MEDTECH, INDUSTRIAL IMPACT
Globenewswire | April 13, 2023
Centogene N.V. the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced the launch of NEW CentoGenome®, an enhanced Next Generation Sequencing (NGS)-based assay. Healthcare professionals can leverage NEW CentoGenome to provide more comprehensive diagnostic information, which could accelerate access to potential treatment options.
Serving as a first-line test, NEW CentoGenome is the most comprehensive commercially available Whole Genome Sequencing (WGS) test on the market for both rare and neurodegenerative disorders – covering almost all disease-causing variants, including the most relevant repeat expansions associated with neurological diseases, in a single assay. NEW CentoGenome also detects Copy Number Variations (CNVs) associated with Spinal Muscular Atrophy (SMA), as well as complex disease-causing variants associated with Gaucher Disease (GD) and susceptibility to GBA1-related Parkinson's Disease (PD), with the highest levels of sensitivity.
The newly designed WGS assay leverages a Polymerase Chain Reaction (PCR)-free approach. This significantly diminishes the typical bias induced by PCR and provides higher quality sequencing data across the typically challenging regions of the genome by enabling more uniform coverage and superior performance in variant detection.
“As a pioneer of genetic diagnostics, we are committed to enabling access to high-quality diagnostics and ensuring that cutting-edge data analyses are available to support better patient health outcomes,” said Prof. Peter Bauer, Chief Medical and Genomic Officer at CENTOGENE. “Building on this expertise, CENTOGENE’s enhanced whole genome sequencing is the leading solution on the market, reflecting the latest advanced technologies and unique insights that can’t be found anywhere else to provide maximized disease coverage. Ultimately, this will provide physicians with an unparalleled level of certainty when diagnosing, prognosing, and treating patients.”
CENTOGENE’s NEW CentoGenome - Superior Technology With Market-Leading Clinical Coverage in a Single Test
CENTOGENE’s enhanced WGS solution consists of three seamlessly integrated key features
Advanced Technology for Greater Insights
Implementing PCR-free technology to significantly reduce bias and provide high-quality sequencing information for difficult-to-sequence genetic regions – enabling greater insights into coding, regulatory, and intronic regions
Superior Performance for Enhanced Disease Coverage
Delivering superior performance in variant detection thanks to CENTOGENE’s automated CE-IVD bioinformatics pipeline and medical expert-based analysis of the CENTOGENE Biodatabank, which consists of approximately 700,000 patients representing over 120 highly diverse countries
Integrated Variant Reclassification and Confirmatory Testing for a Life-Long Commitment to Patients
Demonstrating CENTOGENE’s unique commitment to improving the lives of patients with rare and neurodegenerative diseases, NEW CentoGenome is paired with life-long diagnostic support and a free-of-charge and proactive diagnosis confirmation and reclassification program
About CENTOGENE
CENTOGENE’s mission is to provide data-driven, life-changing answers to patients, physicians, and pharma companies for rare and neurodegenerative diseases. We integrate multiomic technologies with the CENTOGENE Biodatabank – providing dimensional analysis to guide the next generation of precision medicine. Our unique approach enables rapid and reliable diagnosis for patients, supports a more precise physician understanding of disease states, and accelerates and de-risks targeted pharma drug discovery, development, and commercialization.
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INDUSTRIAL IMPACT, DIAGNOSTICS
Vaxxinity, Inc. | March 21, 2023
Vaxxinity, Inc. a U.S. company pioneering the development of a new class of immunotherapeutic vaccines, announced that the first subjects have been dosed in a randomized, double-blind, placebo-controlled Phase 1 clinical trial of VXX-401, an investigational vaccine designed to lower low-density lipoprotein (LDL) cholesterol, a known factor contributing to heart disease, by targeting proprotein convertase subtilisin/kexin type 9 serine protease (PCSK9). Heart disease remains the leading cause of death globally, claiming over 18 million lives per year, despite the existence of approved treatments that lower LDL.
“This is an exciting milestone for VXX-401 and Vaxxinity in our pursuit to vaccinate the world against heart disease with a preventative option that is convenient and accessible, addressing an unmet need to combat the leading global cause of death,” said Mei Mei Hu, Chief Executive Officer of Vaxxinity. “PCSK9 antibody therapies are well-tolerated and effective, but huge unmet patient need remains. In order to solve the problem of heart disease, the world needs a scalable, accessible technology that can reach the hundreds of millions, if not billions of people at risk. With an LDL-lowering vaccine we can potentially offer an option that’s cost-effective, safe, convenient, long-acting, and deployable.”
The multicenter Phase 1 dose-escalation trial aims to enroll 48 subjects aged 18 to 75 years, with LDL cholesterol between 2.59 and 4.89 mmol/L. The objectives of the trial are to evaluate safety, tolerability and immunogenicity (as measured by serum anti-PCSK9 antibody titers). Pharmacodynamics of the immune response will be measured by LDL cholesterol levels, an established model of PCSK9 inhibition in hypercholesterolemia.
“We’re excited to get this first-in-human trial of VXX-401 started. The concept of a vaccine for cholesterol could be a game-changer in cardiovascular health due to its potentially very broad reach and impact on human health,” said Professor Stephen Nicholls of Monash University and Victorian Heart Hospital in Australia. “Targeting PCSK9 with a monoclonal antibody is a proven and effective approach for lowering cholesterol and reducing the risk of heart attack and stroke. Despite the availability of statins and the approval of PCSK9-targeting medicines, there is still a need for new therapies. VXX-401 has the potential to extend the use of PCSK9 inhibition to the full population who could benefit.”
Earlier this month, Vaxxinity presented pre-clinical data at the American College of Cardiology/World Congress of Cardiology Annual Scientific Session demonstrating that VXX-401 induced robust anti-PCSK9 antibodies and durable LDL cholesterol lowering without affecting high-density lipoprotein (HDL) cholesterol levels, indicating that VXX-401 has the potential to be a safe and effective LDL lowering therapy. Data in non-human primate studies show that VXX-401 was well tolerated and provided durable and significant LDL reduction of 30% to 50% change from baseline. The data also demonstrate VXX-401’s immunogenicity: purified antibodies from immunized animals bound to human PCSK9 with high affinity, and exhibited a dose-dependent functional inhibition of PCSK9 in vitro. Additionally, Peripheral Blood Mononuclear Cells (PBMCs) collected prior to immunization did not release cytokines upon stimulation with various components of the vaccine, suggesting that VXX-401 may safely overcome immune tolerance.
About VXX-401
VXX-401 was designed using Vaxxinity’s proprietary synthetic peptide vaccine platform and is being developed for the treatment of hypercholesterolemia. The platform is designed to harness the immune system to convert the body into its own natural “drug factory,” stimulating the production of antibodies. VXX-401 is designed to induce robust, long-acting antibodies against PCSK9 in order to lower LDL cholesterol.
About Vaxxinity
Vaxxinity, Inc. is a purpose-driven biotechnology company committed to democratizing healthcare across the globe. The company is pioneering a new class of synthetic, peptide-based immunotherapeutic vaccines aimed at disrupting the existing treatment paradigm for chronic disease, increasingly dominated by monoclonal antibodies, which suffer from prohibitive costs and cumbersome administration. The company’s proprietary technology platform has enabled the innovation of novel pipeline candidates designed to bring the efficiency of vaccines to the treatment of chronic diseases, including Alzheimer’s, Parkinson’s, migraine, and hypercholesterolemia. The technology is also implemented as part of a COVID-19 vaccine program. Vaxxinity has optimized its pipeline to achieve a potentially historic, global impact on human health.
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INDUSTRY OUTLOOK
Globenewswire | May 15, 2023
TriLink BioTechnologies a Maravai LifeSciences company and global provider of life science reagents and services, has launched its newest cap analog, CleanCap® M6 . The latest CleanCap analog is expected to help developers and researchers maximize the impact of their mRNA therapeutics and vaccines while reducing overall manufacturing costs, bringing life-changing medicines to market faster.
With the introduction of the CleanCap M6 technology, legacy capping methods like enzymatic and ARCA may be a thing of the past. By combining the modification of 3’OMe on the m7-Guanosine and the additional methyl modification on the +1 Adenosine, the CleanCap M6 analog is expected to improve mRNA potency with the highest protein expression of any CleanCap analog yet. Combined with technology’s single-pot reaction capabilities that shrink manufacturing times and increase mRNA yields with a purity profile of >95%, CleanCap analogs are novel mRNA Cap structures, which may improve the benefits of an mRNA therapeutic or vaccine.
“TriLink’s portfolio of CleanCap mRNA capping technology is expected to be the new go-to capping method in the very near future - it is the future,” shared Kate Broderick Ph.D., Chief Innovation Officer at Maravai LifeSciences. “What excites me most about this novel analog is its ability to make mRNA an even more powerful therapeutic than we have seen previously.”
The company’s first CleanCap analog launched in 2017 and this capping technology has been incorporated in one of the first commercially approved COVID-19 vaccines. With the introduction of CleanCap M6, TriLink has developed its most robust analog to date. Studies have shown that the innovative product increases mRNA expression by 30% or more versus enzymatic capping methods and is estimated to reduce manufacturing costs by 20-40%.
“mRNA is clearly an emerging therapeutic modality where mRNA vaccines and therapeutics are expanding rapidly,” added Drew Burch, EVP of TriLink’s Nucleic Acid Products division. “CleanCap M6, and the entire CleanCap portfolio, joins a strong roster of synthesized nucleic acids, NTPs, and more that we expect to continue to grow.”
In addition to the CleanCap mRNA capping portfolio, TriLink has scale-up expertise and unique mRNA, oligonucleotide & plasmid production capabilities for companies focused on therapeutic, vaccine, diagnostic breakthroughs, leaning on its 25-plus years of experience to provide superior CDMO services.
Data and customer insights garnered from a market assessment conducted by a third-party consulting firm on behalf of TriLink BioTechnologies in November 2022.
About Maravai
Maravai is a leading life sciences company providing critical products to enable the development of drug therapies, diagnostics, and novel vaccines. Maravai’s companies are leaders in providing products and services in the fields of nucleic acid synthesis and biologics safety testing to many of the world’s leading biopharmaceutical, vaccine, diagnostics, and cell and gene therapy companies.
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