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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INDUSTRIAL IMPACT
PRNewswire | May 30, 2023
Guardant Health, Inc. announced that Singapore's Health Sciences Authority has granted regulatory approval of Guardant360® CDx, a liquid biopsy test for tumor mutation profiling, also known as comprehensive genomic profiling in patients with advanced solid cancers. The Guardant360 CDx test was also approved as a companion diagnostic to identify patients with advanced non-small cell lung cancer with epidermal growth factor receptor alterations who may benefit from treatment with TAGRISSO®.
Guardant360 CDx is the first blood test to be approved by Singapore's HSA for comprehensive genomic profiling for all solid tumors. Since being introduced as a laboratory developed test the Guardant360 liquid biopsy has become widely accepted for blood-based CGP with more than 400 peer-reviewed publications.
"We are delighted to receive regulatory approval from HSA for our Guardant360 CDx test, as it supports the value liquid biopsy brings to oncologists and the patients they treat," said Simranjit Singh, CEO of Guardant Health AMEA. "With a simple blood draw from the patient, the Guardant360 CDx test provides CGP results in approximately seven days from receipt of the blood sample in the laboratory, enabling physicians to make faster treatment decisions compared to tissue biopsies. Our hope is that this approval will drive clinical adoption of liquid biopsy testing in Singapore and enable more patients to be matched to potentially life-changing precision medicines."
In 2020, over 23,600 people in Singapore were diagnosed with cancer and there were slightly over 12,000 cancer-related deaths. The most prevalent cancer types among men and women in Singapore include breast (15.5%), colorectal (15.1%) and lung (12.3%) cancer[1]. According to the Singapore Cancer Registry Annual Report 2020, over 70% of all lung cancer cases among men and women were diagnosed at the advanced stage[2]. Pancreatic, stomach and colorectal cancer cases among men and women were also diagnosed at the advanced stage respectively To improve cancer outcomes in Singapore, Guardant Health AMEA is currently collaborating with National Cancer Centre Singapore and National University Cancer Institute, Singapore for several clinical trials using the Guardant360 test in efforts to accelerate clinical trial enrollment by identifying genomic biomarkers in patients with cancer.
The Guardant360 CDx test was the first blood test to be approved by the U.S. Food and Drug Administration (FDA) for comprehensive genomic profiling for all solid tumors, and it is now FDA approved as a companion diagnostic test for four targeted therapies in NSCLC and one in advanced breast cancer. In March 2022, the test was also granted regulatory approval by Japan's Ministry of Health, Labour and Welfare for tumor mutation profiling in patients with advanced solid cancers as well as a companion diagnostic to identify patients with microsatellite instability-high (MSI-High) solid tumors who may benefit from Keytruda® and patients with MSI-High advanced colorectal cancer who may benefit from Opdivo®. In addition, Guardant Health is currently involved in multiple studies with drug development companies to develop Guardant360 CDx as a companion diagnostic for new therapies.
About Guardant Health
Guardant Health AMEA is a wholly owned subsidiary of Guardant Health, Inc., a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary tests, vast data sets and advanced analytics. The Guardant Health oncology platform leverages capabilities to drive commercial adoption, improve patient clinical outcomes and lower healthcare costs across all stages of the cancer care continuum. Guardant Health has commercially launched Guardant360®, Guardant360® CDx, Guardant360 TissueNext™, Guardant360 Response™, and GuardantINFINITY™ tests for patients with advanced-stage cancer, and Guardant Reveal™ for patients with early-stage cancer. The Guardant Health screening portfolio, including the commercially launched Shield™ test, aims to address the needs of individuals eligible for cancer screening.
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MEDICAL, INDUSTRY OUTLOOK
Globenewswire | April 25, 2023
Design Therapeutics, Inc. a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that preclinical data for the company’s novel GeneTAC™ small molecule, DT-168, an eye drop being developed for the treatment of Fuchs endothelial corneal dystrophy (FECD), will be presented during an oral session at the Association for Research in Vision and Ophthalmology 2023 Annual Meeting (ARVO 2023), which is being held in New Orleans from April 23-27, 2023.
FECD is characterized by progressive corneal degeneration leading to vision loss and affects millions of people in the U.S. Approximately 75% of cases are caused by a mutation in the transcription factor 4 (TCF4) gene, consisting of a CTG trinucleotide repeat expansion that leads to the formation of pathogenic RNA foci in the nucleus and the mis-splicing of multiple transcripts. There are no disease-modifying therapies approved for FECD, and advanced cases generally require ocular surgery, including corneal transplant.
DT-168 is designed to selectively target the expanded CTG repeats in the TCF4 gene to reduce RNA foci formation and mis-splicing. In preclinical studies, DT-168 reduced foci in patient-derived primary corneal endothelial cells (CECs) to levels seen in cells from healthy individuals with low nanomolar IC50 values. Treatment with DT-168 also significantly improved mis-splicing in patient-derived CECs across a panel of genes. Additionally, in animal studies DT-168 eye drops were well-tolerated after multiple doses and distributed throughout the cornea with micromolar levels of DT-168 observed in the cornea 24 hours after dosing.
The preclinical data support the potential for DT-168 to address the most common genetic cause of FECD and support the continued development of DT-168 as a potential disease-modifying therapy. Design remains on-track to submit an Investigational New Drug application for DT-168 for the treatment of FECD in the second half of 2023.
About Design Therapeutics
Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC™ gene targeted chimera small molecules. The company’s GeneTAC™ molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. Design is currently evaluating its lead GeneTAC™ small molecule, DT-216, in an ongoing Phase 1 clinical trial in patients with Friedreich ataxia. The company is also advancing programs in Fuchs endothelial corneal dystrophy and myotonic dystrophy type-1.
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