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.
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.
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.
MEDTECH, INDUSTRIAL IMPACT
MaxCyte | January 04, 2023
MaxCyte, a leading commercial cell-engineering company, announced the signing of a strategic platform license (SPL) with Catamaran Bio, a biotechnology enterprise creating off-the-shelf chimeric antigen receptor (CAR)-NK cell therapeutics to cure a wide range of cancers.
While Catamaran acquires non-exclusive clinical and commercial rights to use MaxCyte's Flow Electroporation® technology and ExPERT™ platform per the terms of the agreement, in exchange, MaxCyte will receive platform licensing fees and revenue related to its programs.
Catamaran is actively developing highly potent allogeneic, cryopreserved CAR-NK cell therapeutics using synthetic biology and non-viral cell engineering capable of treating challenging cancers, including solid tumors, by integrating new functional features with the inherent cancer-fighting properties of NK cells. In addition, the company created the TAILWIND® platform, an integrated set of technologies, to engineer, expand, and process NK cells into safe and effective off-the-shelf cell therapy products for numerous cancer types.
The MaxCyte ExPERT™ instrument portfolio is the next generation of clinically verified electroporation technology for complicated and scalable cell creation. The ExPERT™ platform provides the high-end performance required to enable the next era of biological and cellular treatments by delivering high transfection efficiency, seamless scaling, and expanded functionality. MaxCyte's Catamaran agreement is the company's 19th overall SPL.
MaxCyte is a prominent commercial cell-engineering firm dedicated to advancing breakthrough cell-based research and next-generation cell therapy discovery, development, and commercialization. Over the years, it has been developing and commercializing its unique Flow Electroporation® platform, which allows for the intricate creation of a wide range of cells. In addition, its ExPERT™ platform, based on its Flow Electroporation technology, has been created to assist the rapidly increasing cell therapy market and can be used across the high-growth cell therapy sector, from discovery and development to commercialization of next-generation cell-based therapies.
INDUSTRIAL IMPACT, MEDICAL
Twist Bioscience | March 10, 2023
On March 9, 2023, Twist Bioscience Corporation, a leading firm providing high-quality synthetic DNA through its silicon platform, announced the launch of integrated antibody discovery services. This premium offering combines the company's synthetic libraries and AI machine learning with an in vivo immunization approach acquired through its acquisition of Abveris, also known as Twist Boston. This service provides customers with optimized, development-ready antibody candidates.
The combined team, now called Twist Biopharma Solutions, is led by Tracey Mullen, MBA, the senior vice president of biopharma for Twist Bioscience. This newly formed entity leverages years of industry expertise, scientific excellence, and proprietary technology to discover the best antibody candidate for therapeutic targets of interest.
Twist Biopharma Solutions offers an end-to-end workflow for centralized antibody discovery and optimization, including target validation, hit generation, lead selection, lead optimization, and lead characterization. Twist, supported by high-throughput DNA synthesis and IgG antibody production, constructs extensive and particular synthetic antibody libraries with discovery starting with either in vivo or in vitro diversity.
The newly integrated in vivo discovery approach from Abveris of single B cell screening and hybridoma discovery enables parallel paths where multiple technology methods can be leveraged to build a panel of highly diverse antibody leads with broad epitope coverage. In addition, this multi-pronged approach can increase the likelihood of discovering highly specific, high-affinity functional antibodies by sampling synthetic and natural diversity.
The South San Francisco and Boston biopharma teams were combined to create Twist Biopharma Solutions, led by Tracey Mullen. Aaron Sato, Ph.D., remains integrally involved in Twist Biopharma Solutions and, as a chief scientific officer of Twist Bioscience, extends his expertise across the organization. Tracey Mullen joined Twist Bioscience in November 2021 through the acquisition of Abveris. In April 2022, she took on the position of senior vice president of operations, driving the completion of the Factory of the Future in Wilsonville, Oregon, which began shipping products in January 2023. Before joining Twist, she served as CEO of Abveris and worked on the antibody discovery team at Biogen.
About Twist Bioscience
Twist Bioscience is a leading biotechnology company specializing in synthetic biology and genomics. It has developed a proprietary DNA synthesis platform that enables the production of custom-designed DNA strands faster, more accurately, and at a lower cost than traditional methods. In addition, Twist collaborates with partners across industries to develop customized DNA sequences for specific applications and offers a range of other products and services, such as oligonucleotides, synthetic genes, and custom panels for next-generation sequencing. The company has also developed a library of over 100 billion synthetic antibodies, which can be used for diagnostic and therapeutic applications.