD, MBBS, PhD, DM, MD Anderson

This presentation highlights the use of BostonGene’s integrated genomic and transcriptomic analysis platform to provide a comprehensive portrait of the molecular differences between blastoid and pleomorphic mantle cell lymphoma (MCL), revealing mechanistic insights that can be used for the development of more effective therapeutic strategies.

Research conducted in collaboration with The University of Texas MD Anderson Cancer Center

Abstract: 4872
Title: Long-Term Durable Responses in Relapsed/Refractory (r/r) ALL, DLBCL, and FL Patients Treated with Tisagenlecleucel and Its Association with Persistence of CAR T-Cells
Date & time: Monday, December 11, 2023: 6:00 PM – 8:00 PM
Location: Halls G-H – San Diego Convention Center
Presenter: Rakesh Awasthi, PhD, Novartis Institutes for BioMedical Research

This study highlights a positive association between chimeric antigen receptor (CAR) persistence in peripheral blood post tisagenlecleucel (tisa-cel) infusion and durable clinical responses across three indications.

Research conducted in collaboration with Novartis Institutes for BioMedical Research, Novartis Pharmaceuticals Corporation, Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Intermountain Primary Children’s Hospital, Huntsman Cancer Institute, Spencer Fox Eccles School of Medicine at the University of Utah, Goethe University Frankfurt, University Hospital, Knight Cancer Institute, Oregon Health & Science University, Emory University School of Medicine, Medical University of Vienna, Université de Paris, Klinikum der Universität, LMU München, MD Anderson Cancer Center

Online publications:

Title: Test-the-Test: Clinical Utility of Comprehensive Whole Exome Sequencing and RNA-Seq for Patients with Lymphoma

This study shows an acceptable turn-around-time (TAT) of 8 days for the delivery of clinical reports that include clinically relevant findings and matched clinical trials, demonstrating the feasibility of using comprehensive WES and RNA-seq molecular profiling for lymphoma patients.

Research conducted in collaboration with The University of Texas MD Anderson Cancer Center

Title: Molecular Findings on Plasma Cell-Free DNA Analysis Among Adults with Histiocytic Neoplasms

This study describes the use of cell-free DNA (cfDNA) analysis to evaluate the molecular findings in adults with histiocytic neoplasms, highlighting cfDNA as a potential viable tool for discovering driver mutations in multiple cancers.

Research conducted in collaboration with The University of Alabama, Mayo Clinic

In addition to the poster presentations, the abstracts have been published online in the November supplemental issue of "Blood."

For more information, please visit the 65th ASH (Free ASH Whitepaper) Annual Meeting and Exposition website at View Source

ON December 7, 2023 Remix Therapeutics (Remix), a biotechnology company developing small molecule therapies designed to modulate RNA processing and address underlying drivers of disease, reported an upcoming presentation introducing REM-422, the Company’s potent, selective, oral small molecule messenger RNA (mRNA) degrader, as a potential treatment for MYB-driven cancers (Press release, Remix Therapeutics, DEC 7, 2023, View Source [SID1234638270]). Data describing the activity of REM-422 in AML xenograft models will be highlighted at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper), taking place from December 9-12, 2023 in San Diego. Results demonstrate oral dosing of REM-422 degrades MYB mRNA, inducing anti-leukemic activity in multiple xenograft models.

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"These compelling data support the therapeutic rationale of targeting MYB with REM-422 as a potential treatment for acute myeloid leukemia (AML) and myelodysplastic syndromes," said Peter Smith, Ph.D., President and Chief Executive Officer of Remix Therapeutics. "REM-422 is the first compound entering the clinic from the REMaster discovery platform and we look forward to the start of clinical development in patients with MYB-dysregulated malignancies."

Lead author Charles Kung, PhD, Vice President of Biology, concludes "MYB is an important oncogenic transcription factor that is dysregulated in the majority of AML and MDS patients and we look forward to bringing REM-422 to patients in need of new treatment options."

Details for the poster presentation are as follows:

Title: REM-422, a potent, selective, oral small molecule mRNA degrader of the MYB oncogene, demonstrates anti-tumor activity in mouse xenograft models of AML
Abstract Number: 1425
Session Name: Poster Session I – Presentations
Session Date and Time: Saturday, December 9, 2023, 5:30 PM-7:30 PM
Session Location: Poster Hall

On December 7, 2023 Marengo Therapeutics, Inc., a clinical-stage biotech company pioneering a new way to activate T cells by targeting germline-encoded alleles in the Vβ chain of the T cell receptor (TCR) to selectively activate the right T cell subsets to fight cancer, reported a publication in Science Advances describing the novel immunological effects of its foundational anti-Vβ chain TCR targeting antibodies on human T cells (Press release, Marengo Therapeutics, DEC 7, 2023, View Source [SID1234638269]). This unique approach underpins Marengo’s therapeutic T cell platform, which has broad application across a range of diseases. The publication describes observations and insights gained through a collaboration between Marengo scientists and the Kings College London-based lab of Adrian Hayday, Ph.D. FmedSci, FRS.

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"Professor Hayday and his team are true pioneers in advancing the field of innate TCR biology, particularly in the area of gamma delta T cells," said Andrew Bayliffe, Ph.D., Chief Scientific Officer of Marengo. "Through this collaboration, we have extended the concept of innate TCR engagement to αβ T cells. The resulting learnings will be crucial as we progress our platform and pipeline of broad Vβ T cell therapeutics."

The publication, titled "Innate TCRβ-chain Engagement Drives Human T cells Toward Distinct Memory-like Effector Phenotypes with Immunotherapeutic Potentials," highlights the distinct binding modes of Marengo’s Vβ chain-targeting antibodies and their effects on differentiation states of human T cells. Using a battery of immunophenotyping and molecular approaches, researchers in Professor Hayday’s Immunobiology Lab at King’s College London revealed a unique signature induced in subsets of human T cells treated with Vβ chain-targeting antibodies.

Link to publication: View Source

This population of cells show hallmarks of both proliferative memory T cells, and effector T cells that are highly distinct to states induced by other non-clonal TCR activators such as anti-CD3e antibodies and Vβ chain‑targeting bacterial superantigens. Further mechanistic investigations revealed diverse transcriptional changes associated with the changes in T cell phenotype that suggest a fundamental reprogramming of T cell differentiation by Vβ chain-targeting antibodies.

"We undertook these studies to tackle a previously unanswered question; namely, will TCR∝β respond in a distinct and interesting way to stimulating by an atypical mode, that we term ‘innate’. We have discovered that the answer is clearly yes, and we are understandably delighted for cancer patients that this innate response mode may prove therapeutically beneficial," said Professor Hayday.

This publication follows on the heels of a paper published November 29 in the journal Science Translational Medicine, which further underscores the potential of Marengo’s lead asset STAR0602 and the company’s STAR platform to target and activate subsets of T cells expressing different β chain TCRs.

On December 7, 2023 ARTBIO, Inc. (ARTBIO), a clinical-stage radiopharmaceutical company developing a new class of targeted alpha radioligand therapies (ARTs), reported the closing of an oversubscribed and upsized $90 million Series A financing co-led by Third Rock Ventures and an undisclosed healthcare fund (Press release, ARTBIO, DEC 7, 2023, View Source [SID1234638268]). Additionally, seed lead investors F-Prime Capital and Omega Funds participated substantially. The company’s prior seed investment round of $23 million was announced in June 2023.

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Concurrent with the Series A closing, ARTBIO has appointed industry veterans Philippe Dasse, Pharm.D., as Chief Technical Officer and Daniel Rossetto as Head and Senior Vice President of Supply Chain and External Manufacturing. In these roles, Philippe and Daniel will lead the development and expansion of ARTBIO’s unique distributed manufacturing network in support of the company’s clinical pipeline.

"We are thrilled to have the support of our new and existing investors, including Third Rock Ventures, F-Prime Capital and Omega Funds. These groups bring significant expertise in company scale-up and pipeline development that will be invaluable as we continue to progress our programs and pipeline," said Emanuele Ostuni, Ph.D., CEO of ARTBIO. "Next year will be an important one for the company as we advance our lead program, AB001, and our entire pipeline, while further developing a distributed manufacturing network with our AlphaDirect technology. Bringing Philippe and Daniel to the ARTBIO team also deepens our manufacturing expertise to ensure efficient manufacturing and seamless delivery of our novel Pb212 alpha radioligand therapies to patients."

"The foundational work conducted by the stellar team of experts at ARTBIO has established the potential of alpha radioligand therapies and gives us great confidence that this team can revolutionize today’s cancer treatment paradigm," said Jeff Tong, Partner at Third Rock Ventures. "As ARTBIO is in a rapid scale-up phase, we are looking forward to supporting its growth by leveraging our deep expertise in discovery, development and operations."

The appointments of Philippe and Daniel to the management team strengthen the company’s capabilities during this critical stage. In Philippe’s role as Chief Technical Officer, he will lead the development of ARTBIO’s proprietary AlphaDirect technology and establishment of the company’s distributed manufacturing network. Philippe was most recently Head of Technical Operations for Radioligand Therapies at Novartis Oncology. Philippe was also the first employee of Advanced Accelerator Applications in 2002, a company acquired by Novartis in 2018, and covered increasing responsibilities to lead all technical operations before his departure.

"I am excited to join this talented and passionate team to help build the next wave of innovation in radiopharmaceuticals by pushing the boundaries of the platform and creating a new manufacturing paradigm that leverages my past experiences and adapts to the demands of short-lived alpha emitters," said Dr. Dasse.

In Daniel’s role as Head and Senior Vice President of Supply Chain and External Manufacturing, he will lead efforts to strengthen the company’s supply chain logistics and manufacturing partnerships to ensure the seamless distribution of ARTs. Daniel has a long career in pharmaceutical manufacturing and was most recently the Global Head of Supply Chain at Advanced Accelerator Applications, Novartis. In this role, Daniel managed a diverse team to scale up a high-speed and agile internal and external supply network to deliver the clinical and commercial RLT portfolio which included PLUVICTO to prostate cancer patients.

"The strong Series A funding is a clear sign of the differentiated value proposition and potential of the ARTBIO platform and a recognition of the fast progress that the team has made," said Ted Love, M.D., ARTBIO Board Chairman and Biotechnology Innovation Organization’s (BIO) Board Chairman. "We are grateful to attract such high-quality investors and associated board members whose deep experience in drug discovery and development as well as company building will serve us well as we continue driving our novel pipeline forward."