The breakthrough approach is commonly used, but the devil is in the details.

Determining and Optimizing Dynamic Binding Capacity

In this article, a simple chromatographic model is proposed that is capable of predicting the impact of pH and ionic strength on HIC chromatograms.

Purification of Protein by HIC: Mechanistic Modeling for Improved Understanding and Optimization

The COVID-19 vaccines opened the door to powerful market potential for other nucleic acid-based therapies.

Ramping Up Development of Nucleic Acid Therapies

The innovation of going from stainless-steel bioreactors to fixed-bed bioreactors shows an evolution in upstream optimization.

Exploring the Impact of Fixed-Bed Bioreactors in Upstream Processing

Makers of specialty biologics, gene therapies, and other personalized medicines are working to identify and reduce potential supply chain risks both upstream and downstream.

Reducing Vulnerabilities in Upstream and Downstream Supply Chains

FDA has spurred investment to create and develop 600 therapies.

Kudos and Hurdles in Tackling Rare Diseases

What can we take away from Pfizer's acquisition of Seagen?

 Zauberkugel: Pfizer’s Seagen Acquisition

Grant Playter is the Assistant Editor for 

The Federal Trade Commission (FTC) announced on May 16, 2023 that the agency is filing suit against Amgen in conjunction with Amgen’s recent $27.8 billion bid to acquire Horizon Therapeutics. In an agency press release, FTC cited concerns that the acquisition would enable Amgen to use rebates on its existing blockbuster drugs to pressure insurance companies and pharmacy benefit managers into favoring Horizon’s two-monopoly biologics products. The biologics products in question are Tepezza (teprotumumab-trbw), a treatment for thyroid eye disease, and Krystexxa (pegloticase), a treatment for chronic refractory gout.

“Rampant consolidation in the pharmaceutical industry has given powerful companies a pass to exorbitantly hike prescription drug prices, deny patients access to more affordable generics, and hamstring innovation in life-saving markets,” said Holly Vedova, Bureau of Competition director , FTC, in the release. “Today’s action—the FTC’s first challenge to a pharmaceutical merger in recent memory—sends a clear signal to the market: the FTC won't hesitate to challenge mergers that enable pharmaceutical conglomerates to entrench their monopolies at the expense of consumers and fair competition.”

In response to the suit, Amgen released a public statement denying its intentions to bundle coverages.

“The medicines offered by Amgen and Horizon generally treat different diseases and patient populations, and there are no overlaps of competitive concern,” reads the statement. “The FTC's claim that Amgen might ‘bundle’ these medicines (offer a multi-product discount) at some point in the future is entirely speculative and does not reflect the real-world competitive dynamics behind providing rare-disease medicines to patients.”

Amgen’s statement also questioned whether any acquisition could be denied under a “bundling theory,” claiming that there was no such precedent.The company also announced its intent to work with the courts and complete the acquisition by mid-December 2023.

Articles

In a brief, the Federal Trade Commission cited concerns about the acquisition resulting in an increased monopoly on Amgen’s newly acquired thyroid eye disease and chronic refractory gout treatments.

FTC Sues to Block Amgen’s $27.8 Billion Acquisition of Horizon Therapeutics

The National Science Foundation (NSF) awarded The Puerto Rico Science, Technology & Research Trust (Trust) $1 million as part of NSF’s Regional Innovation Engines program. The Trust is one of 44 unique teams to receive the first-ever NSF Engines Development Awards, which aims to help partners collaborate on economic, societal, and technological opportunities for significant growth in their regions. Bright Path Laboratories, a US-based advanced drug development and manufacturing company, will participate as a collaborator in this award.

According to a company press release, the “Advancing Biopharmaceutical Technologies and Manufacturing Practices (P.R.)” award aims to lay the groundwork for establishing a NSF Regional Innovation Engine in Puerto Rico. This includes developing its structure and scope, as well as establishing partnerships to advance biopharmaceutical technologies and manufacturing practices. If sufficient progress is made after two years, the Trust will be eligible for an NSF Engines Award of up to $160 million for up to 10 years.

“The necessity to invest in innovative pharmaceutical manufacturing in the United States has never been greater. Bright Path’s … continuous flow advanced manufacturing platform is an integral part of our nation’s supply chain readiness initiative,” said Tony Quinones, co-founder and CEO, Bright Path Laboratories, in the release. “We are very pleased that NSF has chosen the Trust as an awardee and excited to expand and solve the immediate and long-term challenges in our medicinal supply chain through our transformative technology.”

The NSF-sponsored program aims to help partners collaborate to create opportunities for significant economic, societal, and technological opportunities for growth in their regions.

Bright Path Laboratories to Collaborate in NSF Engines Development Program

Feliza Mirasol is the science editor for 

On May 15, 2023, BiOneCure Therapeutics, a US-based clinical-stage biopharmaceutical company focused on developing antibody-drug conjugates (ADCs), announced that it has entered into a strategic collaboration with China- based Nanjing Leads Biolabs, a clinical-stage biopharmaceutical company focused on second-generation immuno-oncology therapeutics, to develop a range of innovative ADCs for treating solid tumors.

The companies intend to leverage their complementary technologies in the collaboration, which they expect will strengthen their R&D and product development capabilities. BiOneCure’s technology includes several proprietary ADC payload-linker platforms. Its first product, BIO-106, is an anti-Trop-2 ADC that is currently in a Phase I clinical study. Leads Biolabs’ portfolio holds more than 20 novel mono- or bispecific antibody drug projects to fulfill unmet medical needs, according to a company press release.

"We are excited to collaborate with Leads Biolabs to develop ADC drugs," said Haifeng Bao, CEO of BiOneCure, in the press release. "Through this collaboration, we aim to accelerate innovation and development in the ADC field and provide better treatment options for patients."

"We believe that BiOneCure has leading ADC technology platforms, and [its] ADC molecule is in the clinical stage," said Xiaoqiang Kang, founder and chairman of Leads Biolabs, in the release. "Leads Biolabs is a clinical-stage antibody new drug company, with multiple antibody technology platforms. We are convinced that this collaboration will greatly expand the innovative R&D capabilities of both parties."

Under the collaboration, BiOneCure Therapeutics and Nanjing Leads Biolabs intend to develop a range of innovative antibody-drug conjugates for treating solid tumors.

BiOneCure Therapeutics and Nanjing Leads Biolabs Partner to Develop ADCs for Solid Tumors

Synthego, a US-based provider of genome engineering solutions, has opened its new good manufacturing practice (GMP) synthesis facility. According to a May 11, 2023 company press release, the 18,000-ft

 facility will support the growing demand for the development of high-quality, clustered regularly interspaced short palindromic repeats (CRISPR)-enabled 

 therapeutics. Services at the new facility will also include investigational new drug-, new drug application-, and biologics license application-enabling clinical studies.

The new facility features multiple multiplexed manufacturing lines, fully automated controls, and an integrated manufacturing execution system. The company will have the capability to produce a range of oligonucleotides, including synthetic single guide RNAs. The facility also includes dedicated process development, analytical testing, and quality control areas, which will ensure that all aspects of the manufacturing process are closely controlled. The facility meets the requirements of both domestic and international regulatory authorities including FDA, the European Medicines Agency, the Medicines and Healthcare products Regulatory Agency, and Health Canada, according to the press release.

“This facility will be essential in supporting the burgeoning field of clinical phase, first-in-human, CRISPR-enabled medicines both for more common genetic diseases but also for rare diseases affecting children for whom other treatments do not exist,” said Adrian Thrasher, a clinician and professor of Pediatric Immunology at University College London Great Ormond Street Institute of Child Health, in the press release. Thrasher’s laboratory is developing new CRISPR-enabled medicines for children with rare genetic diseases.

“As we move into this new generation of cell and gene therapies, it will be crucial that there are strong, reliable manufacturing capabilities that exist for the necessary components of the CRISPR technology—such as the guide RNA—in order to accelerate the path to clinic and treat patients," Thrasher said in the release.

“Synthego pioneered the mass production of guide RNAs for discovery research that has enabled many of the CRISPR-based cell and gene therapy trials happening today. Having been a key leader in providing the technology for researchers and clinicians, it follows that they are now expanding their capacity to support this research as it translates into the clinic,” said Don Kohn, distinguished professor and clinician of Pediatric Hematology and Oncology at University of California, Los Angeles. Kohn’s laboratory pioneered genetic therapies for immunocompromised children.

Synthego has opened its new GMP-compliant RNA manufacturing facility in Redwood City, Calif., for CRISPR-enabled genomics therapeutics.

Synthego Launches New RNA Manufacturing Facility

Data from a Phase I clinical trial of BioNTech and Roche’s personalized cancer vaccine candidate, adjuvant autogene cevumeran, was published in 

 on May 10, 2023. The study evaluated the vaccine’s ability to fight pancreatic ductal adenocarcinoma (PDAC), an aggressive form of pancreatic cancer that the study noted is the third leading cause of death from cancer in the United States and lethal in approximately 88% of patients.

The treatment is an individualized neoantigen vaccine based on uridine messenger RNA (mRNA)–lipoplex nanoparticles, which was developed using mRNA neoantigen vaccines in real time from surgically resected PDAC tumors. When treated with adjuvant autogene cevumeran, in combination with atezolizumab and mFOLFIRINOX, eight of the 16 patients developed T cells that could potentially fight PDAC. Vaccine-expanded T cells were durable, persisting up to two years after injection.

“High-magnitude vaccine-induced T-cell responses, the focus of our immune response analysis that included a new method to track vaccine-expanded clones, correlated with delayed PDAC recurrence,” reads the study. “Despite the limited sample size, these early results warrant larger studies of individualized mRNA neoantigen vaccines in PDAC.”

The data, published in the scientific journal Nature, demonstrated that half of patients developed T cells that could potentially fight pancreatic ductal adenocarcinoma.

BioNTech and Roche Publish Personalized Vaccine Data

TriLink Biotechnologies launched its new cap analog, CleanCap M6, on May 9, 2023, which is expected to help developers and researchers maximize the impact of their messenger RNA (mRNA) therapeutics and vaccines while reducing overall manufacturing costs, according to a company press release. The introduction of CleanCap M6 technology is intended to improve mRNA potency with the highest protein expression of any CleanCap analog to date.

“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,” said Kate Broderick PhD, chief innovation officer at Maravai LifeSciences, in a press release. “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 first CleanCap analog was launched in 2017, and since then, the capping technology has been incorporated in one of the first commercially approved COVID-19 vaccines.

“mRNA is clearly an emerging therapeutic modality where mRNA vaccines and therapeutics are expanding rapidly,” said Drew Burch, EVP of TriLink’s Nucleic Acid Products division, in a press release. “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.”

TriLink Biotechnologies Introduces CleanCap M6 Analog

The pharmaceutical outsourcing market is uniquely poised for significant growth. A recent market report from Growth+ Markets estimated that the market could grow from a 2021 valuation of $125 billion to as much as $227.8 billion by 2030, with an estimated compound annual growth rate of 6.9% (1). That said, simply measuring net growth lacks the context for actionable information if one fails to identify the market segments that are driving said growth.

One effective way of identifying these growth drivers is to look to the major players in the pharma market. By following the moves of pharma giants, upcoming trends can be identified ahead of their full emergence in the broader outsourcing market. This article examines some recent moves made by big name companies to determine what may be lurking over the horizon.

May 2023 Partnerships for Outsourcing eBook

BioPharm International’s Partnerships for Outsourcing eBook

Grant Playter is associate editor with BioPharm International.

When referring to this article, please cite it as Playter, G. Identifying Outsourcing Market Trends. 

Outsourcing collaborations provide insight into the key drivers of market growth.

Identifying Outsourcing Marketing Trends

Editor of Pharmaceutical Technology Europe

Advanced therapeutic medicinal products (ATMPs) are experiencing a boom, with the global pipeline continuing to experience growth and more than 3700 ATMPs currently under development, according to data from the American Society of Gene and Cell Therapy. However, progressing such complex therapies that target niche disease areas through to commercialization produces a unique set of challenges.

Critically, the technical expertise required for the clinical research of ATMPs is something that can be lacking for developers in-house, driving demand for contract research organizations (CRO). In fact, the cell and gene therapy CRO market is forecast to experience continued and consistent growth until 2035.

In this feature, Rhonda Henry, president of Emmes BioPharma; Ching Tian, chief innovation officer, Emmes; and Raja Sharif, CEO Hataali, detail the challenges facing biopharma companies developing ATMPs and the role that CROs play in gaining a market advantage.

BioPharm International’s May 2023 Partnerships for Outsourcing eBook

Felicity Thomas is European/senior editor for 

When referring to this article, please cite it as Thomas, F. CROs Gaining a Competitive Edge. 

BioPharm International’s Partnerships for Outsourcing eBook 

Shifting toward more technological solutions and ensuring a greater understanding of the workforce’s needs will give both CROs and sponsors a market advantage.

CROs Gaining a Competitive Edge

Claudia Berdugo-Davis, PhD, is director of Process Development, Catalent Biologics.

Danielle Wittenwyler, PhD, is manager, Manufacturing Science & Technology, Catalent Biologics.

A biopharmaceutical or biotech innovator’s knowledge and understanding of a manufacturing process is gradually built during the activities that lead up to the product’s commercialization. FDA’s 2011 process validation guidance advises that a lifecycle approach should be taken as the product progresses through clinical trials and approaches potential commercial approval. This process incorporates three stages of process design, process qualification, and continued process verification. With each process designed and built using these three stages, knowledge accumulated will then inform the approach for subsequent product commercialization.

BioPharm International's May 2023 Partnerships for Outsourcing eBook

Streamlining and Standardizing Process Characterization

 is director of Process Development, Catalent Biologics, and 

 is manager, Manufacturing Science & Technology, Catalent Biologics.

When referring to this article, please cite it as Berdugo-Davis, C.; Wittenwyler, D. Streamlining and Standardizing Process Characterization. 

Understanding processes in the development and manufacture of biological drug substances is crucial to successfully navigating the clinical phases towards commercial launch, all within ever‑tightening time constraints, and regulatory frameworks.

Jeong Jin-hyeok is principal scientist in drug product at Samsung Biologics.

The ability of the biopharma industry to meet the worldwide demand for messenger RNA vaccines was facilitated by contract development and manufacturing organizations, which promptly adapted their facilities and capabilities specifically for vaccine production, offering contract fill/finish and cold chain services required to meet demand. Examining the lessons learned from the COVID-19 pandemic, this article provides a perspective on the strategies and tactics taken to deliver a better drug development program and highlights the importance of remembering key lessons as the industry moves toward an era of new drug modalities.

Preparing for a New Wave of mRNA Technologies

When referring to this article, please cite it as Jin-hyeok, J. Preparing for a New Wave of mRNA Technologies. 

To prepare for emerging mRNA technologies, it is necessary to adapt fill/finish and cold chain capabilities.

Susan Haigney is managing editor of BioPharm International, susan.haigney@ubm.com.

The relationship between pharmaceutical companies and the contract organizations that perform development and manufacturing services comes with the necessary process of transferring intellectual information, processes, and product from one organization to another. Complex products such as biologics and messenger RNA (mRNA) vaccines come with additional challenges during the tech transfer process.

Communication is Key in Complex Tech Transfer

To gain more insight on the complexities of transfer of pharmaceutical technology between contractors and sponsors, 

® spoke with Petra Dieterich, scientific leader, and Jeffrey Mocny, scientific leader-Early Development, both with Abzena; Peter Ercoli, chief operating officer, Biologics, at BIOVECTRA; Deepak Thassu, PhD, MBA, senior vice president, R&D and Regulatory Submission, at LGM Pharma; Emma Rhodes, head of New Product Introduction at Sterling Pharma Solutions; Sara Schemel, senior process engineer, Dona York, principal process engineer, and Gauri Patki, senior process engineer, all at Catalent Biologics; Uwe Hanenberg, PhD, head of Product Development at Recipharm; and Rebecca Powell, associate director, Tech Transfer at Arranta Bio, a Recipharm company.


eBook: Partnerships for Outsourcing
May 2023
Pages: 32–37

When referring to this article, please cite it as Haigney, S. Communication is Key in Complex Tech Transfer. 

BioPharm International, Partnerships for Outsourcing

The complexity of the pharmaceutical product brings challenges to the transfer of information and technology between sponsors and contractors.

Life Biosciences, a biotechnology company, and Forge Biologics, a genetic medicines manufacturing organization, announced on May 8, 2023 that they are in a manufacturing partnership to help advance Life Bio’s partial epigenetic reprogramming platform to further address aging-related diseases, according to a press release.

“Life Bio is an emerging leader in the development of novel therapies for aging-related diseases, and we are thrilled to serve as their cGMP manufacturing partner to help advance the manufacturing of AAV for their innovative cellular rejuvenation technology, which has the potential to benefit millions of aging patients worldwide,” said Timothy J. Miller, PhD, CEO, President, and Co-Founder of Forge, in a press release.

Forge will provide adeno-associated virus (AAV) process development, toxicology, cGMP manufacturing, and analytical services to Life Bio, which will occur at Forge’s gene therapy facility in Columbus, Ohio called the Hearth.

“We are delighted to be working with the Forge team, whose expertise in gene therapy manufacturing is unmatched,” said Jerry McLaughlin, Chief Executive Officer of Life Biosciences, in the press release. “We believe we’re on the cusp of revolutionizing medicine with our cellular rejuvenation capabilities across a range of aging-related diseases, including ophthalmic disorders that involve retinal ganglion cell dysfunction.We are confident our partnership with Forge will have a tremendous impact on our ability to enhance the speed and quality with which we can manufacture our therapeutic candidates as we progress toward the first human clinical trials and continue to develop treatments to reverse diseases of aging by restoring cells to a more youthful state.”