INDUSTRY OUTLOOK

Andelyn Biosciences Selects TetraScience as a Strategic Partner for Innovation in Therapeutics Development and Manufacturing

Andelyn Biosciences | August 26, 2021

TetraScience, the R&D Data Cloud company, announced today that Andelyn Biosciences, Inc. has selected the Tetra Data Platform (TDP) to build a cloud-native development and manufacturing service for its customers. As part of an Andelyn Biosciences initiative to create a Connected Plant that harmonizes data from building and facilities, process and manufacturing, and lab instrument sources, TDP will enable better compliance, easier analysis, faster decisions, and more agile operations. Additionally, Andelyn customers will benefit from secure, convenient access to harmonized data that is prepared for analytics and visualization.

As a Contract Development and Manufacturing Organization (CDMO) for cell and gene therapies, Andelyn must remain GMP-compliant and increase efficiency while leading the industry in the innovative services they provide to customers. By launching a Connected Plant, Andelyn will gain autonomous operation, predictive capabilities, improved process monitoring and control, and increased data integrity.

"Andelyn has been a pioneer in gene therapy with a 15-year history accelerating the development and manufacturing of innovative therapies to bring more treatments to more patients," said Bryan J. Holmes, Vice President, Information Technology at Andelyn Biosciences. "Together with TetraScience, we're transforming how we work with our clients, utilizing fully-digitized, cloud-native services to help connect all the plant and lab data created during complex development and manufacturing cycles."

Andelyn chose TDP for its unique ability to harmonize data from building & facilities systems, lab instruments, and process & manufacturing equipment. By replatforming the data to a future-proof managed cloud environment, Andelyn will benefit from enhanced GMP compliance and data integrity, improved time-to-value, and lower TCO. Additionally, with an open data cloud platform powered by TDP and the Tetra Partner Network, Andelyn will be able to leverage their preferred tooling for analytics while supporting their customers' data consumption needs.

"We're excited to partner with Andelyn Biosciences on their mission-critical data cloud initiative and accelerate their journey towards becoming a digital company," stated Chad Garrett, TetraScience CRO. "As an innovator in providing cloud-native development and manufacturing services to its customers, Andelyn Biosciences recognizes that harmonizing scientific data in the cloud and making it easily accessible to clients will transform the way CDMOs exchange data with clients."

Andelyn's initial deployment of TDP is focused on acquiring and harmonizing historical data across key sources while enabling enhanced data search capabilities. Moving forward, Andelyn expects to complete the rollout of the Connected Plant with additional data sources and instruments, and ultimately launch a groundbreaking digital CDMO experience for their customers.

About Andelyn Biosciences, Inc.
Andelyn Biosciences is a pioneering gene therapy company born out of Nationwide Children's Hospital, where the first FDA-approved systemic gene therapy was discovered. Andelyn is named in honor of two gene therapy pioneers who participated in pivotal Phase I clinical trials at Nationwide Children's Hospital 8-year-old Andrew and 8-week-old Evelyn. Bringing together more than 15 years of experience in AAV vector manufacturing, Andelyn is a full-spectrum Contract Development Manufacturing Organization (CDMO) that helps pioneer solutions that turn hope into reality. Our mission is to accelerate the development and manufacturing of innovative therapies to bring more treatments to more patients. We accomplish this by bringing scalability, capacity, efficiency and expert resources to manufacturing matrices, all to advance the development of viral vector-based therapeutics. Our capabilities include viral vector process and analytical development, small to large scale GMP manufacturing, fill/finish and QC/QA release testing through Phase III with plans to launch commercial services in 2022. Andelyn also offers plasmid manufacturing services, formulation development services and regulatory support.

Spotlight

This technical note demonstrates the comprehensive
characterization and confident identification of glucuronide
metabolites from hepatocyte incubations of midazolam. An
orthogonal fragmentation mechanism was applied to generate diagnostic fragment ions for confident identification of glucuronide metabolites

Spotlight

This technical note demonstrates the comprehensive
characterization and confident identification of glucuronide
metabolites from hepatocyte incubations of midazolam. An
orthogonal fragmentation mechanism was applied to generate diagnostic fragment ions for confident identification of glucuronide metabolites

Related News

INDUSTRIAL IMPACT, DIAGNOSTICS

Vaxxinity Announces First Subjects Dosed in Phase 1 Clinical Trial of VXX-401, Investigational Anti-PCSK9 Vaccine to Treat Hypercholesterolemia

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.

Read More

MEDTECH, INDUSTRIAL IMPACT

ConcertAI Releases CTO 2.0, a Next Generation AI SaaS Solution for Oncology Clinical Trial Design and Optimization

Prnewswire | April 06, 2023

ConcertAI, LLC a leader in AI Software-as-a-Service (SaaS) technology and Real-world Evidence solutions for life sciences and healthcare, announced the release of its next generation of Clinical Trial Optimization (CTO 2.0). "Trial sponsors have a range of new imperatives they're trying to meet: assuring that the trial population is reflective of the ultimate population that would have access to the drug; to set and meet diversity goals for those ethnic, racial and economic groups that are uniquely, negatively impacted by a disease; and meeting ever higher productivity imperatives for identifying and activating sites faster and working with those sites most likely to accrue to the pre-activation feasibilities," said Jeff Elton, Ph.D. CEO of ConcertAI. "Meeting these goals requires working with sites and investigators not historically preferred, designing trials for a minimum of burden on research sites and patients and, perhaps most importantly, moving more studies into community-based research settings." CTO 2.0 now includes a range of features: (1) clinical depth and tools, such as line of therapy assessments, Kaplan-Meier survival curves, and cox-proportional hazard assessments that guide alternative designs with the largest underlying research dataset for oncology; (2) data integrating electronic medical-derived, medical claims, and social determinants of health; (3) site- and investigator-level information on current and previous clinical trials derived from public and private sources; and (4) operational trial metrics and site profile information to highlight performance and capabilities. The solution's foundation is a study digitization layer that can handle any depth of inclusion and exclusion (I/E) criteria. Protocols can be assessed relative to standard-of-care in different settings and in different time periods, all the way down to the individual event or clinical activity level. There are APIs for integrating past study protocols from any legacy systems of records. Optimizer and recommendations engines can provide study-level optimizations and semi-automate site selection. Additional APIs can integrate past site survey data and investigator databases as a means of simplifying and unifying what are often non-integrated solutions within the clinical analytics and clinical operations organization. The SaaS technology will support researchers and clinicians in selecting the most appropriate sites for clinical trials, balancing potential patients available and previous trial performances at those sites to give the highest likelihood of success for a clinical trial. In assurance that trials can be conducted across settings and utilize the latest digital trial solutions that support direct EMR to EDC, CTO 2.0 leverages the latest clinical informatics integrations and data standards to ensure research can be conducted at scale and meeting, or exceeding, enhanced standards of care. "In the past, clinicians used previously published protocols and recently approved studies in the same disease to inform trial designs for new studies," said Ronan Brown, ConcertAI's Chief Operating Officer. "Now that is changing. We'll now see greater reliance on large scale, multi-sourced, clinical datasets that have low latency, reflecting the latest treatments, supporting new designs, lower site burden, greater, diversity, and expanded views of relevant sites. It's a significant step forward in clinical trial planning and operations." About ConcertAI ConcertAI is a leader in Real-World Evidence (RWE) and AI technology solutions for life sciences and healthcare. Our mission is to accelerate insights and outcomes for patients through leading real-world data, AI technologies, and scientific expertise in partnership with the leading biomedical innovators, healthcare providers, and medical societies.

Read More

INDUSTRY OUTLOOK

Monte Rosa Therapeutics Advances Second Development Candidate, MRT-6160, a Novel, Highly Selective Molecular Glue Degrader Targeting VAV1

Globenewswire | May 29, 2023

Monte Rosa Therapeutics, Inc. a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, announced its second development candidate, MRT-6160, a novel, potent, and selective molecular glue degrader of VAV1. The Company plans to file an Investigational New Drug (IND) application for MRT-6160 in the first half of 2024 and to develop the molecule as a potential treatment for autoimmune diseases. “MRT-6160 is a potent, orally bioavailable MGD designed to degrade VAV1, an important protein involved in the signaling pathways of T and B cells. Our in vitro studies have shown that MRT-6160 selectively degrades VAV1 without detectable effects on other proteins. By targeting VAV1, MRT-6160 attenuates multiple aspects of T- and B-cell function and inhibits disease progression in established in vivo models of autoimmunity,” said Owen Wallace, Ph.D., Chief Scientific Officer of Monte Rosa. “The underlying biology and our preclinical data both demonstrate that MRT-6160 acts as an immune modulator, which has the potential to avoid the broad immune suppression seen with other approaches. We look forward to progressing our clinical plan developed with the goal of providing early insights into safety, PK and PD, and proof of concept regarding differentiated effects on key immunomodulatory signaling pathways.” “Our goal centers on pioneering therapeutically meaningful new drugs for patients with serious diseases. By addressing VAV1, a validated but previously undruggable target, we believe we've created a potentially groundbreaking therapy for patients suffering from a range of serious autoimmune conditions, particularly those involving both T- and B cell-mediated autoimmunity,” said Markus Warmuth, M.D., CEO of Monte Rosa. “MRT-6160 is expected to be our second MGD to enter clinical trials, showcasing the continued productivity of our QuEEN™ platform. We anticipate significant progress and milestones in our portfolio in the upcoming year, including initial clinical data from our GSPT1 MGD, MRT-2359, in the second half of this year and filing of an IND application for MRT-6160 in the first half of next year.” About Monte Rosa Monte Rosa Therapeutics is a biotechnology company developing novel molecular glue degrader (MGD) medicines for patients with serious diseases such as oncology, autoimmune and inflammatory diseases. MGDs are small molecule protein degraders that employ the body’s natural mechanisms to selectively eliminate therapeutically relevant proteins. The Company’s QuEEN™ (Quantitative and Engineered Elimination of Neosubstrates) platform enables it to rapidly identify protein targets and design highly selective degraders by combining diverse libraries of proprietary MGDs with in-house proteomics, structural biology, AI/machine learning, and computational chemistry capabilities.

Read More