Having a clear clinical strategy early on can shave time off overall development projects.

Early Strategizing Tightens Development Timelines

With recent approvals of bispecific antibodies, these complex molecules are fast moving out of the research box and into clinical pipelines.

Bispecific Antibodies are Moving from Research to Clinical Development

Extensive work has greatly expanded the possibilities for fetal bovine serum sourcing.

Considerations for US Fetal Bovine Serum Sourcing

Thanks to technological advances, process chromatography challenges are surmountable, but increasingly complex therapies in the pipeline will demand more efficient processes in the future.

Advancing Process Chromatography in DSP

The complex nature of biologics creates challenges for GMPs in sterile manufacturing, and the EU’s Annex 1 is making an impact.

GMPs for Sterile Manufacturing of Biologics

Precompetitive consortiums seek solutions to industry-wide challenges.

Coming Together to Enable Cell and Gene Therapy Manufacturing

When considering whether to outsource work to a CRO, there are a number of factors to assess, including the type of work that may be outsourced, regulatory considerations, and needs for the study.

When Is It Appropriate to Outsource Bioanalysis Work to a CRO?

Aptamer Group and Flip Gene Therapeutics have negotiated a deal to use Aptamer Group’s Optimer technology to support Flip Gene Therapeutics’ gene therapy platform. Under the agreement, Aptamer Group will develop Optimer binders to several small-molecule targets, which will be incorporated into Flip Gene Therapeutics’ gene therapy platform with the goal of developing pharmacologically inducible gene therapeutics that are controllable with a gene switch.

The agreement includes initial upfront payments for Optimer development, with the potential for further licensing payments for the developed Optimer binders, and development and commercial milestone payments upon clinical and commercial success.

Optimer binders are oligonucleotide-based affinity ligands. They offer a solution to support novel gene therapy development through their potential for increased success in discovery over a broader target range and the ability to tune their half-life.

“I am delighted to have established this agreement to develop Optimer binders to enable inducible treatments in this exciting field of gene therapy,” said Dr. Arron Tolley, CEO of Aptamer Group, in a press release. “We will be working closely with our partner Flip Gene Therapeutics on developing and assessing the Optimer binders to function as part of their gene therapy platform. The interest in pursuing Optimer binders as part of their therapeutic modality offers a strong endorsement of the technology.”

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Aptamer Group has signed a deal with Flip Gene Therapeutics to support the development of inducible gene therapies.

Aptamer Group Signs Deal with Flip Gene Therapeutics

SCHOTT has announced plans to triple its ready-to-use vial production capacity at its site in Lebanon, PA, to meet increasing demand for storing injectable drugs. The company currently provides a comprehensive product portfolio for ready-to-use vials with more than 750 product combinations.

Ready-to-use vials are particularly suitable for drugs, such as biologics and vaccines. The vials are pre-washed and sterilized and packaged in a standardized tub to simplify the filling process for pharmaceutical companies. The solution enables pharma companies to efficiently perform pre-clinical and clinical trials as well as commercial filling.

“With our investment, we are underlining our commitment to the global pharma industry and particularly the U.S. market. Additionally, we are enhancing our regional manufacturing footprint in Lebanon, Pennsylvania, where we are proudly celebrating our site’s 30th anniversary,” said Andreas Reisse, executive vice president of SCHOTT Pharma, in a press release.

“We are increasing our production capacity intending to meet the growing need for this product category and contribute to bringing the latest life-saving treatments to patients faster,” said Hakan Gerdan, who was appointed as SCHOTT’s new Plant Manager in Lebanon, PA at the start of 2022, in the release.

SCHOTT is planning to triple its ready-to-use vial production capacity in the United States to meet increasing demand for storing injectable drugs.

SCHOTT to Triple Vial Production Capacity in the US

Boehringer Ingelheim announced on June 14, 2022 that it has signed an option to acquire Trutino Biosciences, a San Diego-based biotech company.

Trutino Biosciences is a pre-clinical stage biotech company that focuses on the discovery and development of cytokine therapies to treat immuno-oncology and autoimmune diseases. The acquisition will advance Boehringer Ingelheim’s effort to mobilize a patient’s immune system to fight cancer through cancer cell-directed and immune cell-targeting compounds.

Under the agreement, Boehringer Ingelheim reserves the rights to purchase all shares of Trutino Biosciences once specified program milestones have been achieved within a given timeframe. Trutino will continue to operate as an independent company until that time.

“Boehringer Ingelheim is excited to extend our partnership with Dr. Kim and his outstanding team at Trutino Biosciences. Our existing collaboration has made rapid and impressive progress in a short time frame, and we expect this field to have potential combination benefits with our existing immune-targeting assets,” said Clive R. Wood, PhD, corporate senior vice president and global head of discovery research, Boehringer Ingelheim, in a press release. “The selective activation of specific cytokines localized in the tumor microenvironment holds enormous promise and has the potential to be a key element in fully harnessing the power of the immune system to fight cancer.”

Boehringer Ingelheim has signed an option to acquire Trutino Biosciences.

Boehringer Ingelheim to Acquire Trutino Biosciences

 Washington Editor, jillwechsler7@gmail.com.

A broad bill carrying multiple proposals for revising and expanding FDA programs and policies, in addition to reauthorizing user fees critical for funding FDA operations, was endorsed by the Senate Health, Education, Labor and Pensions (HELP) Committee this week. The legislators’ approval of the FDA Safety and Landmark Amendments (FDASLA) by a 13–9 committee vote sets the stage for full Senate reauthorization of industry fees to support oversight and development of prescription drugs, generic drugs, biosimilars, and medical devices.

The measure adopts a host of new programs and requirements that stand to make significant changes in how FDA regulates a range of medical and food products. However, many of the specific additions and revisions in the Senate bill differ from legislation already passed by the 

(HR 7667). The situation requires the legislators to reconcile the two measures in order to enact final legislation by the current target of August 1, 2022.

The extensive Senate HELP bill builds on a 

backed by HELP committee chair Patty Murray (D-WA) and ranking Republican Richard Burr (R-NC). An important provision backed by Murray clarifies the pathway for importation of drugs from Canada, expanding the range of eligible products. Murray also endorsed new proposals for strengthening FDA oversight of cosmetics and dietary supplements, which establish a broad range of new requirements and regulatory processes for these products. In addition, the Verifying Accurate Leading-edge IVCT Development (VALID) Act backed by Burr updates FDA policies for authorizing 

 diagnostics, with an eye to enhancing the agency’s ability to evaluate effective lab tests in public health crises.

Moreover, the legislators moved to address the current infant formula crisis by establishing an Office of Critical Foods in FDA’s Center for Food Safety and Applied Nutrition (CFSAN). It provides a premarket review process for manufacturers of infant formula and requires FDA notification of production disruptions and recalls and switches to appropriate substitutions. A number of FDA reports to Congress on the program are included, along with revised procedures for FDA’s mailroom operations to prevent the loss of whistleblower reports and other important documents related to these and other regulated products.

Additional sections of the bill offer a range of enhancements for testing and marketing drugs and medical products. The development of treatments for rare diseases gains support from a new pilot program and other provisions. Competition from generic drugs may be enhanced by authorization of labels with certain differences from brands, while import is permitted for certain medical devices made overseas. FDA’s accelerated approval pathway is updated to ensure its support for innovative cardiology and neurology therapies, as well as cancer treatments. Added provisions aim to discourage the use of animal tests in developing drugs, cosmetics, and other products. And FDA is required to move forward with policies to authorize over-the-counter hearing aids.

Important to FDA officials are provisions designed to expedite and simplify agency hiring practices and its ability to retain talented scientists and medical personnel. At the same time, the legislators extend oversight for FDA’s IT modernization program, requiring reports and clear goals and priorities to ensure that the added funds requested for this agency-wide initiative are effective and well-spent.

The wide scope of the legislation is not surprising, given the long history of policy makers adding multiple pet initiatives to this must-pass measure. As fees from regulated companies have become a main source of support for FDA oversight of the testing and development of critical medical products, the challenge remains to continue this financial support for the agency without enacting measures that will overload or undermine FDA and industry operations.

Measure tackles infant formula, cosmetics, diagnostics plus drug development and access. 

FDA User Fee Legislation Advances in Senate

Late-stage clinical biopharmaceutical company, Humanigen, and global contract development and manufacturing organization (CDMO), PCI Pharma Services, have entered into an agreement for the commercialization of lenzilumab, it was announced on June 16, 2022.

Lenzilumab is a first-in-class monoclonal antibody that has been shown to neutralize granulocyte-macrophage colony-stimulating factor (GM-CSF) associated with COVID-19 and other indications. Results from a Phase III study (LIVE-AIR) have suggested that when using lenzilumab as an early intervention it has the potential to prevent the consequences of a full-blown cytokine storm in hospitalized patients with COVID-19.

“We continue our commercial preparation in the UK [United Kingdom] and in parallel are working closely with the Medicines and Healthcare products Regulatory Agency (MHRA) to address regulatory requirements for a potential Conditional Marketing Authorization. With its global reach, PCI will provide a critical function in the supply chain, by directly purchasing lenzilumab for further distribution in the UK and facilitating this key process for Humanigen,”saidEdward Jordan, chief commercial officer, Humanigen.“It is anticipated that we will complete our response to MHRA soon after the top-line results from the ACTIV-5/BET-B clinical trial with lenzilumab are received.”

Humanigen and PCI Pharma Servicesare partnering up for the commercialization of lenzilumab.

Humanigen, PCI Pharma Services Enter Agreement for Lenzilumab Commercialization

The United Kingdom’s regulator, the Medicines and Healthcare products Regulatory Agency (MHRA), has been accepted as a full member of three international work-sharing partnerships aimed at making sure medicines and medical devices are regulated safely and efficiently across the world.

Two of the bodies that MHRA will join are the International Medical Device Regulatory Forum (IMDRF) and the International Council for Harmonisation (ICH), which are concerned with the harmonization of medicines and medical devices regulations globally. The third body is the Medical Devices Innovation Consortium (MDIC), which is an American public-private partnership aimed at progressing innovation and getting transformational medical technology to people sooner.

“We are delighted to join these three international organizations to collaborate on regulatory alignment that will help deliver safer, innovative, and more cost-effective medicines and medical devices to the people who need them sooner,” said Glenn Wells, chief international and partnerships officer at the MHRA, in a June 16, 2022 press release.

“We are currently building a world-leading regime for regulating medicines and medical devices in the UK that prioritises patient safety while fostering innovation, and we look forward to sharing expertise with partner organisations for the benefit of patients not just in the UK but worldwide.”

MHRA has been accepted as a full member of three international work-sharing partnerships aimed at making sure medicines and medical devices are regulated safely and efficiently across the world.

MHRA to Play Greater Role in International Medicines Regulation

Science and technology company, Merck, has entered into a collaboration with Agilent Technologies with the aim of filling the industry gap in process analytical technologies (PAT) for downstream processing (DSP), it was revealed in a June 14, 2022 press release.

Under the terms of the partnership, the companies will combine Merck’s bioprocess portfolio with Agilent Technologies’ advanced analytical products to extend real-time monitoring and automated process control of critical process parameters (CPPs) and critical quality attributes (CQAs) in DSP. PAT is key to enabling real-time release and Bioprocessing 4.0.

“The biopharmaceutical industry is on a rolling transformation to evolve and digitalize the next generation of bioprocessing to increase accessibility and lower the cost of biologics,” said Darren Verlenden, head of BioProcessing, Life Science business sector at Merck, in the press release. “By combining our advanced bioprocess portfolio with Agilent’s leading analytical solutions, we will be able to offer integrated capabilities for enhanced downstream process monitoring and control, bringing us one step closer to making the facility of the future a reality.”

“Integrating analytical capabilities and intelligence into bioprocessing enables the biopharma industry to accelerate biological drug development and manufacturing while supporting continuous manufacturing and real-time release,” added Stefan Schuette, vice president and general manager of Agilent´s Liquid Phase Separations Division. “This collaboration focuses on integrating Agilent’s new online liquid chromatography (LC) solution with Merck’s bioprocessing platforms and will support customers on their journey to develop and produce biological drugs faster, with greater confidence, and at lower cost.”

Merck has entered into a collaboration with Agilent Technologies with the aim of filling the industry gap in PAT for downstream processing.

Merck Collaborates with Agilent Technologies on PAT for Downstream Processing

According to a June 15, 2022 press release, a new polymer surface material has been invented by researchers at Chalmers University of Technology (Gothenburg, Sweden) that uses electrical signals to both capture and release biomolecules.

The new material may have an impact in the way that biomedicines are developed and could provide opportunities in the development of remote-controlled medication and electronic pills. The results of the research were published in a scientific journal, 

“Our polymer surfaces offer a new way of separating proteins by using electrical signals to control how they are bound to and released from a surface, while not affecting the structure of the protein,” said Gustav Ferrand-Drake del Castillo, doctor in Chemistry, Chalmers, CEO of Nyctea Technologies, and lead author of the study, in the press release.

The initial research behind the material was performed by Ferrand-Drake del Castillo while studying under the stewardship of Chalmers professor Andreas Dahlin within the university’s Division of Applied Surface Chemistry. The research project involved polymer surfaces that can change their state when the pH of the surrounding solution is adjusted—so, changing between neutral and charged.

“Shortly afterwards we discovered that we could use the electrical signals to control the binding and release of proteins and biomolecules, and that the electrode material works in biological solutions such as serum and centrifuged blood. We believe and hope that our discoveries may be of great benefit in the development of new medicines,” explained Dahlin, in the press release.

Additionally, the material was found to also work within biological fluids with the ability to counteract changes in pH values. Through this specific property of the polymer surface material, it may be possible for the researchers to create a new technique for implants and electronic pills, whereby the medicine is released within the body through electronic activation.

“You can imagine a doctor, or a computer program, measuring the need for a new dose of medicine in a patient, and a remote-controlled signal activating the release of the drug from the implant located in the very tissue or organ where it’s needed,” added Ferrand-Drake del Castillo. “Being able to control the release and uptake of proteins in the body with minimal surgical interventions and without needle injections is, we believe, a unique and useful property. The development of electronic implants is only one of several conceivable applications that are many years into the future. Research that helps us to link electronics with biology at a molecular level is an important piece of the puzzle in such a direction.”

A new polymer surface material has been invented by researchers in Sweden that uses electrical signals to both capture and release biomolecules.

Researchers Invent New Material that Can Capture and Release Biomolecules

Viral vector contract development and manufacturing organization (CDMO), Exothera, has announced its collaboration with clinical-stage genetic medicine company, LogicBioTherapeutics, and provider of upstream process solutions, Polyplus-transfection, for the development of a scalable adeno-associated virus (AAV) manufacturing platform.

The project, which was revealed in a June 15, 2022 press release, will be led by Exothera at its facility located in Jumet, Belgium. LogicBio will deliver its proprietary mAAVRx plasmid technology and AAV expertise, and Polyplus will provide access to its FectoVIR-AAV transfection reagent and its process development expertise for large-scale transient processing.

“Exothera’s team is proud to work with the experienced teams at biotech companies like LogicBio and Polyplus,” said Hanna Lesch, chief technology officer at Exothera, in the press release. “The success of AAV-based therapeutics requires a change in the manufacturing paradigm and through this collaboration, we aim to enable a new approach to consistently scale AAV manufacturing.”

“We believe our proprietary mAAVRx™ plasmid technology could dramatically help to improve manufacturing yields and consequently reduce AAV manufacturing costs,” added Matthias Hebben, global vice president, Technology Development at LogicBio, in the press release. “We are hopeful that the work done under this collaboration will be an important step forward in making gene therapy products accessible for a greater number of patients.”

“We are delighted to scale up AAV Viral vector Manufacturing processes to larger bioreactor volumes with innovators in the industry like Exothera and LogicBio,” said Roel Gordijn, chief commercial Officer at Polyplus, in the press release. “The collaboration will use a combination of state-of-the-art manufacturing technologies, innovative plasmid technologies and scalable transfection reagents which will make gene-therapy viral vector therapies affordable.”

Exothera, LogicBioTherapeutics, and Polyplus-transfection, are collaborating on the development of a scalable AAV manufacturing platform.

Exothera, LogicBioTherapeutics, and Polyplus Collaborate on Industrial-Scale AAV Manufacturing 

FDA published draft guidance on June 15, 2022 that describes the Center for Biologics Evaluation and Research’s (CBER) standards recognition program for regenerative medicine therapies (SRP-RMT) to identify Voluntary Consensus Standards (VCS) for the development of regenerative medicine therapy (RMT) products. CBER states that the use of VCS can assist stakeholders in meeting regulatory requirements. “By leveraging stakeholders’ efforts to develop standards, FDA can eliminate costs to the Federal government associated with the development of government-unique standards and promote international harmonization of standards that are acceptable to FDA,” FDA states in the guidance. 

The document describes the procedures for evaluating VCS and documenting the use of a standard recognized by CBER. It also answers industry questions about VCS and SRP-RMT.

The new draft guidance describes a standards recognition program for regenerative medicine therapies.

FDA Publishes Guidance on Regenerative Medicine Therapies

FDA announced on June 15, 2022 that it has published the International Council for Harmonisation’s (ICH) 

draft guidance document. The draft version details the general quality risk management (QRM) process including risk assessment, risk control, risk communication, and risk review. It also discusses methodology for risk management and risk-based decision making.

The integration of QRM into the industry and regulatory operations is also discussed. The document includes two annexes. Annex I provides information on QRM methods and tools such as failure mode effects analysis, fault tree analysis, hazard analysis and critical control points, and more. Annex II breaks down QRM as parts of integrated quality management.

“In the pharmaceutical sector, the principles and framework of ICH Q9, coupled with the official ICH training material that supports this guideline, are instrumental in enhancing the application of effective quality risk management by industry and regulators. The importance of quality systems has been recognized in the pharmaceutical industry, and it is evident that quality risk management is a valuable component of an effective quality system,” the agency stated on its website.

The draft ICH Q9(R1) document details the importance of quality risk management principles.

FDA Publishes Draft Quality Risk Management Guidance

Yourway, an integrated courier and clinical packaging company, announced the construction of a new flagship European depot in Dublin, Ireland on June 8, 2022. The new good manufacturing practice facility will support Yourway’s clinical trials offering, including courier services, primary and secondary packaging, and temperature-controlled storage.

According to a company press release, Yourway chose the Dublin Airport Logistics Park site because it’s convenient for major pharma and biopharma companies in Ireland. The facility will also offer easy access to a significant domestic clinical trials market. It is scheduled to be audit ready by Q4 2022.

“With Ireland’s strength in biopharma, Yourway has operated there for over 15 years, which contributed to our decision to build our flagship depot in Dublin,” said Gulam Jaffer, founder and president, Yourway, in the press release. “We’re anticipating notable market growth in Europe, and Yourway will be perfectly placed to support it.”

The site will be supported by the company’s other European depots in London, UK; Kyiv, Ukraine; Minsk, Belarus; and Moscow, Russia. The company’s global depot network exists across these locations as well as sites in 15 other countries across the world.

Yourway intends to construct a new manufacturing facility in the Dublin Airport Logistics Park.