On July 12, 2021 Tempus, a leader in artificial intelligence and precision medicine, reported results from validation studies demonstrating the reliable analytical performance of the Tempus|xF liquid biopsy (Press release, Tempus, JUL 12, 2021, View Source [SID1234584789]). When validated against methods such as ddPCR, the Roche AVENIO kit, and the Tempus|xT solid tumor assay, xF demonstrated high sensitivity and specificity for calling SNVs, indels, CNVs, and gene rearrangements, making the liquid biopsy next-generation sequencing assay a preferred sequencing option should tumor tissue not be available.

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xF is Tempus’ non-invasive, 105-gene liquid biopsy panel focused on oncogenic and resistance mutations in cell-free DNA. Designed to provide clinical decision support for solid tumors, xF is an alternative to tissue-based biopsies in identifying biomarkers, detecting resistant mutations, monitoring response to treatment or disease progression, and spotting early recurrence in real time. npj Precision Oncology published xF’s analytical validation, highlighting the assay’s high sensitivity and specificity for detecting single-nucleotide variants, insertions/deletions, copy number variants, and gene rearrangements. The validation results show high accuracy in detecting clinically-actionable targets compared to orthogonal testing methods.

"This study underscores the clinical value of liquid biopsies, a non-invasive sequencing option and another powerful tool for oncologists to determine clinically-actionable alterations necessary for highly-effective and personalized treatment options," said Dr. Kimberly Blackwell, Chief Medical Officer at Tempus. "At Tempus, we have also found that xF is especially beneficial when used in combination with standard tissue sequencing for patients with advanced cancer, and therefore it is important to offer oncologists both a liquid biopsy and solid tumor assay on a single platform."

xF is part of Tempus’ library of assays conducted in its CLIA-certified and CAP-accredited laboratory, which also includes xE, an assay that analyzes the whole exome and xT, an assay that analyzes 648 genes in solid tumor and hematologic malignancies.

On July 12, 2021 Caribou Biosciences, Inc., a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported that the first patient has been dosed in its open-label, multicenter ANTLER phase 1 clinical trial (NCT04637763) to evaluate the company’s lead product candidate, CB-010, in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) (Press release, Caribou Biosciences, JUL 12, 2021, View Source [SID1234584788]). CB-010 is an allogeneic anti-CD19 CAR-T cell therapy derived from healthy donor T cells that have been engineered using Caribou’s chRDNA technology to reduce the risk of graft versus host disease (GvHD) and knock out PD-1 to boost the persistence of CAR-T cell antitumor activity.

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"Advancing our first allogeneic CAR-T cell therapy into the clinic represents a major milestone for Caribou," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "Using our proprietary chRDNA CRISPR technology, we have developed a pipeline of off-the-shelf CAR-T and CAR-NK cell therapies with the potential to serve a greater number of patients than autologous approaches. We believe that improving cell persistence is the key to unlocking the full potential of these therapies. Using our technologies, we edit the genome of healthy donor-derived T cells to enable highly specific and efficient insertion or deletion of genes at multiple sites. This allows us to create sophisticated allogeneic cell therapies with enhanced characteristics to potentially improve their effectiveness and durability of antitumor activity compared to other allogeneic cell therapies. With CB-010, we are evaluating the potential persistence-enhancing effects of removing the PD-1 protein from the surface of these CD19-targeted CAR-T cells."

Cherry Thomas, M.D., senior vice president of clinical development, added, "We are thrilled to advance CB-010 in the clinic and believe it potentially represents a promising, differentiated therapy for patients. Initially, we will evaluate escalating doses of CB-010 in relapsed or refractory B cell non-Hodgkin lymphoma patients with the goals of assessing safety and tolerability and to establish a dose level for the expansion phase of the study. We look forward to having initial clinical data from this clinical trial in 2022."

About CB-010

CB-010 is an allogeneic anti-CD19 CAR-T cell therapy derived from healthy donor T cells. The cells are engineered using Caribou’s chRDNA CRISPR technology to integrate a CD19-CAR site-specifically into the T cell genome at the site of the TRAC gene locus, thus eliminating expression of the T cell receptor to reduce the risk of GvHD. The cells are modified further using chRDNA to knock out the gene encoding PD-1, thus preventing the expression of the PD-1 protein on the CAR-T cell surface. Elimination of PD-1 expression is designed to boost the persistence of CAR-T cell antitumor activity by reducing CB-010 exhaustion and thereby potentially providing a better therapeutic index compared to other allogeneic CAR-T cells. An additional step in the manufacturing process for CB-010 is designed to remove residual T cells expressing a T cell receptor, further reducing the risk of GvHD.

About the Phase 1, Dose Escalation Study of CB-010 (ANTLER Study, NCT04637763)

The ANTLER study is an open-label, multicenter phase 1 clinical trial designed to evaluate CB-010 in adults with r/r B-NHL. In the dose-escalation part of the study, adults who have failed at least two lines of chemo and/or immunotherapy will receive CB-010 following lymphodepletion. In a standard 3+3 dose escalation design, increasing doses of CB-010 will be evaluated to determine safety and tolerability and to establish a dose level for the expansion part of the study. The primary objective of the dose escalation is to evaluate the safety and tolerability of CB-010 and the primary objective of the expansion part of the study is to evaluate the efficacy of CB-010 in a defined population. Patients who have received a prior CD19-targeted therapy will be excluded from the study.

About Caribou’s Novel Next-Generation CRISPR Platform

CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Type II CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems occasionally edit unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed chRDNAs (pronounced "chardonnays"), RNA-DNA hybrid guides that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of the chRDNA technology to carry out high efficiency multiple edits, including multiplex gene insertions, to develop CRISPR-edited therapies.

On July 12, 2021 OPKO Health, Inc. (NASDAQ: OPK) reported it has entered into an exclusive worldwide agreement with privately held CAMP4 Therapeutics Corporation (CAMP4) for the development, manufacture and commercialization of therapeutics utilizing the AntagoNAT technology, an oligonucleotide platform developed under OPKO CURNA (Press release, Opko Health, JUL 12, 2021, View Source [SID1234584787]). AntagoNATs are oligonucleotide compounds that target non-coding natural antisense transcripts leading to an upregulation of a desired functional protein. CAMP4 has prioritized OPKO’s lead AntagoNAT compound to progress into clinical trials for the treatment of Dravet syndrome.

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Under the terms of the agreement, OPKO will receive an upfront payment and shares of CAMP4. In addition, OPKO will be eligible to receive up to $93.5 million and additional shares upon the achievement of certain development and sales milestones for products developed from this technology and associated intellectual property. CAMP4 will also pay OPKO double-digit royalties on product sales.

"We are delighted to enter into this licensing agreement to accelerate the development and commercialization of potential disease-modifying therapeutics. With a clear focus on therapeutics that restore healthy gene expression, CAMP4 has the expertise to advance our Dravet syndrome compound through the necessary patient trials. This agreement validates our technology and allows OPKO to focus resources on other areas of our business. We continue to seek license opportunities to monetize and leverage our early-stage assets," said Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO.

According to the Epilepsy Foundation, Dravet syndrome is a rare, drug-resistant epilepsy that begins in the first year of life in an otherwise healthy infant. It is estimated that 1-in-20,000 to 1-in-40,000 individuals have Dravet syndrome. Most cases are due to SCN1A gene mutations and most children develop varying degrees of neurodevelopmental disability.

CAMP4 is pioneering a novel approach to programmable therapeutics that combines a deep understanding of regulatory RNA and gene expression with a complementary and customizable oligonucleotide modality. The company’s RNA Actuating Platform’s proprietary insights enable drug discovery and development that aims to harness the power of RNA to upregulate the expression of genes and unlock the potential to create treatments for hundreds of diseases affecting millions of patients.

On July 12, 2021 Vernal Biosciences, Inc., a manufacturer of high purity mRNA for research and clinical use, reported the closing of a seed round of financing (Press release, Alloy Therapeutics, JUL 12, 2021, View Source [SID1234584786]). Alloy Therapeutics led the round and was joined by the Vermont Center for Emerging Technologies and individual angel investors. The proceeds of the financing will be used to scale Vernal’s technical development and commercial operations in the Burlington, VT region.

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Vernal is focused on achieving and maintaining high purity throughout the scaling of all mRNA manufacturing processes for clients in the research and ultimately clinical stages. The company was founded by CEO Christian Cobaugh, who previously held mRNA process and manufacturing leadership roles at Omega Therapeutics, Translate Bio, Arcturus Therapeutics, and Alexion Pharmaceuticals, where he was the first researcher in the Alexion-Moderna partnership. He brings a deep understanding of the mRNA manufacturing value-chain—including mRNA design and specifications, regulatory expectations, and quality product profiles—to improve Vernal clients’ odds of success through a highly adaptable and well-characterized manufacturing process.

"We are inspired to realize the potential of mRNA, a validated and revolutionary medicines modality, through democratizing access to unparalleled mRNA manufacturing technologies," said Christian Cobaugh, founder and CEO of Vernal. "The associated knowledge, technology, and talent has long been cloistered within select mRNA therapeutics companies. It is critical to remove barriers to mRNA manufacturing technologies for the benefit of patients and investors seeking novel use cases of mRNA. Equal access to high purity mRNA manufacturing will encourage growth in innovation, competition, and collaboration, accelerating the clinical impact of mRNA as a foundational therapeutic modality."

Through collaborative research and partnerships, Vernal is developing innovative process and analytical technologies to overcome the challenges of manufacturing high purity mRNA. The company’s universal platform, which emphasizes Quality-by-Design and documented process characterization, will accelerate client projects and simplify regulatory interactions. Vernal is fine-tuning this platform so that clients do not "hurry up and wait" for clinical manufacturing processes to start from scratch—enabling clients to focus on other high-value operations such as target biology, drug discovery, and clinical development. Vernal’s mRNA solutions will scale with client needs, regardless of discovery or clinical stage or use case.

Already engaging with clients on research and preclinical projects, Vernal’s seed financing will be used to scale its technology and business to add the GMP clinical manufacturing capability operations for ongoing and future client discovery projects. As part of the Series Seed financing, Errik Anderson, CEO and founder of Alloy Therapeutics, will join Vernal’s Board of Directors. Pat Sacco, longtime biotech technical operations and manufacturing executive (senior vice president of technical operations at both Translate Bio and Shire plc/TKT), will also be joining Vernal as a board advisor. While at Shire, Pat’s teams supported development, manufacturing, and commercialization of REPLAGAL, ELAPRASE, and VPRIV.

"Backing Vernal’s vision for mRNA manufacturing is fully aligned with Alloy’s ecosystem approach and our commitment to reinvest 100% of Alloy’s revenues back into innovation and access to innovation," said Anderson. "Collaboration and democratization of foundational drug discovery capabilities are critical in ensuring our industry delivers on its promise to get the best drugs to patients faster. Alloy is thrilled to support Christian and the Vernal team in their mission to bring high quality mRNA to innovative companies and projects of all sizes."

Vermont Seed Capital Fund Manager David Bradbury added, "We are thrilled to welcome mRNA veteran Christian Cobaugh and the Vernal team into Vermont and support the launch of this high potential new life sciences company."

On July 12, 2021 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery and development of drugs for the treatment of cancer, reported that it has entered into a development and commercialization collaboration with LegoChemBio, a clinical stage South Korea-based biotechnology company, for the development and commercialization of novel first-in-class phospholipid drug conjugates (PDCs) (Press release, Cellectar Biosciences, JUL 12, 2021, View Source [SID1234584785]).

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Under the agreement, the two companies have the option to jointly develop three new small molecule PDCs utilizing Cellectar’s proprietary drug targeting platform, phospholipid ether (PLE) technology and LegoChemBio’s proprietary drug conjugate linker-toxin platform. The co-development option is exercisable at defined points with either party allowed to acquire full global commercialization rights. The parties have further agreed to focus development of the drug candidates on solid tumors with significant unmet medical need and potential for accelerated regulatory pathways . Details of the financial terms of the agreement have not been disclosed.

"This partnership reflects the shared commitment of LegoChemBio and Cellectar to rapidly provide novel targeted therapies to patients with difficult to treat cancers. LegoChemBio’s proprietary and validated ADC linker-toxin platform technology is well-suited to be combined with our validated PLE tumor targeting technology to generate new PDC’s" said James Caruso, president and CEO of Cellectar. "This collaboration has potential to further enrich our oncology pipeline and builds upon our strategy of developing our PDC platform across a multitude of targeted cancer treatment modalities, including radioisotopes small molecules as well as others."

Dr. Yong-Zu Kim, CEO of LegoChemBio said, "This collaboration is of great significance for the expansion of the application of LegoChemBio’s ADC linker-toxin platform using an innovative drug delivery platform technology with a novel mechanism beyond antibodies. Through this cooperation with Cellectar and its’ validated competitive platform technology in the field of targeted therapies, we will drive our research capabilities to create novel PDC clinical candidates with full speed."