Cell and Gene Therapy

AbbVie and Caribou Biosciences Announce Collaboration and License Agreement for the Research and Development of CAR-T Cell Therapeutics

AbbVie | February 11, 2021

AbbVie and Caribou Biosciences, Inc., a leading clinical-stage CRISPR genome altering biotechnology organization, reported today that they have gone into a cooperation and permit arrangement for the innovative work of fanciful antigen receptor (CAR)- T cell therapeutics. Albeit allogeneic, "off-the-shelf" CAR-T cell therapies have indicated early guarantee in some malignancy patients, the requirement for beating the dismissal of allogeneic CAR-T cells by the host immune system stays a vital test to their more extensive turn of events. Utilizing Caribou's CRISPR genome editing platform to engineer CAR-T cells to withstand have resistant assault would empower the advancement of the next-generation of "off-the-shelf" cellular therapies to benefit a broader patient population.

"We are excited to partner with AbbVie on the development of new CAR-T cell therapies. This collaboration validates Caribou's differentiated next-generation CRISPR genome editing technologies that provide best-in-class efficiency and specificity," said Rachel Haurwitz, Ph.D., President and Chief Executive Officer of Caribou. "We believe AbbVie is an ideal partner for Caribou as we expand upon the number of targets and diseases addressable by our technologies. Genome-edited CAR-T cell therapies hold tremendous potential for patients, and this partnership accelerates our ability to address significant unmet medical need."

About AbbVie
AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio.

About Caribou Biosciences, Inc.
Caribou is a leading clinical-stage CRISPR genome editing biotechnology company founded by pioneers of CRISPR biology. Outside of this collaboration, Caribou is advancing an internal pipeline of allogeneic cell therapies for oncology. CB-010, Caribou's lead allogeneic CAR-T cell program, targets CD19 and is being evaluated in a Phase 1 clinical trial for patients with relapsed/refractory B cell non-Hodgkin lymphoma. CB-011, Caribou's second allogeneic CAR-T cell therapy, targets BCMA for multiple myeloma, and CB-012, Caribou's third allogeneic CAR-T cell therapy, targets CD371 for acute myeloid leukemia. CB-011 and CB-012 are in preclinical development. Additionally, Caribou is developing iPSC-derived allogeneic natural killer (NK) cell therapies for solid tumors. Through its next-generation CRISPR genome editing technologies, Caribou is implementing multiple strategies to boost CAR-T and NK cell persistence to overcome cell exhaustion and to prevent rapid immune-mediated clearance. These sophisticated edits drive the durability of clinical benefit of these off-the-shelf medicines.

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MedTech

Watchmaker Genomics Announces Co-Exclusive License to Disruptive DNA Methylation Technology and Strategic Supply Agreements with Exact Sciences

businesswire | August 31, 2023

Watchmaker Genomics, a supplier of innovative products for molecular analysis, today announced a multi-year, co-exclusive agreement with Exact Sciences Corporation, a leading provider of cancer screening and diagnostic tests, to develop and commercialize the breakthrough DNA methylation analysis technology, TET-assisted pyridine borane sequencing (TAPS). Watchmaker will apply its expertise in engineering DNA-modifying enzymes to further improve the TAPS chemistry, ultimately enabling advanced applications, including cancer screening and minimal residual disease (MRD) testing. Under the licensing agreement, Watchmaker may develop and commercialize the TAPS technology under its own brand. DNA methylation patterns offer dynamic and rich insights into the status of both healthy and diseased tissue. These signatures are stable, relatively abundant, and often measurable in the blood, making them promising biomarkers in minimally invasive liquid biopsies. TAPS has the potential to dramatically improve DNA methylation analysis accuracy by enabling a direct readout of cytosine methylation with less template damage and noise when compared to existing methods.1 "Exact Sciences is a leader in cancer screening, and this agreement is a great fit with Watchmaker’s core competencies in next-generation sequencing and genomics applications," said Brian Kudlow, Ph.D., Chief Scientific Officer at Watchmaker Genomics. "It aligns with our belief that multimodal genomic and epigenomic assays represent the frontier of numerous liquid biopsy applications, including early cancer detection." In addition to the development and licensing agreement, the companies have entered into a long-term strategic supply agreement. Under its terms, Watchmaker will provide Exact Sciences with access to its portfolio of best-in-class sequencing reagents and precision enzymes. These products have the potential to enhance the performance of Exact Sciences’ tests while significantly reducing associated costs. “Our company’s mission is to develop and broadly commercialize tools that improve the efficiency of capturing and interpreting the biological information required for many advanced genetic testing applications,” said Trey Foskett, CEO of Watchmaker Genomics. “This arrangement provides us with an exciting opportunity to accelerate the development and commercialization of a game-changing technology to the benefit of the broader research and clinical genomics markets.” About Watchmaker Genomics Watchmaker Genomics applies advanced enzymology to enable breakthrough applications for the reading, writing, and editing of DNA and RNA. The company combines domain expertise in protein engineering with large-scale enzyme manufacturing to address the demanding quality, performance, and scale requirements of high-growth clinical genomics applications. Watchmaker's product portfolio includes enzymes and kits for next-generation sequencing, synthetic biology, and molecular diagnostics. A leading provider of cancer screening and diagnostic tests, Exact Sciences gives patients and health care professionals the clarity needed to take life-changing action earlier. Building on the success of the Cologuard® and Oncotype® tests, Exact Sciences is investing in its pipeline to develop innovative solutions for use before, during, and after a cancer diagnosis.

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Medical

Invivoscribe Announces FDA Approval of the LeukoStrat CDx FLT3 Mutation Assay to Select Patients with FLT3-ITD Positive AML

PRNewswire | July 27, 2023

Invivoscribe is excited to announce that the LeukoStrat® CDx FLT3 Mutation Assay has been approved by the U.S. Food and Drug Administration (FDA) to aid in the selection of patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia (AML) who may be eligible to receive treatment with Daiichi Sankyo's VANFLYTA® (quizartinib). The LeukoStrat CDx FLT3 Mutation Assay identifies FLT3-ITD positive AML patients who may be treated with this newly approved targeted therapy. US Intended Use The LeukoStrat CDx FLT3 Mutation Assay is a PCR-based in vitro diagnostic test designed to detect internal tandem duplication (ITD) and tyrosine kinase domain (TKD) mutations D835 and I836 in the FLT3 gene in genomic DNA extracted from mononuclear cells obtained from peripheral blood or bone marrow aspirates of patients diagnosed with acute myelogenous leukemia (AML). The LeukoStrat CDx FLT3 Mutation Assay is used as an aid in the assessment of patients with AML for whom RYDAPT® (midostaurin) treatment is being considered. The LeukoStrat CDx FLT3 Mutation Assay is used as an aid in the assessment of patients with AML for whom XOSPATA® (gilteritinib) treatment is being considered. The LeukoStrat CDx FLT3 Mutation Assay is used as an aid in the assessment of patients with FLT3-ITD+ AML for whom VANFLYTA® (quizartinib) treatment is being considered. The test is for use on the 3500xL Dx Genetic Analyzer. "FDA approval of the LeukoStrat CDx FLT3 Mutation Assay as a companion diagnostic to VANFLYTA is a significant milestone for patients with newly diagnosed FLT3-ITD positive AML," said Invivoscribe CSO, CEO, and founder Jeffrey Miller. "Timely and accurate testing for FLT3-ITD mutations in newly diagnosed patients is critical to identify those who may be eligible for treatment with VANFLYTA, and we are happy to collaborate with Daiichi Sankyo to help bring this important new therapy to patients." AML is a blood cancer that affects the blood and bone marrow and is characterized by the rapid growth of abnormal white blood cells.1 AML has the lowest 5-year survival rate (31.7%) among people diagnosed with leukemia.2 About 25% of AML patients have a FLT3-ITD mutation which contributes to the growth and survival of cancer cells and is associated with a poor prognosis. Previously, the LeukoStrat CDx FLT3 Mutation Assay was approved by the Japan Pharmaceuticals and Medical Devices Agency (PMDA) for use as a companion diagnostic to select FLT3-ITD positive AML patients eligible for VANFLYTA in Japan. About Invivoscribe Invivoscribe is a global, vertically-integrated biotechnology company dedicated to Improving Lives with Precision Diagnostics®. For nearly thirty years, Invivoscribe has improved the quality of healthcare worldwide by providing high quality standardized reagents, tests, and bioinformatics tools to advance the field of precision medicine. Invivoscribe has a successful track record of partnerships with global pharmaceutical companies interested in developing and commercializing companion diagnostics, and provides expertise in both regulatory and laboratory services. Providing distributable kits, as well as clinical trial services through its globally located clinical lab subsidiaries (LabPMM), Invivoscribe is an ideal partner from diagnostic development, through clinical trials, regulatory submissions, and commercialization.

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MedTech, Industry Outlook

Vaxxinity Demonstrates Target Engagement of Toxic Alpha-Synuclein in Parkinson’s Patients

Globenewswire | July 18, 2023

Vaxxinity, Inc. a U.S. company pioneering the development of a new class of medicines, announced new data from a Phase 1 clinical trial demonstrating that antibodies derived from its investigational immunotherapeutic for Parkinson’s disease (PD), UB-312, slows seeding of alpha-synuclein (aSyn) in cerebrospinal fluid (CSF) of patients with PD as demonstrated using multiple target engagement assays. These data signify that UB-312 has established clear target engagement in PD patient CSF, and provides further validation of Vaxxinity’s platform technology in neurodegenerative disease. “This is a major milestone for Vaxxinity in our quest to help Parkinson’s patients. Our candidate has shown target engagement of the toxic species of alpha-synuclein in patients, demonstrating not only proof of our technology platform, but also proof of the mechanism of our vaccine-derived antibodies specifically engaging with the toxic target in vivo,” said Mei Mei Hu, CEO of Vaxxinity. “Showing target engagement has always been a key challenge to overcome in neurodegeneration, and is of critical importance when demonstrated – a milestone worth celebrating. It is beyond our expectation to see this in our Phase 1 trial. We are endlessly grateful to the patients who participated, and to The Michael J. Fox Foundation and our collaborators for their work on these cutting-edge assays that supported this breakthrough.” UB-312 is designed to target aggregated forms of aSyn, the toxic species that underlies Parkinson’s disease and other synucleinopathies. Last month, Vaxxinity announced clinical data from Part B of its Phase 1 clinical trial of UB-312 demonstrating that UB-312 was well-tolerated and induced anti-aSyn antibody responses in participants with early PD, and that antibodies were detectable in the CSF. As part of this trial, The Michael J. Fox Foundation (MJFF) funded a 2-year collaborative project between Vaxxinity, the Mayo Clinic, and UTHealth Houston to analyze CSF collected from patients, and to conduct exploratory research to characterize the anti-aSyn antibodies produced after UB-312 administration and assess target engagement. Analyses from this and related research yielded insights about the pharmacodynamic effects of anti-aSyn antibodies generated by UB-312 in the Phase 1 trial. UB-312-derived antibodies show preferential binding to aggregated aSyn isolated from patients with PD and Multiple System Atrophy (MSA), as measured by dot blot. Preclinical data published in Alzheimer’s Research & Therapy in 2020 showed similar characteristics of UB-312-derived antibodies. UB-312-derived antibodies successfully demonstrate inhibition of aggregation of aSyn in both a seed amplification assay (SAA) and a protein misfolding cyclic amplification assay (PMCA). These techniques can potentially be used to identify people with PD, and also to measure the treatment response and pharmacodynamic properties of UB-312-derived antibodies from subjects in clinical trials. Importantly, aSyn aggregation was slowed down in CSF samples from PD patients who received UB-312, as compared to those who received placebo, in the Phase 1 trial. Vaxxinity plans to continue analyses of the clinical data as part of the collaborative project with MJFF, in addition to completing other target engagement assays and additional antibody characterization studies for binding kinetics and specificity. Mark Frasier, Ph.D., Chief Scientific Officer of MJFF, commented, “Integration of critical biomarker insight into therapeutic development programs is essential for building confidence in the treatment approach, and for designing informative trials. We’re pleased to support efforts of this kind that can have major impact for people with Parkinson’s disease.” About Parkinson’s Disease Parkinson’s disease (PD) affects approximately one million people in the United States and more than 10 million people worldwide. PD is a chronic and progressive neurodegenerative disorder that affects predominately dopamine-producing (“dopaminergic”) neurons in the substantia nigra area of the brain. While today’s approved products are aimed at providing symptomatic relief, they often produce significant side effects and lose their beneficial effects over time. There are no currently approved disease-modifying therapeutics for PD. Alpha-synuclein (aSyn) is a protein highly expressed in neurons, mostly at presynaptic terminals, suggesting a role in synaptic vesicle trafficking, synaptic functions and in regulation of neurotransmitter release at the synapse. Mutations in the gene encoding aSyn are known to cause or increase the risk of developing PD or dementia with Lewy bodies (DLB) and have been shown to alter the secondary structure of aSyn, resulting in misfolded and aggregated forms of the protein (i.e., pathological forms). While mutations in the aSyn gene are rare, aggregates of aSyn in the form of Lewy bodies (LB) and Lewy neurites are common neuropathological hallmarks of both familial and sporadic PD, suggesting a key role of aSyn in PD neuropathogenesis. Immunotherapy approaches targeting aSyn have been shown to ameliorate aSyn pathology as well as functional deficits in mouse models of PD and are now being investigated in the clinic. About UB-312 UB-312 is a vaccine candidate targeting pathological forms of alpha-synuclein (aSyn) for the disease-modifying treatment and prevention of Parkinson’s disease (PD) and other synucleinopathies. Preclinical data indicated that UB-312 elicits antibodies that preferentially recognize pathological forms of aSyn, and improve motor performance in mouse models of synucleinopathies. Clinical data from the Phase 1 trial indicate that UB-312 elicits antibodies that target aggregated aSyn, and that these antibodies slow the aggregation of alpha-synuclein in the cerebrospinal fluid of patients with PD. The European Medical Agency has granted UB-312 orphan designation for multiple system atrophy. About Vaxxinity Vaxxinity, Inc. is a purpose-driven biotechnology company committed to democratizing healthcare across the globe. The company is pioneering a new class of medicines aimed at disrupting the existing treatment paradigm for chronic disease, increasingly dominated by monoclonal antibodies, which suffer from prohibitive costs and cumbersome administration. The company’s proprietary technology platform has enabled the innovation of novel synthetic peptide immunotherapy candidates designed to bring the efficiency of vaccines to the treatment of chronic diseases, including Alzheimer’s disease, Parkinson’s disease, migraine, and hypercholesterolemia. The technology is also implemented as part of a COVID-19 vaccine program. Vaxxinity has optimized its pipeline to achieve a potentially historic, global impact on human health.

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