Researchers use gene editing with CRISPR to treat lethal lung diseases before birth

medicalxpress | April 17, 2019

Using CRISPR gene editing, a team from Children's Hospital of Philadelphia (CHOP) and Penn Medicine have thwarted a lethal lung disease in an animal model in which a harmful mutation causes death within hours after birth. This proof-of-concept study, published today in Science Translational Medicine, showed that in utero editing could be a promising new approach for treating lung diseases before birth. "The developing fetushas many innate properties that make it an attractive recipient for therapeutic gene editing," said study co-leader William H. Peranteau, MD, an investigator at CHOP's Center for Fetal Research, and a pediatric and fetal surgeon in CHOP's Center for Fetal Diagnosis and Treatment. "Furthermore, the ability to cure or mitigate a disease via gene editing in mid- to late gestation before birth and the onset of irreversible pathology is very exciting. This is particularly true for diseases that affect the lungs, whose function becomes dramatically more important at the time of birth." The lung conditions the team is hoping to solve—congenital diseases such as surfactant protein deficiency, cystic fibrosis, and alpha-1 antitrypsin—are characterized by respiratory failure at birth or chronic lung disease with few options for therapies. About 22 percent of all pediatric hospital admissions are because of respiratory disorders, and congenital causes of respiratory diseases are often lethal, despite advances in care and a deeper understanding of their molecular causes. Because the lung is a barrier organ in direct contact with the outside environment, targeted delivery to correct defective genes is an attractive therapy.

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BUSINESS INSIGHTS

Lifespin Launches Commercial Access to its Metabolic Profiler Software and Database for Pharma and Biobank Services

Lifespin | September 16, 2022

Lifespin GmbH, based in Regensburg with offices in Boston, Massachusetts, announced that it has launched a new commercial service that, for the first time, will provide the pharmaceutical and biobanking industry with access to Lifespin’s proprietary metabolic database, along with its advanced interpretive software to assist in various phases of drug research, development, and manufacturing. Immediate applications of Lifespin’s commercial services will include quality control for synthetic/natural compounds, quantitative profiling of metabolites in liquid samples, monitoring of drug responses and organ-specific metabolic phenotyping, and precision nutrition to therapeutic drug monitoring and longitudinal treatment monitoring. “Making our technology platform accessible to the pharma and biobanking industry will provide the field with deeper clinical insights and improve stratification of patient data in clinical trials, enabling pharmaceutical companies and others in the field to perform a range of precision phenotyping of cell or animal models and patient cohorts. Our goal is to provide the pharma and biobank scientists with yet another powerful resource in their development toolbox, including our advanced algorithms and database, to enhance their earlier phase analysis and to help better identify the strengths and weaknesses of compounds in study during research through clinical trials.” Dr. Ali Tinazli, CEO of Lifespin GmbH Lifespin, a deep data company that maps human health based on snapshots of metabolic states, has built one of the largest and most comprehensive databases of metabolic health profiles across healthy and diseased individuals covering multiple age and gender groups as well as specific diseases in neurology, oncology, and inflammation. Utilizing proprietary technology, Lifespin is performing quantitative in-house measurements of metabolomes, digitizing metabolic profiles that include billions of metabolic relationships. These digital metabolic profiles allow systematic mapping across various health conditions and may enable differential diagnosis and early detection of health conditions, staging of diseases, monitoring of treatment success and personalized medicine. Lifespin’s advisory board consists of key opinion leaders such as James Rothman (Nobel Laureate in Physiology/Medicine, Sterling Professor of Cell Biology, Yale University, New Haven, CT USA) and other prominent leaders in the relevant fields of study.

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PHARMA TECH

ArisGlobal Accelerates Growth Announces Executive Evolution

ArisGlobal | September 08, 2022

ArisGlobal, the leading provider of life sciences software that automates core drug development functions with its end-to-end technology platform LifeSphere®, today announces an expansion to its C-suite. James Jarrett is the company's new Chief Financial Officer, Kathleen Turland joins as the first Chief Legal Officer, and Aman Wasan has stepped into the new role of Chief Commercial Officer. The three appointments are happening during a time of significant momentum for ArisGlobal, which has experienced annual recurring revenue growth of 34% in the first half of 2022 YoY. James Jarrett, Chief Financial Officer Jarrett brings over a decade of executive leadership experience driving revenue and cash flow growth. He has partnered with management teams and boards of directors of high-growth, highly leveraged companies undergoing rapid change. Prior to joining ArisGlobal, he served as the CFO for Emergence Health Holdings, Carewell Urgent Care, and ProCure Proton Therapy Center. Kathleen Turland, Chief Legal Officer Turland is a global strategic leader with more than 20 years of legal experience. She brings her broad range of legal and business skills to ArisGlobal, having worked with companies experiencing significant growth and expansion over the years, including General Electric, and FUSE Group. She started her legal career in New York, clerking for a Federal Court Judge, and working at two revered New York law firms. Aman Wasan, Chief Commercial Officer Wasan is an expert in general management and commercial leadership with a deep understanding and knowledge of drug safety and clinical research. Since joining ArisGlobal in 2018, Wasan has excelled in six different positions, most recently as Senior Vice President of Global Commercials, where he managed global enterprise business. Before joining the ArisGlobal team, Wasan worked for Bioclinica as the company's Global Head of Safety and Regulatory Services. "As we continue scaling our business and building new functions to better serve our customer base, expanding our C-suite will help us execute our mission and further drive our success in the life sciences industry. James, Kathleen, and Aman carry a wealth of industry knowledge instrumental in empowering ArisGlobal to further expand its customer pipeline, continue to develop innovative technology, and maintain a role as a trusted leader in the market." CEO Mike Gordon Turland's addition to the company also represents consistent and focused efforts by ArisGlobal to continually put women in positions of leadership. Turland joins ArisGlobal veterans Ritu Shrivastava, Vice President Corporate Development, and Heidi Hattendorf, Vice President of Marketing, on the executive team. Currently, ArisGlobal also has women in core leadership positions across business, product, quality, people, and technology functions. About ArisGlobal ArisGlobal is led by passionate individuals who support life sciences leaders in developing and monitoring breakthrough medicines and therapies. With more than 35 years of expertise in the life sciences industry, ArisGlobal develops technology products within the platform LifeSphere to power pharmaceutical and biotech research and development. Building on our commitment to corporate social responsibility and sustainability initiatives, we give back to the communities where we live and work – in the U.S, Europe, India, Japan, and China.

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PHARMA TECH

Bluestar Genomics Receives CAP Accreditation for Its Clinical Laboratory

Bluestar Genomics | October 11, 2022

Bluestar Genomics, Inc., an early cancer detection company leading the development and commercialization of next-generation liquid biopsy tests focused on non-invasive detection of high-mortality cancers in high-risk patient populations, today announced it received accreditation for its clinical laboratory from the College of American Pathologists (CAP). The certification enables the company to serve physicians and patients globally as it works towards broad commercialization of its first test for the detection of pancreatic cancer. CAP Accreditation is an internationally recognized program that awards laboratories that meet stringent requirements of quality, accuracy, and consistency. This milestone further solidifies Bluestar Genomics’ commitment to providing the highest standards of testing results. The company’s proprietary approach to detecting cancer early is anchored in epigenomic process of tracking dynamic changes in cells in the body by measuring levels of the biomarker 5-hydroxymethylcytosine (5hmC) in a person’s blood. “This accreditation—on the heels of our CLIA certification and combined with our growing body of scientific evidence supporting the utility of our 5hmC-based epigenomic testing—positions us to become the first cancer detection company to offer non-invasive epigenomic blood-based testing for early pancreatic cancer detection. These milestones are a testament to our focus on preparing our company to serve customers around the world, helping millions of patients avoid late-stage, untreatable cancer diagnosis.” David Mullarkey, chief executive officer of Bluestar Genomics Bluestar Genomics’ pancreatic cancer early detection test has been analytically validated and previously received FDA Breakthrough Device Designation. It is designed to analyze a person’s changing biology and uses novel 5hmC-based epigenomic analysis to detect when cells become cancerous. The company has a prospective, large clinical validation study underway to further study its test. About Bluestar Genomics Bluestar Genomics is an early cancer detection company focused on the development and commercialization of non-invasive epigenomic tests to detect cancer through a standard blood draw, earlier than existing methods and when the disease is still treatable. The company uses its one-of-a-kind epigenomic platform that combines best-in-class bioinformatics and genomic technologies to analyze individuals’ changing biology that is yet to produce symptoms. Leveraging its novel liquid biopsy technology, Bluestar Genomics is initially focused on high-mortality cancers with the greatest need for early detection, starting with pancreatic and women’s cancers. With locations in San Diego and the San Francisco Bay Area, Bluestar Genomics collaborates with top research institutions and is supported by multiple global healthcare and technology investors and pharmaceutical collaborations.

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PHARMACY MARKET

ICON selected by BARDA to conduct anthrax vaccine clinical trial

ICON plc | October 07, 2022

ICON plc. a world-leading healthcare intelligence and clinical research organisation, announces that it has been selected by the US Biomedical Advanced Research and Development Authority part of the Administration for Strategic Preparedness and Response in the Department of Health and Human Services to execute an anthrax vaccine clinical trial. BARDA is providing funding to the Government and Public Health Solutions team at ICON to develop and execute a clinical trial that will evaluate the immune response to the next-generation anthrax vaccine AV7909. The team will support the evaluation of AV7909 immunogenicity kinetics by conducting clinical trial activities including but not limited to project management, clinical operations and quality assurance. ICON has extensive experience in supporting vaccine clinical trials. It has participated in over 160 vaccine studies in the past five years. ICON’s vaccine clinical research professionals are therapeutically aligned with a leadership team that brings more than 200 years of combined experience in vaccine clinical development for commercial businesses, government, NGOs and multinational health organisations. “We are pleased to have been selected to execute this important clinical trial. The potential for an anthrax attack is still a very real and serious threat, and we are proud to be playing our part to increase the understanding of treatment options for anthrax exposure, ultimately supporting human health.” Edward Wright, President, ICON Government and Public Health Solutions This clinical study will inform operational logistics and use of AV7909 should an anthrax emergency occur. The clinical studies will not expose participants to anthrax. The trial will enrol healthy adults aged 18 and older and will be conducted in the United States starting in early 2023. BARDA, part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services, is responsible for developing medical countermeasures to secure the U.S. against chemical, biological, radiological, and nuclear threats, as well as pandemic influenza and emerging infectious diseases. Since 2014, ICON has been a member of BARDA’s Medical Countermeasures Clinical Studies Network (CSN). The network is responsible for planning and executing clinical studies to support medical countermeasure development. Bacillus anthracis, the agent that causes anthrax, is a serious bioterrorism threat due to the delivery of aerosolised spores that can cause high-mortality disease through gastrointestinal, cutaneous, inhalational or injection routes. Although clinical manifestations of the disease differ by route, inhalational anthrax is the most lethal. Inhalational anthrax infection usually develops within a week after exposure but can take up to two months. Without treatment, only about 10% to 15% of patients survive after exposure to inhaled B. anthracis spores. Since 2015, BARDA has supported the late-stage development of AV7909, a next-generation anthrax vaccine which uses the U.S. Food and Drug Administration licensed anthrax vaccine Biothrax in combination with an immune system stimulant or adjuvant. The vaccine is being developed in partnership with its manufacturer Emergent Biosolutions Inc. to protect against anthrax post-exposure when used in combination with the recommended antibiotics. About ICON plc ICON is the world’s leading clinical research organisation, powered by healthcare intelligence. From molecule to medicine, we advance clinical research providing outsourced development and commercialisation services to pharmaceutical, biotechnology, medical device and government and public health organisations. We develop new innovations, drive emerging therapies forward and improve patient lives. With headquarters in Dublin, Ireland, ICON employed approximately 40,500 employees in 119 locations in 53 countries as at June 30, 2022. About ICON Government & Public Health Solutions ICON Government & Public Health Solutions is ICON's business unit focused on direct US Government engagement, Non-Government Organisations (NGOs) and academia, providing full service and functional research solutions. In addition, ICON GPHS coordinates a Government Centre of Excellence (CoE) to lend internal federal government subject matter expertise that can be leveraged by other ICON business units when commercial clients receive federal funding on ICON awarded contracts. About BARDA’s Medical Countermeasures Clinical Studies Network (CSN) The CSN provides BARDA partners and medical countermeasure developers with essential tools and services to develop vaccines, therapeutics, diagnostics, and medical devices. The CSN provides comprehensive (Phases 1-4) clinical study services.

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Custom ICM solutions are built for you, not auditors, so they typically don’t offer visibility into actions performed over time. Auditors may lack confidence in your homegrown system, especially if they find errors or omissions. In fact, audit risk from errors and overpayments is often the most compelling reason we see customers switch from build to buy. New regulations place additional burdens on your commissions system.

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