INDUSTRIAL IMPACT, MEDICAL
prnewswire | April 04, 2023
Sensible Biotechnologies, an early stage biotechnology company, today announced a partnership to develop an in vivo microbial mRNA manufacturing platform with Ginkgo Bioworks which is building the leading platform for cell programming and biosecurity.
Today, the vast majority of mRNA used in vaccines, therapeutics and other applications is produced by in vitro transcription (IVT), a cell-free process, which is driven by purified enzymes. In conventional mRNA manufacturing, production runs are typically limited in reaction volume size, and the resulting mRNA needs to go through expensive purification processes to eliminate potentially harmful byproducts like dsRNA, which can cause adverse immune responses in patients. Moreover, it is difficult to produce high yields of certain kinds of mRNA in an IVT reaction. By contrast, the in vivo mRNA manufacturing method Sensible and Ginkgo are working to develop is designed to scale to upwards of 100,000L, with the goal of superior quality mRNA over traditional IVT, allowing for the production of mRNA molecules with increased length and expanding the potential of the mRNA platform to novel therapeutic modalities.
"mRNA technology has a potential to bring many life-saving therapeutics and vaccines, but its current, cell-free production represents one of the major bottlenecks. In vivo mRNA manufacturing could enable scalable mRNA manufacturing, which has long relied on production methods that face quality control challenges and are inherently difficult to scale," said Miroslav Gasparek, CEO at Sensible. "By working with Ginkgo, we aim to create a scalable commercial-grade manufacturing platform that produces mRNA of higher quality than is possible through in vitro expression and enable the advent of novel mRNA medicines."
v"As the market for mRNA continues to expand, biopharma companies are looking for more efficient and scalable production platforms to produce high-quality mRNA," said Austin Che, co-founder and Head of Strategy at Ginkgo Bioworks. "As Ginkgo deepens its commitment to the emerging field of nucleic acid therapeutics, we are excited to work with the team at Sensible Biotechnologies to optimize their microbial mRNA production platform with the goal of unleashing a new generation of mRNA products."
This partnership with Ginkgo builds on Ginkgo's existing pipeline of mRNA-related programs. Ginkgo worked with Moderna to support process optimization for raw materials used to make mRNA vaccines. Furthermore, through its partnership with Aldevron, Ginkgo optimized the production of vaccinia capping enzyme, an important component often required to manufacture mRNA vaccines and therapeutics. Most recently, Ginkgo announced the acquisition of Circularis, a biotechnology company with a proprietary circular RNA and promoter screening platform, and is actively engaged in improving circular RNA efficacy and manufacturing yields.
About Ginkgo Bioworks
Ginkgo Bioworks is the leading horizontal platform for cell programming, providing flexible, end-to-end services that solve challenges for organizations across diverse markets, from food and agriculture to pharmaceuticals to industrial and specialty chemicals. Ginkgo's biosecurity and public health unit, Concentric by Ginkgo, is building global infrastructure for biosecurity to empower governments, communities, and public health leaders to prevent, detect and respond to a wide variety of biological threats.
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MEDICAL, INDUSTRY OUTLOOK
PRNewswire | May 17, 2023
REGENXBIO Inc. announced that preclinical research in Mucopolysaccharidosis type IVA also known as Morquio syndrome, was selected for inclusion in the Foundation for the National Institutes of Health Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC), clinical trial portfolio. The Consortium brings together partners such as NIH and FDA, as well as partners from private and non-profit sectors.
Sponsored by Nemours Children's Hospital, MPS IVA is one of eight programs selected as part of AMP® BGTC's first clinical portfolio to help accelerate the development of bespoke gene therapies, with the goal of streamlining the regulatory approval process. MPS IVA is a metabolic condition that primarily affects the skeleton, and is estimated to impact 1 in 200,000 to 300,000 individuals.
"I am proud of our scientists who led this important clinical research work for REGENXBIO. Our mission to deliver the curative potential of gene therapy reflects not only patients that can be impacted by our clinical pipeline, but also all patients who should have the opportunity to be positively impacted by gene therapy," said Kenneth T. Mills, President and CEO REGENXBIO. "As a partner of the BGTC, REGENXBIO is pleased to see this program advancing important science into the clinic."
Criteria for selection included the adequacy of the gene for insertion into an adeno-associated virus (AAV) vector, sufficient proof of concept and natural history data, the existence of an established disease model, a lack of available treatment and an overall readiness for entering into a clinical trial. REGENXBIO will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases. Use of the NAV® Technology Platform will help address the goal of making gene therapy more accessible by creating a platform approach with standardized processes to deliver novel therapies for many different genetic disorders. These programs together further validate the versatility of NAV® vectors and will provide additional data that collectively drive the advancement of the AAV gene therapy field.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
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INDUSTRIAL IMPACT
PRNewswire | May 22, 2023
Menarini Silicon Biosystems (MSB), a pioneer of liquid biopsy and single cell technologies, announced today its partnership with Alivio Health. This new agreement opens the door to preferred access for Alivio Health clients and providers to the valuable information offered by the CELLSEARCH CTC tests, which allow longitudinal monitoring of cancer patients at any time during the course of the disease. Starting from a simple blood draw, CELLSEARCH CTC tests provide valuable information on a patient's cancer at diagnosis and during therapy, which enables doctors to make more informed and timely decisions to optimize their therapeutic strategy.
According to Daryl Spinner Vice President, Market Access and Reimbursement at Menarini Group "we are delighted by this new partnership that makes it easier for Alivio Health's members to access and their physicians to order our tests. All members of Alivio Health can now benefit with preferred access and pricing from the improved and more rapid diagnostic information and therapeutic approaches these tests inform". Indeed, MSB's Gold Standard CELLSEARCH® CTC test is the first and only clinically validated, FDA-cleared test for capturing and enumerating circulating tumor cells (CTCs) in cancer patients with metastatic breast, prostate and colorectal cancer to help inform clinical decision-making. New CELLSEARCH LDTs (Laboratory Developed Tests) are also available to clinicians for CTC enumeration and HER2 and PD-L1 targeted therapy-associated biomarker analysis in patients with various solid tumors, as well as for patients with melanoma and multiple myeloma. The minimally invasive nature of the CELLSEARCH tests makes them convenient to monitor patients with serial CTC testing for real-time clinical information at any time during the course of their disease.
The new partnership will benefit the members of Alivio Health whose main mission is to facilitate access to the right test at the right time. Specifically, among the over 200,000 members of Alivio Health's participating health plans, patients with cancer will now have access to more accurate, timely, affordable and reimbursable MSB liquid biopsy tests, so that they may benefit from a more informed therapeutic strategy.
For Fabio Piazzalunga, President and CEO of MSB, "we are particularly excited by this opportunity to reach out to a broader pool of professionals and patients thanks to this new partnership. As we pursue our relentless endeavors to advance liquid biopsy tests that can aid in the diagnosis, prognosis, and treatment of patients, we are also eager to ensure that our technology is accessible to the clinicians who can prescribe them". For both MSB and Alivio Health, this partnership will contribute to their common and primary ambition, which is to expand access to innovative testing approaches that allow providers to better understand the disease dynamics of their patients and therefore offer improved care to patients.
About Menarini Silicon Biosystems (MSB)
MSB offers unique rare-cell technologies and solutions that provide clinical researchers with access to unparalleled resolution in the study of cells and their molecular characterization.
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