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 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 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 Inotiv, Inc. (NASDAQ:NOTV) (the "Company", "We", "Our" or "Inotiv"), a leading contract research organization specializing in nonclinical and analytical drug discovery and development services, reported that the Company has acquired key genetic toxicology assets from MilliporeSigma’s BioReliance portfolio, including standard operating procedures, stock cultures, historic control data and client list (Press release, MilliporeSigma, JUL 12, 2021, View Source [SID1234584808]). While Inotiv did not disclose the specific financial terms, the transaction consists of a sales-based royalty agreement and does not require upfront funding from the Company.

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Robert Leasure, Jr., President and CEO of Inotiv, commented, "This transaction with MilliporeSigma adds genetic toxicology to our suite of internal capabilities. We now have assembled in-house all of the critical nonclinical services necessary to support our clients’ goals of advancing to human clinical trials."

MilliporeSigma is discontinuing their Genetic Toxicology operations. The agreement with Inotiv is designed to ensure continuity of services for existing clients. Inotiv also plans to offer employment to certain MilliporeSigma employees who otherwise may have been displaced.

To facilitate entry into the genetic toxicology business, Inotiv also announced that Gopala Krishna, PhD, MBA, has joined Inotiv as Senior Vice President, Genetic Toxicology, to lead the Company’s genetic toxicology service offering. Dr. Krishna is a Diplomate, American Board of Toxicology, a Fellow of the Academy of Toxicological Sciences, and has over three decades of pharmaceutical industry experience as a drug discovery and development nonclinical leader at large and small Pharma companies, such as Pfizer, Abbott Labs, MGI Pharma, Enzon, and Supernus. He has also served as Professor (Adjunct) at the University of Michigan teaching Pharmacology & Toxicology in the School of Public Health, Ann Arbor, MI. Most recently he served as Principal Consultant – Nonclinical at PAREXEL International. Currently, he is the President of National Capital Area Chapter (NCAC) of Society of Toxicology (SOT) and has been a contributing senior member of the Environmental Mutagenesis and Genomics (Genetic Toxicology) Society (EMGS) for over 35 years.

"Evaluation of the potential for novel therapeutics to interact with and alter DNA is a key element of the safety assessment of those drug candidates," said John E. Sagartz, Chief Strategy Officer of Inotiv. "Inotiv historically has relied on other vendors to provide these key assessments. I’m excited to announce the addition of the BioReliance assets and Dr. Krishna, and we look forward to developing the genetic toxicology business. We believe this is another significant milestone for Inotiv and our clients."

Dr. Krishna commented, "I am delighted to join Inotiv with the task of expanding the service offering to include genotoxicity capabilities and look forward to leveraging the assets of the BioReliance portfolio to assist our clients in the development of safe novel therapeutics."

In support of this new offering, Inotiv expects to lease space near the current MilliporeSigma’s facilities in Rockville, Maryland.

On July 12, 2021 MaxCyte, Inc., (LSE: MXCT) (LSE: MXCN), a leading provider of platform technologies for cell engineering, reported the public filing of a registration statement on Form S-1 with the U.S. Securities and Exchange Commission (the "SEC") relating to a proposed dual listing of its shares of common stock on the Nasdaq Global Market ("Nasdaq") under the symbol "MXCT (Press release, MaxCyte, JUL 12, 2021, View Source [SID1234584773])." As part of the proposed Nasdaq dual listing, MaxCyte is planning a public offering of shares of its common stock in the United States (the "Offering"). Upon completion of the Offering, MaxCyte’s common stock will continue to be admitted to trading on the AIM market of the London Stock Exchange under the symbols "MXCT" and "MXCN."

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All shares to be sold in the Offering will be offered by MaxCyte. The number of shares to be offered and the price range for the proposed Offering have not yet been determined. The Offering is expected to commence after the SEC completes its review process, subject to market and other conditions. Stockholders and potential investors should note that there is no guarantee as to whether or when the potential Offering will proceed.

Cowen, Stifel and William Blair are acting as joint book-running managers for the Offering and as representatives of the underwriters for the proposed Offering. BTIG and Stephens Inc. will also act as co-managers of the Offering.

The proposed Offering will be made only by means of a prospectus. When available, copies of the preliminary prospectus relating to and describing the terms of the Offering may be obtained from the offices of Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attn: Prospectus Department, by telephone at (833) 297-2926 or by email at [email protected]; Stifel, Nicolaus & Company, Incorporated, Attention: Prospectus Department, One Montgomery Street, Suite 3700, San Francisco, CA 94104, by telephone at +1 (415) 364-2720 or by email at [email protected]; or William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, by telephone at 1-800-621-0687 or by email at [email protected].

A registration statement relating to these securities has been filed with the SEC, but has not yet become effective. These securities may not be sold, nor may offers to buy these securities be accepted, prior to the time the registration statement becomes effective. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities law of any such state or jurisdiction.