Pharmaceutical Technology Editors

The US Pharmacopeia (USP), a global independent scientific organization, has identified the need for guidelines and analytical procedures regarding quality assessments for messenger (mRNA) vaccines and technology. USP is seeking scientific input on new draft guidelines, 

Analytical Procedures for mRNA Vaccine Quality

mRNA-based therapies and other innovative technologies must be of good quality, safe, and effective in order to gain trust and confidence to the public. The success in the application of mRNA technology is still relatively new, and guidelines and industry standards are still evolving. Developing common standards for the assessment of mRNA quality would allow developers, manufacturers, regulatory agencies, and national control laboratories worldwide to be able to accelerate the development of safe and effective mRNA-based products.

“We all witnessed the first successful use of mRNA technology in the global fight against COVID-19, and we are very optimistic about the promise of mRNA technology applied to therapies and vaccines for a variety of conditions. The quality of mRNA products must be a top priority in order to facilitate market entry and build patient and provider trust,” said Jaap Venema, PhD, USP’s executive vice president and chief science officer, in the press release. “USP convenes industry and scientific experts in emerging technology areas to identify best practices for understanding quality as that technology continues to grow. Without that common understanding, manufacturers must develop their own in-house methods, taking attention and resources away from the successful application of that technology.”

Articles

USP is developing mRNA quality guidelines to help companies and regulators bring innovative medicines to market faster.

USP Developing mRNA Quality Guidelines to Help Bring Innovative Medicines to Market Faster

Remix Therapeutics, a biotechnology company focusing on reprogramming RNA processing, announced a strategic collaboration with Janssen Pharmaceutica NV on Feb. 17, 2022. The strategic partnership will see Janssen use Remix’s REMaster drug discovery platform for the discovery and development of small molecule therapeutics that modulate RNA processing.

Per the terms of the agreement, Janssen will have exclusive rights to three specific targets with applications in oncology and immunology. In exchange, Remix will receive an initial payment of $45 million, but could receive over $1 billion in payments should it meet regulatory approvals and various other conditions.

“We look forward to collaborating with this innovative team,” said Peter Smith, co-founder and CEO of Remix Therapeutics, in a company press release. “Janssen is an ideal strategic partner for our REMaster platform. This collaboration further validates the therapeutic potential of our proprietary REMaster drug discovery platform and provides additional resources to translate our cutting-edge science into new medicines.”

Janssen will pay upwards of $1 billion for exclusive rights to use Remix’s RNA-based drug discovery platform on three specific targets.

Remix Therapeutics Collaborates with Janssen to Advance Small Molecule Therapeutics

Eli Lilly and Company (Lilly) announced the launch of the Lilly Institute for Genetic Medicine and an investment of approximately $700 million to establish a state-of-the-art facility at a new site in Boston on Feb. 22, 2022. Lilly intends to use the Institute to develop genetic medicines for diseases that have been challenging or impossible to treat with traditional medicines.

The Institute will be located in a 12-story, 334,000 ft² building in Boston’s Seaport district. It will work with Prevail Therapeutics, a gene therapy company based in New York City acquired by Lilly in 2020, to leverage promising RNA and DNA-based technologies.

"Establishing the Lilly Institute for Genetic Medicine will allow us to pair cutting-edge technologies with our deep biological expertise in several areas including neuroscience and diabetes," said Andrew C. Adams, vice president of genetic medicine at Lilly and co-director of the Institute, in a company press release. "Lilly will focus on medicines acting at the nucleic acid level to advance an entirely new class that target the root cause of diseases, an approach that is fundamentally different than medicines available today."

According to the press release, genetic medicines already account for more than 20% of Lilly’s diabetes, immunology, and central nervous system research portfolio. Within five years, Lilly expects the number of researchers working at the Boston facility to grow from 120 to 250.

"The Institute will enhance our efforts on neurodegenerative diseases and integrate Lilly's genetic medicine research and platforms with the goal of advancing promising and potentially life-altering new medicines from the lab to clinical studies and ultimately to patients," said Franz Hefti, CEO of Prevail Therapeutics at Lilly and co-director of the Institute, in the press release. "We look forward to working with hundreds of scientists and researchers who share a common goal—to create and develop innovative genetic medicines that make life better for people around the world."

Eli Lilly and Company are investing $700 million into a Boston-based facility for the newly announced Institute for Genetic Medicine.

Lilly Launches Institute for Genetic Medicine and Invests in Boston Seaport Site

Taconic Biosciences, a provider of drug discovery animal model solutions, announced its Cage+ product on Feb. 21, 2022. Cage+ is a holistic colony management model that is intended to offer comprehensive coverage for contract breeding services.

According to a company press release, project planning and management burden is often placed on investigators, who likely do not have the time or expertise to design and manage scaled production of complex animal models. Cage+ is meant to provide a solution to this, as it applies a holistic approach that combines standard animal breeding and husbandry elements with breeding design expertise, project-specific methodology, budget monitoring, and proactive project management and communication.

Uses expert-led embryology methods to expand breeding production

Delivers internationally harmonized animal health standards

Includes the eTACONIC web-based project management tool, which provides users 24/7 access to colony information.

“Researchers should demand better from contract breeding service providers, and this view is the driving factor behind our Cage+ stewardship-based approach to colony management solutions,” said John Couse, vice-president, scientific services, Taconic Biosciences, in the press release. “With Cage+, researchers will view Taconic as an extension of their team, leveraging the collaboration of scientists and experts. Our approach allows investigators to focus their time, energy, and resources on research while trusting their custom model animal development and production to Taconic.”

Taconic Biosciences Cage+ colony management service aims to offer comprehensive coverage for contract breeding services.

Taconic Biosciences Launches Cage+ Contract Breeding Service

MilliporeSigma announced a collaboration between its life science business sector and Waters with the aim of building and expanding an extractables and leachables (E&L) reference library to include ion mobility measurements.

According to a Feb. 15, 2022, press release, the library will enable analytical labs to use Waters’ ion mobility-enabled liquid chromatography mass spectrometry (LC–MS) instruments to identify potential extractables and leachable compounds. The identity and quantity of these compounds will then be confirmed through MilliporeSigma’s Supelco reference materials.

Per the terms of the agreement, Waters will use high-quality analytical standards and reference materials from MilliporeSigma to build and expand an E&L library of collision cross-section (CCS) values for Waters’ LC–MS instruments. The library will be available for download from the Waters Marketplace (login required) and cross-linked to the MilliporeSigma business sector online product catalog to provide users access to reference materials to confirm their results.

“Accurate screening for extractables and leachables is imperative to ensuring consumer safety, especially in pharmaceuticals, food packaging, or medical devices,” said Heike Petri, head of Advanced Analytical and Industrial & Testing, Life Science business sector, Merck KGaA, Darmstadt, Germany, in the press release. “This collaboration will provide manufacturers with unrivalled confidence in their results, help improve workflow efficiency for labs, and ultimately contribute to consumer safety.”

MilliporeSigma and Waters will work together to build an extractables and leachables (E&L) reference library.

MilliporeSigma Announces E&L Reference Library Collaboration with Waters

Valneva, a France-based specialty vaccine company, announced on Feb. 21, 2022 that its subsidiary, Valneva Scotland, has received a research and development (R&D) funding award of up to £20 million (US$27 million) from Scottish Enterprise, Scotland’s national economic development agency.

The award will be comprised of two grants that build on Scottish Enterprise’s long-standing engagement with Valneva. The grants will benefit Valneva’s manufacturing site in Livingston, Scotland, and are expected to be received over the next three years, starting in March 2022.

The first grant of up to £12.5 million (US$17 million) will support R&D related to the manufacture of VLA2001, Valneva’s inactivated, whole virus COVID-19 vaccine candidate. The second grant of up to £7.5 million (US$10 million) will support R&D connected to Valneva’s manufacturing processes for other vaccines.

Valneva’s R&D portfolio includes VLA1553, a single-shot vaccine candidate against the mosquito-borne viral infection chikungunya, which will also be manufactured at the Livingston site. The company reported positive topline Phase III results in 2021 for both VLA2001 and VLA1553.

“This investment bolsters Valneva’s longstanding relationship with Scottish Enterprise as well as our position at the forefront of life sciences and vaccine development in Scotland. From the only inactivated, whole virus COVID-19 vaccine candidate in clinical development in Europe to the most clinically advanced vaccine candidate against chikungunya in the world, Scottish Enterprise’s investment will support progress across Valneva’s research and development portfolio—as well as jobs and growth in Scotland. We are grateful to Scottish Enterprise, and to the Scottish Government, for their continuing support,” said Thomas Lingelbach, CEO of Valneva, in a company press release.

“Valneva’s decision to develop and manufacture its COVID-19 vaccine here in Scotland is extremely welcome. It is a huge vote of confidence in our life sciences sector and its highly skilled workforce, with one of the largest and most advanced vaccine manufacturing sites in the world now firmly anchored in Scotland and set to export its life-saving vaccines across the world. We look forward to supporting Valneva’s continued growth in Scotland, and to help it realize its international growth ambitions,” said Adrian Gillespie, chief executive of Scottish Enterprise, in the press release.

Scottish Enterprise has awarded £20 million (US$27 million) to Valneva Scotland to advance vaccine development.