First U.S. Patients Are Treated With CRISPR as Echoes of Scandal in China Still Linger

Biospace | April 17, 2019

The first clinical trials using CRISPR technology are expected to get underway in the United States, even as echoes of a scandal involving the use of CRISPR on embryos in China are still being felt across the globe.A sickle cell trial using CRISPR is expected to begin later this year in the U.S., NPR said. Also, Editas Medicine, another gene-editing company, plans to initiate clinical trials using CRISPR to treat a genetic form of blindness known as Leber congenital amaurosis. As NPR notes, the Editas trial is significant as the study looks to edit the genes while still in the body. The other trials involved removal of cells from the body, editing them and then reinserting them back into the patient.

Spotlight

The MSAP® Method of composting was originally developed to mitigate odors at several biosolids composting facilities, which were being encroached upon by housing developments.

Spotlight

The MSAP® Method of composting was originally developed to mitigate odors at several biosolids composting facilities, which were being encroached upon by housing developments.

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INDUSTRIAL IMPACT

Quantum Genomics Signs an Exclusive License and Production Agreement with Gulf Pharmaceuticals Industries Julphar

Quantum Genomics | December 06, 2021

Quantum Genomics, a biopharmaceutical company specializing in the development of first-in-class drugs directly targeting the brain to treat complex/resistant hypertension, has announced the signing of an exclusive license and production agreement with Julphar to market and produce firibastat in the Middle East, all African countries, Commonwealth of Independent States and Turkey. A pharmaceutical company based in the United Arab Emirates and leader in the MENA region, Julphar will receive exclusive rights to manufacture and market firibastat in the following geographic areas: the MENA1 region, CIS2, Turkey and all other countries on the African continent. With its state-of-the-art manufacturing facilities in the emirate of Ras Al Khaimah, making it possible to mass produce firibastat, Julphar will also be Quantum Genomics’ exclusive supplier for said regions. Quantum Genomics will receive up to $20M in upfront, development and sales milestone payments as well as royalties on future firibastat sales. Julphar has also pledged to invest $2M in Quantum Genomics through a private placement. Details of the transaction will be announced shortly. "Julphar, which was founded forty years ago, is one of the largest pharmaceutical companies in the MENA region. Its success has been built on its comprehensive portfolio including anti-infectives, dermatology and diabetes products and its presence in all key MENA markets. Its business is expanding fast, and cardiology is one of its key areas of investment. Julphar is also among the region’s largest producers and is the ideal partner to support firibastat’s future production needs. We’re already exploring how Julphar could meet some of those requirements for the United States and Europe," Jean-Philippe Milon, CEO of Quantum Genomics Commenting on the agreement, Dr. Essam Mohammed, Julphar’s CEO said: “Julphar is delighted to partner with Quantum Genomics to commercialize firibastat, a first in class molecule to treat resistant and difficult-to-treat hypertension, and to produce it in our state-of-the-art manufacturing facilities in Ras Al Khaimah. We are confident that firibastat will add strong value to the treatment of this unmet medical need and to Julphar’s portfolio.” Dr. Essam Mohammed added: “The new landmark agreement brings together a stronger focus on innovative ways to address critical diseases in the MENA region through manufacturing high-quality medications locally.” About Julphar Julphar is one of the largest pharmaceutical manufacturers in the Middle East and Africa, and for more than four decades, the company has been delivering high quality, innovative and affordable healthcare solutions to families across the globe. Established under the guidance of His Highness Sheikh Saqr Bin Mohammed Al Qasimi in 1980, Julphar employs 2,500 people and distributes pharmaceutical products to more than 50 countries across the globe. Julphar’s business is centered on three core business units – Julphar Diabetes Solutions, General Medicines and its consumer division, Julphar Life – which target major therapeutic segments including Gastrology, Pain Management, Wound Care, Antibiotics and Cardio-metabolism. Julphar has 13 internationally accredited facilities in the Middle East and Africa. In 2012, Julphar became one of the largest producers of Insulin with its UAE-based biotechnology production unit. About Quantum Genomics Quantum Genomics is a biopharmaceutical company specializing in the development of a new class of cardiovascular drugs, based on the Brain Aminopeptidase A Inhibition (BAPAI) mechanism. It is the only company in the world to pursue this innovative approach directly targeting the brain, founded upon more than twenty years of research work by Paris-Descartes University and the INSERM/CNRS laboratory led by Dr. Catherine Llorens-Cortès at the Collège de France. Quantum Genomics thus aims to develop innovative treatments for complicated or even resistant hypertension and heart failure.

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INDUSTRIAL IMPACT

TransPerfect Life Sciences and Innovaderm Research Expand Trial Interactive Partnership

TransPerfect Life Sciences | February 16, 2022

TransPerfect Life Sciences, a leading provider of services and technologies to support global clinical trials and product development for the biopharmaceutical industry, today announced that Innovaderm will scale their implementation of Trial Interactive to further enable and optimize remote clinical operations. Innovaderm, a North America-based contract research organization (CRO) that specializes in clinical studies for dermatology, leveraged Trial Interactive remote monitoring rooms to drive research forward and continue helping partners discover new therapies during the COVID-19 pandemic. By incorporating customized Trial Interactive remote monitoring rooms into its service offering, Innovaderm allowed customers to access Trial Interactive’s Study Document Collaboration solution, enabling successful remote trial execution. Innovaderm’s partnership with TransPerfect began several years ago with language translation services. To eliminate its paper-based approach for clinical trial document management, Innovaderm then implemented Trial Interactive’s eTMF. As Innovaderm further optimized its clinical research strategy, TI Study Start-Up replaced the CRO’s manual process for distribution and collection of essential documents from sites, resulting in a more transparent, efficient path for site activation and reduced timelines for starting new studies. Trial Interactive delivers user-friendly document and trial management solutions to help pharmaceutical, biotechnology, and CRO companies effectively streamline product development life cycles. A connected eClinical experience, Trial Interactive improves speed, quality, and compliance across site identification, selection, and activation, as well as study teams and site personnel training, content management, eISF, eTMF management, CTMS, and closeout. The platform offers industry-leading, fast-to-implement, cost-effective solutions in a secure 21 CFR Part 11-compliant environment. “Trial Interactive worked with us to ensure our remote monitoring room configuration met our key requirements so we could conduct our studies without interruptions, delays, or significant changes. As we’ve faced unexpected shifts in the way we run and monitor studies, Trial Interactive has been key to enabling us to perform remote monitoring visits.” Isabel Dorion, Director of Clinical Operations at Innovaderm Michael Smyth, TransPerfect Life Sciences Solutions Division President, stated, “We’re moving into an era where decentralized clinical trials are becoming more common, and conducting clinical research processes remotely is key to keeping trials moving forward and getting life-saving products to market faster. With our solution of remote monitoring rooms, Trial Interactive has been able to help clients like Innovaderm shift to remote processes without losing momentum for their studies.” About Trial Interactive TransPerfect’s Trial Interactive is an industry leader in practical eClinical innovation that simplifies and automates clinical processes for sponsors, CROs, and sites around the world. The 21 CFR part 11 compliant unified platform delivers an author-to-archive collaboration experience with solutions for clinical document management, site selection, site activation, eLearning, compliance training, quality, and CTMS with seamless solution interoperability and indexing to the eTMF. Trial Interactive is consistently selected by clinical professionals for providing the most comprehensive yet intuitive experience with the most complete offering of technology and expert TMF services. Trial Interactive helps study teams streamline their operations by cutting unnecessary expenses, expediting timelines, reducing compliance risks, and improving operational excellence. About TransPerfect Life Sciences TransPerfect Life Sciences specializes in supporting global development and commercialization of drugs, treatments, and devices designed to improve and save lives. Our comprehensive solutions include eTMF and eClinical technologies, paper TMF migration, pharmacovigilance and safety solutions, translation and language services, and call center support. With offices in over 100 cities worldwide, TransPerfect is the ideal partner to ensure that your global launch makes a global impact. About Innovaderm Research, Inc. Innovaderm is a prominent contract research organization (CRO) collaborating with the biotech companies in the management of Phase 1 to 3 dermatology trials across many regions. Founded in 2000, the company provides a broad range of services including study design, protocol development, site selection, patient recruitment, regulatory and ethics submissions, clinical trial management, clinical monitoring, data management, biostatistics, medical monitoring, pharmacovigilance and medical writing. Committed to quality, Innovaderm is managing its activities in accordance to current Good Clinical Practice (GCP) from the International Conference of Harmonization (ICH), and being fully compliant with major international regulations such as the FDA (USA), Health Canada and EMA (Europe).

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MEDTECH

CosmosID Launches Accurate and Easy Functional Microbiome Analysis with New Software Platform, CosmosID-HUB: Microbiome

CosmosID | September 27, 2021

A complete analysis of the microbiome should combine taxonomic profiling ("who is there?") with functional characterization ("what are they doing?") to discover the capabilities of an entire community of microorganisms. But research teams often require specialized training and extensive computational infrastructure to establish an in-house suite of tools for processing shotgun metagenomic data – and even with the right know-how, the processing can be both time-consuming and challenging. CosmosID®, known for the most accurate, multi-kingdom, strain level taxonomic profiling technology, has added a functional analysis module to its popular online user-interface, renamed 'CosmosID-HUB: Microbiome'. Now, in addition to microbiome sequencing services out of its Germantown, MD, laboratory, its industry-leading team of bioinformaticians and software developers, has unveiled a suite of new features for seamless analysis of the genomic potential of microbial communities: Direct links from the platform to MetaCyc and GO terms databases. Data import via direct upload, Illumina BaseSpace & automated SRA import from NCBI with analysis speeds up to 19X faster than open-source tools. Find your organism or gene/pathway of interest with search functions, exportable charts & visualizations. Comparative analysis software, complete with dynamic charts, graphs, and statistical significance testing. Easy-to-navigate user interface OR command-line access. Applicable to almost any sample type Applicable to both metagenomic and metatranscriptomics data Knowing the combined genomic potential of a microbial community is proving indispensable for practical applications of translational microbiome science, This cutting edge, accurate, easy and fast functional analysis feature is part of our ongoing mission to provide everything researchers need to complete their end-to-end microbiome analysis and lower the barrier to entry for those interested in the field. - CosmosID CEO, Manoj Dadlani. The updated web application is part of CosmosID's microbial genomics platform, CosmosID-HUB, which is continually expanding with various apps and modules for analyzing microbial communities and for inferring translational and actionable microbiome insights. Currently, CosmosID-HUB: Microbiome provides options for discovering antimicrobial resistance and virulence markers, microbial taxa (composition) and function whereas CosmosID-HUB: COVID-19 is a separate module for detecting the presence of COVID-19 and characterizing variants of concern/interest in both clinical and wastewater specimens. Researchers have the flexibility to access and pay for the type of analysis they require. Soon, CosmosID-HUB will include a revolutionary web application for microbiome multi-dimensional feature testing and multi-omics analysis. In addition to strain-level resolution enabled by its curated databases of over 170,000 genomes and gene sequences, CosmosID® also offers next-generation sequencing services. To date, the company has demonstrated its best-in-class technology and won three different challenges for its detection accuracy, including both PrecisionFDA challenges as well as the Janssen/DNAnexus Mosaic 'Strain' challenge. About CosmosID® CosmosID® provides end-to-end solutions unlocking the microbiome. A provider of CLIA-certified & ICH-GCP compliant NGS Services and Bioinformatics Solutions, CosmosID offers a range of validated and optimized workflows for a range of applications including pharmaceutical R&D, infectious disease diagnostics, environmental testing, microbiome research, animal health, cosmetics & personal care as well as many more. CosmosID also offers independently validated, industry-leading pipelines for processing metagenomic data, yielding multi-kingdom, strain-level resolution with leading sensitivity and precision as well as functional characterization

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