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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Professor Magdalena Zernicka-Goetz and her team at the University of Cambridge have developed a way to grow embryos in vitro for up to 14 days, double the time it has previously been possible to keep the embryos alive in the lab. This will allow scientists to study embryos past the time they would normally implant in the womb, giving invaluable insight into the development of the early embryo.


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IAMA Therapeutics Partners with the Italian Institute of Technology to Embark on Landmark Research on Neuroscience Drug Discovery

IAMA Therapeutics | March 31, 2022

IAMA Therapeutics, a Series A preclinical-stage pharmaceutical company focused on the discovery, development, and commercialization of novel medicines to make a difference in the lives of individuals suffering from brain disorders, today announced a strategic collaboration with the IIT-Istituto Italiano di Tecnologia where they will design and profile new classes of compounds targeting cation cotransporters in the CNS area. This partnership consolidates a scientific collaboration between IAMA Therapeutics and IIT, which signed, together with the Telethon Foundation earlier in 2021, a license agreement, granting IAMA therapeutics an exclusive, worldwide license to research, develop, manufacture, and commercialize a novel class of modulators of intracellular chloride concentration. "We are pleased with and excited by this partnering opportunity with IAMA Therapeutics, a clear example of IIT technology transfer approach. We are setting the Italian way of producing science and tech-based innovation, where the collaboration among brilliant researchers, important founding agencies like Fondazione Telethon, and investors creates the right ecosystem enabling new businesses to emerge." Dr. Matteo Bonfanti, Director of Technology Transfer at Istituto Italiano di Tecnologia "We are very happy for the partnership between IAMA Therapeutics and IIT," said Francesca Pasinelli, General Manager of Fondazione Telethon. "This collaboration is based on innovative science, which was selected and nurtured earlier on by Fondazione Telethon. As part of our focus on fostering development, we funded the project and supported researchers through the technology transfer process together with IIT. Now, this promising research avenue is accessing the resources and competencies necessary to take and accelerate the long and expensive path to patients. It shows what can be accomplished through a timely and coordinated effort to maximize research impact". About IAMA Therapeutics IAMA Therapeutics is a pharmaceutical company that couples emerging advances in drug discovery and neurobiology to build a leading, next-generation neuroscience pipeline. We have developed a platform to identify and advance diverse classes of small molecules to selectively inhibit the sodium potassium chloride cotransporter and other therapeutic targets relevant to treating central nervous system disorders characterized by neuronal imbalance, including primary and secondary autism, epilepsy, and movement disorders. About Istituto Italiano di Tecnologia Istituto Italiano di Tecnologia is a foundation established jointly by the Italian Ministry of Education, Universities, Research, and the Ministry of Economic and Finance to promote excellence in basic and applied research and contribute to Italy's economic development. The primary goals of the IIT are the creation and dissemination of scientific knowledge and the strengthening of Italy's technological competitiveness. To achieve these two goals, the IIT cooperates with academic institutions and private organizations, fostering scientific development, technological advances, and training in high technology. About Fondazione Telethon Fondazione Telethon is a major biomedical charity in Italy whose mission is to advance biomedical research to cure rare genetic diseases. Throughout its 32 years of activity, Fondazione Telethon has invested almost €600 million in funding over 2,700 projects to study 580 diseases, involving more than 1,600 researchers in Italy. As part of its goal to foster development and maximize the therapeutic impact of research, Fondazione Telethon further pursues cooperation with academia, industry, and venture capital through the creation of partnerships and start-ups.

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PHARMACY MARKET

BioMatrix Specialty Pharmacy Granted Preferred Status with AscellaHealth for Home Infusion Therapies

BioMatrix Specialty Pharmacy | February 09, 2022

BioMatrix Specialty Pharmacy announced inclusion as a preferred provider in AscellaHealth's home infusion pharmacy network. AscellaHealth, a global specialty pharmacy and healthcare solutions company, has launched a site of care optimization strategy designed to cut costs for third-party payers without compromising quality of care. BioMatrix was included in the network to help the organization achieve its goals of improving drug affordability; increasing price transparency; reducing disparities in quality of care and safety; enabling care delivery in the highest-quality, lowest-cost care setting; and increasing patient convenience. Determining the optimal site of care for patients to receive therapy is an important component of treatment success. BioMatrix Specialty Pharmacy provides robust specialty infusion services, including site of care coordination. Whether administering in-home with assistance from one of our home infusion nurses, in-office, or at an ambulatory infusion center, our clinical team works with patients, payers, and prescribers to make therapy administration as safe and convenient as possible. Using a nationwide network of highly trained specialty infusion nurses, BioMatrix coordinates over 1,200 home nursing visits every month. "We are pleased to be included as a preferred provider in AscellaHealth's home infusion network. Our shared focus on positive outcomes for all patients, reduced cost, and price transparency will help AscellaHealth's members effectively manage their specialty infusion spend without compromising quality care." Ken Trader, BioMatrix VP of Managed Markets "By selecting top pharmacies for preferred status, we are taking an important step toward reducing the cost of specialty biologic medications and encouraging patients to move from higher-cost settings, like big hospitals and institutions, to lower-cost settings, such as physician's in-office infusion suites or stand-alone infusion centers," says Dea Belazi, president and CEO, AscellaHealth. "As part of our mission to help our clients better manage the high costs of specialty medication, this initiative exemplifies our efforts to increase care accessibility by encouraging the use of clinically appropriate, lower-cost care settings." About BioMatrix Specialty Pharmacy BioMatrix Specialty Pharmacy, an Inc. 5000 company, offers comprehensive, nationwide specialty pharmacy services and digital health technology solutions for patients with chronic, difficult to treat conditions. Our commitment to every patient is to provide individualized pharmacy services, timely access to care, and focused education and support. We offer a tailored approach for a wide range of therapeutic categories, improving health and empowering patients to experience a higher quality of life. About AscellaHealth LLC AscellaHealth is a global Specialty Pharmacy and Healthcare services organization serving patients, payers, life sciences and providers, offering a comprehensive portfolio of customized, tech-enabled specialty pharmaceutical and medical management services. An Inc. 5000 2021 winner, AscellaHealth's unique, patient-centric approach is built upon proprietary technology processes for innovative programs and services optimizing health outcomes and quality of life for patients with complex chronic conditions or rare diseases that require specialty medications and/or gene and cell therapies.

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IACTA Pharmaceuticals and TALLC Corporation Announce Strategic Collaboration to Utilize Novel SmartCelle Topical Delivery Technology

IACTA Pharmaceuticals, Inc. | December 16, 2021

IACTA Pharmaceuticals, Inc. an innovation leader in ophthalmic therapeutics, and TALLC Corporation Inc. a pioneering ophthalmology company focused on the growing unmet needs of patients with corneal and retinal conditions, announced that they are entering into a strategic collaboration and licensing agreement. The agreement grants IACTA the use of TALLC’s proprietary SmartCelle™ micellar technology, a novel platform for pan-ocular drug delivery, for future clinical development. IACTA plans to utilize TALLC’s SmartCelle technology to deliver one or more of its novel ophthalmic therapeutics, as well as co-develop new ophthalmology therapeutic candidates that leverage IACTA’s formulation and clinical expertise with TALLC’s novel delivery technology. The collaboration also includes an option agreement, granting IACTA exclusive rights to negotiate a separate license agreement for the right to co-develop TALLC’s lead product candidate TA-A001, a novel anti-inflammatory anti-angiogenic molecule with analgesic properties in various disease state models. “I am thrilled to be able to partner with TALLC, a company with a shared vision focused on addressing the growing and significantly underserved ocular pain market. IACTA was formed with the mission of developing and commercializing innovative ocular therapies through novel compounds and utilizing leading drug delivery technologies, and we view this collaboration as the perfect synergy to realize the full potential of our ophthalmology pipeline. We are excited to explore how best to deploy the SmartCelle technology within our current pipeline, as well as the future co-development opportunities this partnership provides.” Damon Burrows, Chief Executive Officer of IACTA Pharmaceuticals TALLC’s proprietary SmartCelle pan-ocular drug delivery technology employs 30nm micelles that enable delivery of difficult to formulate drugs into corneal and retinal tissues after topical delivery. The company’s pipeline is led by SmartCelle TA-A001, a novel CB2 receptor activator, delivered topically to treat the effects of dry eye syndrome and age-related macular degeneration. “We are delighted to partner with the IACTA team for this important program. IACTA, with its in depth scientific and commercial understanding of the ophthalmic space is an ideal partner for TALLC as we drive towards further commercialization of the SmartCelle platform and TA-A001,” said Damon Smith, Ph.D., Chief Executive Officer of TALLC. “We look forward to working closely with the IACTA team and to co-developing SmartCelle versions of the IACTA pipeline.” About TALLC, Inc. TALLC is a Montreal-based developer of ophthalmic medicines actively engaged in the commercialization of SmartCelle TA-A001, and its pipeline of SmartCelle-delivered drug candidates, as treatments for acute and chronic inflammation. Focused initially on unmet needs in inflammatory front of eye indications, TALLC plans also to develop SmartCelle TA-A002 and TA-A003 in other chronic conditions including macular degeneration and glaucoma. SmartCelle enables pan ocular delivery of drug candidates by topical application directly to the ocular surface, by injection or by insufflation. About IACTA Pharmaceuticals, Inc. IACTA is an innovation leader in ophthalmic therapeutics, led by former top executives from one of the leading eye care companies in the world. The company currently has four products in development for major market opportunities. IC 800 is a novel, non-opioid, dual enkephalinase inhibitor, being studied for the treatment of acute ocular pain. In addition, IACTA’s IC 265, is a SYK kinase inhibitor being developed as a potential novel treatment for dry eye disease.

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PHARMACY MARKET

Veneno Technologies, a Novel DRP Drug Discovery Company, Raises $2Million (¥200M Yen) in Seed Funding Round

Veneno Technologies Co. Ltd. | January 27, 2022

Veneno Technologies Co. Ltd. is pleased to announce that it has completed fundraising of 200 million yen through a third-party allocation of new shares to SBI Investment Co., Ltd., Tsukuba Institute of Research, Ltd., and SBI regional activation support Co., Ltd. The funds raised will be used to strengthen the recruitment and organizational structure, to further develop the Company's proprietary peptide drug discovery platform technology, and invest in the research and development of the Company's own drug discovery pipeline using DRP functional peptides as basic molecules to further promote DRP drug discovery. Objective of the Fund Procurement Veneno Suite™ is the Company's proprietary integrated DRP drug discovery technology. The Veneno Suite™ consists of a large gene library that is created by artificially accelerating the evolution of natural DRPs using them as templates, a screening system that enables the search for target DRPs from the library in a fast and efficient manner, and technology that enables the efficient production of various DRPs in a short period of time. With this funding, we will further refine these technologies and accelerate DRP drug discovery. Our mission is to contribute to the advancement of medicine by providing new drugs for targets such as membrane proteins that have been difficult to discover, and for intractable diseases related to these targets. In order to fulfill this mission, we will strengthen the following points through this fundraising. Appointment of high-level researchers and opening of new laboratories to advance DRP drug discovery. Expansion of the DRP focused library (DRP Space™). Strengthen and expand the pipeline of the DRP rapid search system (PERISS™) (promote joint research). Development of DRP manufacturing technology (Super Secrete™) including establishment of high-efficiency, low-volume, high-mix manufacturing technology. This funding will enable us to further refine our proprietary large-scale library, high-speed screening technology, and high-efficiency DRP manufacturing technology to accelerate our goal of DRP drug discovery. We expect that we will be able to take a concrete step toward the establishment of a new paradigm using DRP as a new drug discovery platform molecule following antibody drugs. Investor Comments Mr. Makoto Hasegawa, Manager, Investment Division, SBI Investment Co. noted, "Veneno Technologies has developed an innovative technology to turn functional peptides, called DRPs, into pharmaceuticals. We have decided to invest in Veneno Technologies because we see the potential in their technology and want to take on the challenge of developing new modalities together. We hope that our technology will lead to the development of many innovative new drugs in the future." "Veneno Technologies is a drug discovery venture that uses this technology to develop drugs that target ion channels, which remain untapped as drug targets." Mr. Masato Suzuki, Senior Researcher, Consulting Department, Tsukuba Research Institute, Inc About Veneno Technologies, Inc. Veneno Technologies was established to accelerate the research and development of DRP functional peptides and to contribute to advanced and sustainable medicine and society. The company was established to accelerate the research and development of DRP functional peptides and to contribute to advanced and sustainable medicine and society. By integrating innovative DRP discovery technologies that have been researched for many years at the National Institute of Advanced Industrial Science and Technology (AIST) and DRP manufacturing technologies that are currently being researched and developed, we will lead the research and development of various DRP drug discovery technologies such as new drugs, research reagents, agrochemicals and biostimulants.

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Professor Magdalena Zernicka-Goetz and her team at the University of Cambridge have developed a way to grow embryos in vitro for up to 14 days, double the time it has previously been possible to keep the embryos alive in the lab. This will allow scientists to study embryos past the time they would normally implant in the womb, giving invaluable insight into the development of the early embryo.

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