Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Navigating FDA Shakeups and Obesity Drug Wars

Behind the Headlines Episode 28

Ask the Expert

Closing the Gap in Modern Process Control and Automation for QC/QA

Drug Digest December 2025

How It Helps Avoid Facility and Equipment Problems

A “Plan, Prevent, Prove” Approach

Feliza Mirasol is the science editor for 

interest and investment in rare disease drug development, interview with SynaptixBio CEO, Dan Williams

The Evolving Landscape of Rare Disease Pharmaceuticals (Part 1)

Rare disease drug development is emerging as a focal point for innovation and investment amid an evolving landscape of pharmaceutical technologies, according to 

® Group. In the interview, Williams highlights the unique challenges and opportunities in targeting such ultra-rare conditions as TUBB4A-related leukodystrophy, a disorder affecting the myelination of nerves in children.

Williams’ experience spans the full spectrum of drug development, from bench science to clinical trials and corporate operations, providing insight into the complexities of bringing novel therapies to market. “About 80% of rare diseases are genetic and have to do with modifications of genes,” he notes, emphasizing the potential for targeted therapies using advanced technologies. For professionals in drug manufacturing and development, the rare disease sector offers scientific challenges as well as significant incentives, including accelerated regulatory pathways and high market value for successful therapies. This shift is drawing increased attention from investors and companies alike, signaling a broader trend toward precision medicine and the rapid advancement of pharmaceutical technology.

Dan Williams, PhD, studied biochemistry and physiology at the University of Dundee, after which he worked his way up to senior scientist—managing a cell research group and preclinical development. Following this, he moved to drug development, focusing on the organization and management of both manufacturing and clinical trials. Williams then moved to Adaptimmune, developing cell therapies. He set up the development groups, project managing preclinical research and clinical trials, before becoming vice-president of Research Operations. From there, he moved to Meatable as chief product officer. Williams co-founded SynaptixBio in 2021.

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Drug development for rare diseases leverages genetic targeting and drives innovation and investment in pharmaceutical technology as well as precision medicine, according to Dan Williams, PhD, CEO, SynaptixBio.

Dave Miller, PhD, discusses how shifting drug manufacturing to domestic CDMOs can improve quality, reduce rework, and enhance long-term efficiency.

Onshoring’s Unexpected Benefits: Quality, Efficiency, and Supply Chain Resilience

In part 3 of our 5-part interview series on the current CDMO landscape, Dave Miller, PhD, chief scientific officer at AustinPx, addresses the growing trend of onshoring and nearshoring in pharmaceutical development and manufacturing, noting its roots in the current geopolitical climate. He views the shift as both a proactive strategy and a necessary contingency to respond to global uncertainty. “I think it’s a good strategy… either as a primary approach for developing and manufacturing drug products, as well as, the whole supply chain flowing into that drug product,” Miller said.

While many companies have been outsourcing to Eastern markets for speed and cost advantages, Miller notes that some AustinPx clients who have returned operations to the United States are finding unexpected benefits that include reduced miscommunication, fewer data inconsistencies, and avoidance of repeated work. Although domestic operations may initially appear slower and more expensive, the overall process can save time and resources by eliminating inefficiencies and improving quality control, he adds.

Miller observes that what began as a reaction to tariffs and geopolitical instability has evolved into a broader recognition of the value offered by high-quality domestic CDMOs. The trend highlights a shifting emphasis from cost and speed alone to prioritizing quality, reliability, and regulatory confidence—factors that can ultimately shorten timelines and improve outcomes when “doing it right the first time.”

Check out previous parts of the interview series with Miller:

Part 1 - Collaborative Innovation in CDMOs: Dave Miller, PhD, on Early Engagement, IP Strategy, and Advanced Formulation

Part 2 - The Importance for CDMOs of Partnering With a Sponsor’s Mindset

And be on the lookout for parts 3-5 to drop in the coming days!

Dr. Miller brings over two decades of pharmaceutical development experience and has spent his career investigating ways to improve the bioavailability of poorly soluble molecules. As CSO of AustinPx, Dr. Miller leads the pharmaceutics and analytical development teams and oversees the application of the KinetiSol Technology. Prior to his current position, Dr. Miller served as Vice President of Research and Development at DisperSol Technologies and Senior Principal Scientist at Hoffmann-La Roche. Dr. Miller specializes in formulation and processing technologies for improving oral bioavailability of insoluble small molecules. He has applied his expertise toward advancing numerous drug candidates through all stages of development from early discovery to line extension. Dr. Miller is a co-inventor of the pharmaceutical applications of the KinetiSol technology and continues to be a key innovative driver for application and expansion of the platform. He has published over 40 research articles in peer-reviewed journals, authored 8 book chapters, and is co-editor of the First, Second, and Third Editions of the textbook, Formulating Poorly Water-Soluble Drugs. He is a co-inventor on numerous granted and pending patents worldwide. Dr. Miller holds a BS in Chemical Engineering and a PhD in Pharmaceutics from the University of Texas at Austin.

*Editor’s Note: This transcript is a direct, unedited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.

I think we are. We're seeing the effects of onshoring manufacturing. The geopolitical environment right now is uncertain. I think that it's a good strategy, you know, onshoring/ nearshoring either as a primary approach for developing and manufacturing drug products, as well as, the whole supply chain flowing into that drug product.

If not as a primary strategy, as a contingency, as a backup plan, you know, so that they can react to changes in the geopolitical environment, and it seems like they're changing every day. So I think in most of our sponsor companies that we work with and partners, I think it's a very wise strategy to start developing these contingency plans, if not just shifting completely to onshoring domestic development and manufacturing.

We have a number of clients who've begun shifting what they've done historically in China back to the US for those reasons. I think also, you know, what was driving a lot of that work towards the Eastern markets was, you know, speed and cost, right?

These other CDMOs abroad, they offer services for the price is very low. The speed is very high. But a number of our clients as a side effect of, you know, bringing stuff back to the US that they've done historically in China, are recognizing the inefficiencies even though it's on face faster and cheaper.

The issues that you deal with in terms of miscommunications, reiterating work, you know, data inconsistencies, manufacturing issues, the amount of work that needs to be reiterated or redone or the doubt that's created when the data or the documentation isn't complete be taken into account.

And so, these clients that have brought these activities back to the US that were traditionally done in China are starting to recognize the value in onshoring, just from a perspective of it may be more costly and slightly more time consuming on the front end, but you save on the back when you're avoiding these issues that I've just described.

So, what's been driven by the geopolitical environment and tariffs and uncertainty, creating this trend of onshoring, what started with that has, I think, evolved into becoming reacquainted with the value that's earned by working with high-quality domestic CDMOs, like us. It's been a pleasant side effect where a cost and time are at least on the front aren't quite as valued now as the quality of deliverable and the ultimate time saving in doing right the first time. That's been our perspective on the hot topic of conversation.

Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - © Artur - stock.adobe.com

on Aug. 13, 2025 commending Florida Attorney General James Uthmeier’s emergency rule filing to classify certain concentrated 7-hydroxymitragynine (7-OH) opioid drugs as Schedule 1 controlled substances in Florida. The rule makes it illegal to sell, possess, or distribute certain 7-OH drugs in the state.

“The proliferation of concentrated 7-OH products is a major concern for the FDA. I am particularly concerned with the growing availability of concentrated 7-OH products appealing to children and teenagers, such as fruit-flavored gummies, candies, and ice cream cones,” Makary said in the statement (1). “These concentrated 7-OH products have snuck into every community in America, and I'm glad to see Florida's local leaders fighting back and educating Floridian parents, law enforcement professionals, teachers and community leaders about this threat to public health. I encourage other states to act now and use common sense regulation to safeguard the well-being of our youth and Make America Healthy Again.”

In July 2025, FDA recommended a scheduling action to control certain 7-OH products under the Controlled Substances Act (2). A concentrated byproduct of the kratom plant, 7-OH has the potential for abuse, according to the agency, because it can bind to opioid receptors. FDA released a report about the health concerns (3) of 7-OH and how it differs from the kratom plant leaf. The agency also warned about vape and other products that contain 7-OH, calling these products a major concern for the agency, especially those that might appeal to children and teenagers. Labeling for these products might not be accurately labeled, the agency stated, and they might be disguised as kratom.

“Vape stores are popping up in every neighborhood in America, and many are selling addictive products like concentrated 7-OH. After the last wave of the opioid epidemic, we cannot get caught flat-footed again,” said Makary in a press release (2).“7-OH is an opioid that can be more potent than morphine. We need regulation and public education to prevent another wave of the opioid epidemic.”

FDA also announced labeling changes in July for all opioid pain medications that emphasize the risks with long-term use of these products.Data from two large FDA-required observational studies, postmarketing requirements (PMR) 3033-1 and 3033-2, provided new data on how long-term opioid use can lead to serious side effects. These data combined with public comments, medical research, and a lack of studies on the long-term effectiveness of opioids led the agency to require the safety labeling changes so that health care professionals and patients can make more informed treatment decisions.

“The death of almost one million Americans during the opioid epidemic has been one of the cardinal failures of the public health establishment,” said Makary, in a press release (4). “This long-overdue labeling change is only part of what needs to be done—we also need to modernize our approval processes and post-market monitoring so that nothing like this ever happens again.”

“I know firsthand how devastating addiction is—not just for individuals, but for entire families and communities,” said Health and Human Services Secretary Robert F. Kennedy, Jr., in the release. “Today’s FDA action is a long-overdue step toward restoring honesty, accountability, and transparency to a system that betrayed the American people.”

Makary, M.A. Statement from FDA Commissioner Marty Makary, M.D., M.P.H., on Florida Restriction of 7-OH Opioid Products. 

https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-marty-makary-md-mph-florida-restriction-7-oh-opioid-products

FDA. FDA Takes Steps to Restrict 7-OH Opioid Products Threatening American Consumers. Press Release. July 29, 2025. 

https://www.fda.gov/news-events/press-announcements/fda-takes-steps-restrict-7-oh-opioid-products-threatening-american-consumers

Reissig, C. J.; et al. 7-hydroxymitragynine (7-OH): An Assessment of the Scientific Data and Toxicological Concerns Around an Emerging Opioid Threat. 

FDA. FDA Requires Major Changes to Opioid Pain Medication Labeling to Emphasize Risks. Press Release. July 31, 2025. 

https://www.fda.gov/news-events/press-announcements/fda-requires-major-changes-opioid-pain-medication-labeling-emphasize-risks

Articles

FDA Commissioner Marty Makary praised Florida's emergency rule to classify concentrated 7-hydroxymitragynine (7-OH) as a Schedule 1 controlled substance, addressing concerns over its appeal to youth. The FDA had recommended this scheduling action due to 7-OH's potential for abuse, as it binds to opioid receptors. The agency also announced labeling changes for opioid pain medications, emphasizing risks associated with long-term use. These actions aim to prevent another opioid epidemic wave and enhance public health safety by promoting informed treatment decisions and regulatory oversight.

Florida’s emergency rule will classify certain concentration forms of 7-hydroxymitragynine opioid products as Schedule 1 controlled substances in Florida, a step FDA recommended for 7-OH opioids in July 2025.

FDA Commissioner Applauds Florida Restriction on 7-OH Opioids

Dave Miller, PhD, explains how a development-first approach, scientific depth, and regulatory trust build lasting partnerships in the CDMO space.

The Importance for CDMOs of Partnering With a Sponsor’s Mindset



In part 2 of our 5-part interview series on the current CDMO landscape, Dave Miller, PhD, chief scientific officer at AustinPx, discusses how his organization’s evolution from a drug sponsor to a contract development and manufacturing organization (CDMO) shapes its approach to client partnerships. Initially founded to develop novel medicines, the company went on to reforumulate drug products to address unmet therapeutic needs. Once those assets were ready, they were partnered out, leaving behind a fully integrated infrastructure encompassing drug product development, CMC, clinical operations, and regulatory capabilities. This shift allowed the team to apply its expertise not just to its own assets, but across the industry.

Miller describes this as a “by developers for developers” mindset: “We were developers, we’re working with developers, so we are walking in step with them as we move forward.” This perspective means viewing each client’s molecule through the lens of a sponsor—tailoring development strategies as if the product were their own. That includes addressing complex formulation challenges, such as bioavailability issues in small molecules, and extending support beyond standard CDMO services into preclinical, clinical, and regulatory advice.

Miller sees differentiation in the CDMO market as requiring more than technical capabilities; companies must offer something unique and demonstrably useful, backed by a strong scientific and engineering foundation. Trust, he emphasizes, is a critical differentiator. A longstanding track record with regulatory agencies and intellectual property protection builds client confidence, particularly compared to some overseas markets where concerns about IP security persist. For Miller, the goal is long-term partnership, guiding a molecule from formulation through manufacturing tech transfer, while safeguarding client assets and enabling successful regulatory filings. This blend of technical depth, sponsor mindset, and trust-based collaboration, he argues, positions the company as an uncommon partner in a crowded CDMO landscape.

part 1 (Collaborative Innovation in CDMOs: Dave Miller, PhD, on Early Engagement, IP Strategy, and Advanced Formulation

), and be on the lookout for parts 3-5 to drop in the coming days!

*Editor’s Note: This transcript is a direct, unedited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.



I think we're really unique and I've already touched on it a couple times in our previous questions in that we evolved from a sponsor ourselves. So we started with our novel KinetiSol platform. We had this unique tool that differentiated us from any other developers, and we utilized that tool to reformulate drug products to address specific unmet therapy needs.

So that was the evolution. Had a drug, we developed a tool and started utilizing that tool to create differentiated medicines and developed those for almost a decade ourselves. And then at the point that we had developed those assets to a place where they could be partnered, we partnered those assets off.

But what remained as the company was a fully integrated company that was really focused on drug product development as well as, you know, your standard sort of CMC, clinical operations, regulatory activities. So we had this infrastructure, and we realized that whereas before we were sort of leveraging one tool in KinetiSol to develop products, now we had all sorts of tools. And rather than just focus those on our own assets, we could open those to the entire industry, let the whole industry leverage our tools, our expertise towards advancing molecules. So that's kind of where our “by developers for developers” mindset comes from. Like we still feel, we still carry in our DNA that sponsor perspective. So when a client approaches us with their molecule and their problem statement and their target product profile, we view it through their lens. And so we want to develop that product as if it were our own asset. We were developers, we're working with developers, so we are walking in step with them as we move forward.

We've developed all sorts of novel products for our own pipeline. Like I said, with compounds like deferasirox and abiraterone and rivaroxaban and nintedanib, looking at the problem statements for those molecules and tailoring a development program and ultimately an end product to address the deficiencies of those drug products.

And so that framework is exactly how we interact with our clients. We understand their molecule, the challenges, the target product profile, the clinical development plan, and we implement the same sort of development strategies and approaches we took to our own pipeline. So, you know, even though we're an oral development, oral drug product development CDMO, and those are the tangible services we provide, there's a whole host of intangible services where we'll advise a client, on, say, their preclinical strategy, their clinical strategy, regulatory strategy, sort of just as sort of bonus we say, look, we've been here before. We've done this in the way that you're proposing and here's the issues they're going to face, and here's what we would suggest.

So it's sort of these intangible, soft services that our clients get that you don't get anywhere else because we once were a sponsor, we were developers ourselves, and now we work in tandem with other sponsors and other developers, not only to provide innovative formulation solutions that are tailored to their molecule, but also to be a partner, sort of an advisor at the various stages, just based on our own history.

That sort of mentality of a CDMO is becoming more common. But I think we're really unique as you look at CDMOs in the US and around the world. Not too many CDMOs that are fee for service now have a history of developing their own products and are able to apply that history to their clients' assets.

So I think we stand out in that regard. And, like I said, we still very much carry in our DNA that sponsor mentality and we bring that to the table when we start working with clients. And they feel that sort of partnership approach, that intangible advisor, they sign up for pharmaceutical development services, but they get so much more.

And I think that's the feedback we get from our clients. It's a unique experience in working with, Austin Pharmaceuticals.

Again, I think what we are leveraging is scientific expertise. So high science, we have, coming out of our history, we're focused primarily on enabling formulations, bioavailability and for very challenging small molecules that had various issues that impacted bioavailability.

So we developed a deep expertise and a wide array of tools for addressing bioavailability and issues, and creating enabled formulations. We have world class in that regard, but also carry a ton of knowledge in terms of just conventional pharmaceuticals, developing conventional dosage forms, manufacturing efficiency, GMP manufacturing, release, regulatory strategy.

So, we've got the high science approach, but also with a well-rounded experience in general. I think the high science is key, offering novel platforms that differentiate your company against what's out there in the landscape. There’s a lot of noise in the CDMO space.

I think what companies need to be differentiated is offering something different, something useful, and something that has a depth of history like we do with our bioavailability platform, but as I said, it's also about, again, co-development, not putting a client's compound through sort of a decision tree type structured development, but developing a tailored approach and tailored solution specifically for that molecule and demonstrating the scientific expertise and depth, as well as the engineering expertise and depth to take that molecule through formulation development, onto manufacturing tech transfer, to ensure that you're going to be the development and manufacturing partner for your client from start to finish.

The foundation of all of that, what underlies all of that, is having a robust quality system, a history with regulatory agencies, a history with IP generation and maintenance and retention that creates a feeling of trust among your clients that I think still, in comparison to Eastern markets, there's still some uncertainty there with respect to trustworthiness in terms of regulatory and IP, and I think that's still some… there's a natural sort of trustworthiness for domestic CDMOs and particularly CDMOs like us who have such an extensive regulatory and IP history that we will be trustworthy, will protect their asset, not only in terms of the services we provide, but also retaining their IP, generating new IP. And we can be a partner for them in providing what's needed for regulatory filing and standing behind it.

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

AbbVie’s facility in Silicon Valley, South San Francisco, California, USA - June 7, 2023. AbbVie Inc. is an American pharmaceutical company. | Image Credit: © JHVEPhoto - stock.adobe.com

 is expanding its domestic API production in the United States with a $195 million investment in its manufacturing plant in North Chicago, Ill., the company announced on Aug. 12, 2025. (1). Construction on a new API facility at the site is scheduled to commence in the fall of 2025, with the facility expected to be fully operational sometime in 2027.

AbbVie said the new site will allow the company to deepen its chemical synthesis capabilities in the US, supporting production of both current and next-generation medicines in such areas of concentration as neuroscience, immunology, and oncology (1). The investment is just one portion of a previously announced $10 billion capital commitment by AbbVie, intended to encourage innovation and expand critical manufacturing capability and capacity.

"Over the next decade, AbbVie will expand production of API, drug product, peptides, and medical devices in the US to support future medical breakthroughs," said 

, chairman and chief executive officer, AbbVie, in a company press release (1). "This is an important step to maintain US leadership in pharmaceutical innovation and deliver next-generation medicines that make a remarkable impact on patients' lives."

received letters from US President Donald Trump

 on July 31, 2025, in a follow-up to previous correspondence between the White House and the pharmaceutical industry, outlining the steps each company must take within 60 days to lower prescription drug prices in the US to the lowest price offered among other developed nations, a strategy known as most-favored-nation pricing (2).

In June 2025, with companies strategizing their next domestic moves in the wake of the Trump administration’s enactment of high tariffs on key trade partners, AbbVie announced plans to acquire San Diego-based Capstan Therapeutics (3).

That transaction was analyzed by the panel for Episode 20 of 

Given these geopolitical shifts, AbbVie is hardly alone when it comes to doubling down on US investment and expansion. In the summer of 2025 alone, 

 it will invest $1 billion by 2030 to expand generic-drug R&D and manufacturing capabilities in Ohio and New Jersey; 

 on a new sterile injectables development and manufacturing facility in Lexington, Ky.; 

 $50 billion in manufacturing and R&D facilities in the US by 2030; and 

Bora Pharmaceuticals announced plans to invest

 in the expansion of manufacturing and packaging capabilities at its Maple Grove, Minn. facility (4–7).

Jason Waite, international trade expert at Alston & Bird, had much more to say about tariff impacts in a 

AbbVie is headquartered in Illinois and currently has a workforce of more than 11,000 employees, including 6000 jobs across 11 manufacturing sites in the US plus thousands of additional jobs at US suppliers (1).

AbbVie Announces $195 Million Investment to Expand Active Pharmaceutical Ingredient Manufacturing in the US.

 Press Release. Aug. 12, 2025.
2. The White House. 

Fact Sheet: President Donald J. Trump Announces Actions to Get Americans the Best Prices in the World for Prescription Drugs.

AbbVie to Acquire Capstan Therapeutics, Further Strengthening Commitment to Transforming Patient Care in Immunology.

 Press Release. June 30, 2025.
4. Hikma Pharmaceuticals. 

Hikma Pharmaceuticals USA Announces $1 Billion of New US Investment to Further Expand its Domestic Manufacturing and Development of Essential Generic Medicines.

 Press Release. June 28, 2025.
5. Piramal Pharma Solutions. 

Piramal Pharma Solutions Breaks Ground on $90M Expansion Plan.

 Press Release. June 30, 2025.
6. AstraZeneca. 

AstraZeneca Plans to Invest $50 Billion in America for Medicines Manufacturing and R&D.

 Press Release. July 21, 2025.
7. Bora Pharmaceuticals. 

Bora Pharmaceuticals Announces Multi-Year Expansion for Midwest Manufacturing Site, as Upsher-Smith Entity Split is Finalized.

AbbVie is investing $195 million to expand its active pharmaceutical ingredient (API) production in North Chicago, Illinois, with a new facility expected to be operational by 2027. This expansion is part of a broader $10 billion commitment to enhance innovation and manufacturing capabilities in the US, focusing on neuroscience, immunology, and oncology. The move aligns with geopolitical shifts and industry trends, as other pharmaceutical companies also increase US investments. AbbVie, among others, is responding to US government pressure to lower drug prices through most-favored-nation pricing strategies.

The company’s commitment to its North Chicago, Ill. plant is part of a previously announced $10 billion capital investment earmarked for the expansion of US innovation and manufacturing capacity.

AbbVie to Invest $195 Million in US API Manufacturing

Coronavirus 2019-nCov novel coronavirus concept resposible for asian flu outbreak and coronaviruses influenza as dangerous flu strain cases as a pandemic. The virus is made up of purple pills. | Image Credit: © lunarts_studio - stock.adobe.com

 a South San Francisco, Calif.-based clinical-stage biotechnology company that specializes in the development of oral solid-dose recombinant vaccines, received notification that 

 a nonprofit manager of R&D collaborations, would be placing a stop work order on a Phase IIb trial of Vaxart’s oral pill vaccine candidate against COVID-19 (1–3).

 signed by Vaxart CEO Steven Lo, the company reported the stop work order to the United States Securities and Exchange Commission (SEC), affirming that it had so far enrolled approximately half of the targeted number of 10,000 participants for the comparative study, for which a contract was reached with the Biomedical Advanced Research and Development Authority (BARDA) of the US Department of Health and Human Services (HHS) in June 2024 (3).

“[Vaxart] has, to date, not been provided with any further details, including the reason for such stop work order,” the SEC submission said (3). “As of July 14, 2025, the date of its last meeting, the independent Data Safety Monitoring Board for the trial tasked with assessing the safety of the trial had determined that the study could continue to proceed without modification.”

Yet the stop work order was delivered on the same day that 

 it would be winding down its activities related to 

messenger RNA (mRNA)-based vaccine development

 under BARDA, including the canceling or de-scoping of more than 20 projects worth nearly a combined $500 million (4).

Vaxart was not named among the companies with whom HHS is terminating contracts, or rejecting or canceling proposals, but an HHS spokesperson 

 for the reasoning behind the order by sending 

a link to the Aug. 5 press release from HHS

 (4,5). According to the Fierce Biotech article, Vaxart is not developing an mRNA vaccine, though does use one as the control in a Phase IIb trial (5).

While the SEC filing did say that Vaxart was being allowed to “continue efforts associated with the per protocol follow-up” for those in the study who are already dosed, the stop work order is the latest change of direction in what has become a boomerang of a drug candidate for the company. A previous stop work order in February 2025 led Vaxart to lay off 10% of its workforce, only for that order to be lifted in late April (6,7).

Fierce Biotech reported that the total estimated ceiling for the funding from BARDA for the Phase IIb trial was approximately $460.7 million (5).

® Group has reached out to Vaxart for comment on this latest development but did not receive a response at time of publication. This article will be updated with further information pending receipt of a response.

, accessed Aug. 12, 2025.
2. Advanced Technology International. 

, Aug. 5, 2025 (accessed Aug. 12, 2025).
4. HHS. 

HHS Winds Down mRNA Vaccine Development Under BARDA.

 Press Release. Aug. 5, 2025.
5. Taylor, N. P. 

Vaxart Seeks Answers as BARDA Funding for Stop-Start Vaccine Project Dries Up Again.

Vaxart Provides Business Update and Reports Full Year 2024 Financial Results.

, April 24, 2025 (accessed Aug. 12, 2025).

Vaxart, a biotechnology company, received a stop work order for its Phase IIb trial of an oral COVID-19 vaccine candidate, coinciding with the HHS's decision to wind down mRNA vaccine projects. Despite enrolling half of the targeted 10,000 participants, the reason for the order remains undisclosed. Vaxart was not listed among companies with terminated contracts, and the order allows continued follow-up for already dosed participants. This development follows a previous stop work order in February 2025, which was later lifted, affecting Vaxart's workforce and funding.

The company is not developing an mRNA vaccine, but had been using one as the control in a 10,000-subject Phase IIb trial.

BARDA Pulls Plug on Vaxart Oral COVID Vaccine Trial After mRNA Rollback

Dave Miller, PhD, discusses how tailored solutions, trust, and lifecycle planning are redefining partnerships in bio/pharmaceutical development.

Collaborative Innovation in CDMOs: Dave Miller, PhD, on Early Engagement, IP Strategy, and Advanced Formulation



In part 1 of our 5-part interview series on the current CDMO landscape, Dave Miller, PhD, chief scientific officer at AustinPx, emphasizes that the role of contract development and manufacturing organizations (CDMOs) is evolving beyond traditional, transactional service models toward deeper, collaborative partnerships. Miller describes this shift as “true co-development,” where innovation and scientific engagement form the foundation of client relationships. Central to the company’s approach is the application of novel technologies, such as its KinetiSol platform, to solve complex formulation challenges through tailored, science-driven solutions.

Miller notes that differentiation in today’s CDMO landscape hinges on high-touch, high-science engagement, intellectual property (IP) protection, and regulatory expertise. Drawing from AustinPx’s history as a former sponsor company, the team leverages extensive experience with the FDA and international regulatory bodies to guide clients in both domestic and global markets. IP retention, including overlooked compositional IP for drug products, is a priority.

The company increasingly engages with partners earlier in the molecule’s lifecycle—often shortly after candidate nomination—to ensure formulation strategies align with clinical and regulatory objectives. This early involvement supports smoother transitions from preclinical to clinical phases and fosters product designs with a clear line of sight toward commercialization.

According to Miller, the future of the CDMO sector lies in “collaborative innovation” that spans the entire spectrum of development, from early-stage design to market readiness. By operating as an extension of the sponsor’s own development team, AustinPx aims to provide customized, end-to-end solutions that address both immediate formulation needs and the long-term protection and differentiation of the therapeutic asset.

Be on the lookout for parts 2-5 to drop in the coming days!

I think from our experience and what we are dealing with, it's much more about partnerships these days than sort of transactional services. True co-development. And at the heart of that is sort of the novel platforms. You know, we have our KinetiSol platform that really is a driving force behind, you know, how we're partnering and developing products with our clients, but also high science, high touch.

You know, we're dealing with very complex formulation problems, and we offer novel solutions and novel insights. So I think it really comes down to just offering differentiating technologies, really focusing on partnering at the scientific level as opposed to just providing commodity services aimed at, you know, providing deliverables on time.

Also, with respect to ex-US partnerships, our clients also still value what they consider IP retention and trustworthiness, not just with respect to intellectual property, but also in regulatory settings as well. We have a long history as a company; in our prior incarnation, we were a sponsor company ourselves, developing our own pipeline of products and clinical development. So we have a long history of dealing with the FDA and other regulatory agencies outside of the United States. So that's certainly helpful for sponsors that are considering their options both domestic and abroad, as to who to select.

And going forward, it's all about differentiation and trustworthiness. And I think that's where we're really succeeding and as offering tailored solutions as well. Not just taking a molecule and putting it into some rigid framework, decision tree-type project progression, but rather, treating each molecule and each problem as unique and developing customized solutions.

So I think that's how we're at AustinPx differentiating ourselves from, from partners abroad, as well as domestically. I think we've seen sort of a paradigm shift towards much earlier engagement in the development and path of a molecule, interacting with us on the front end, shortly after even candidate nomination, toxicology studies, designing formulations, not just for clinic, but also to successfully transfer a molecule from preclinical into clinical development. So really early engagement. Establishing a CDMO partner who's going to be there as a co-developer, you know, the whole way through the process. So having capabilities that span the full spectrum of product development has been key for us. Um, 'cause we feel like the future in the CDMO space is collaborative innovation, and starting from the beginning in planning the development with the partner with all phases in mind and with the full lifecycle of the product in mind. So again, thinking about formulation insights, you know, that are tailored towards the exact phase of development that the molecule’s in now, but also with line of sight to the next step, aligning that with clinical strategy and regulatory positioning.

Also, again, talking about IP, I think compositional IP for drug products has been overlooked. I think a lot of companies focus primarily on the IP around the molecule as being what ultimately protects the asset. But we've kind of developed our approach, again, stemming from our own history as a sponsor company where the drug product, the composition of matter around the drug product, that IP is what really was protecting the asset.

So, we build that into our programs as well, where we're designing delivery systems specific for that molecule, solving simultaneous problems by tailoring the composition. And then in doing so, those problems… and providing the most robust, and high-performing dosage form, we sort of naturally generate novel composition matter IP and we're looking at that from day one.

In collaboration partners, even though it's years out, we're thinking about the full lifecycle of the molecule and protection of the asset on commercialization. So, that's where I think CDMOs… again, going back to just not just offering transactional services and checking the basic boxes of providing deliverables on a given timeline. It's more of an extension of the sponsor's development team and true collaborative innovation co-development. I think that's the expectation now. That's what we're doing.

Tokyo-based Nxera Pharma (Nxera) announced on Aug. 6, 2025 the launch of an expanded drug discovery pipeline targeting obesity and related metabolic diseases. The company’s efforts build on independent research in addition to existing collaborations with Pfizer and Eli Lilly and Company, focusing on a range of validated G protein-coupled receptor (GPCR) targets (1–3).

Nxera’s lead asset is a wholly owned oral small molecule glucagon-like peptide-1 (GLP-1) agonist program, which it developed with differentiated chemistry and managed separately from Pfizer’s PF-06954522. Nxera said in a company press release that the separation allows it to maintain full control over development timelines and strategy.

“Nxera scientists were among the pioneers in elucidating the structure of the GLP-1 receptor and other key receptors being targeted, giving us a clear advantage in developing potentially best-in-class next generation drug candidates. For the past 12 months, Nxera scientists have been advancing a broad metabolic disease pipeline to capitalize independently on our differentiated science,” said Chris Cargill, president and CEO, Nxera, in the release (1).

Multiple GPCR-targeted programs in development

In parallel with the GLP-1 program, Nxera is progressing six additional obesity and weight-management drug programs, including:

three small-molecule agonists targeting GIP, apelin, and amylin

one small molecule antagonist targeting GIP

These programs employ Nxera’s NxWave GPCR-focused structure-based design platform with which the company can identify novel lead molecules with distinctive chemistry. The process combines proprietary NxHit small-molecule libraries with NxStaR proteins for DNA-encoded library screening. The company aims to produce candidates that differ from other drugs in global development.

“Today, we reveal the addition of these programs to our discovery portfolio and are confident in achieving significant progress over the mid-term, delivering clear value for shareholders and patients in the rapidly expanding obesity market, projected to exceed US$100 billion annually in global sales [4],” Cargill added in the release.

According to Nxera, its pipeline strategy addresses several industry and clinical priorities such as:

long-term weight maintenance through convenient, scalable oral therapies

treatment of obesity-related co-morbidities, including cardiovascular, renal, and liver diseases

reduced side effects and tailored options for populations such as the elderly, post-menopausal women, and patients with sarcopenia.

Beyond obesity and metabolic disorders, Nxera continues research in neurology, gastroenterology, and immunology, applying the same GPCR-targeted discovery methods.

Pfizer program discontinued, future discussions planned

Separately, Pfizer recently ended development of its Phase I small-molecule GLP-1 agonist PF-06954522 in early August 2025 (5), which originated from a collaboration with Nxera. The decision, according to Nxera, was due to portfolio reprioritization rather than safety concerns. Nxera plans to discuss with Pfizer the possibility of advancing GLP-1 molecules identified during their joint research.

Nxera’s announcements underscore a broader trend in obesity drug development: expanding beyond injectables into oral therapies, diversifying receptor targets, and applying high-throughput, structure-based screening to accelerate discovery. With obesity drug sales forecast to surpass $100 billion annually, the field is poised for increasingly intense scientific and commercial activity.

Nxera Launches Broad Proprietary Pipeline Targeting Obesity and Chronic Weight Management

. Press Release. Aug. 6, 2028.
2. Pfizer. 

Sosei Subsidiary Heptares Enters Strategic Drug Discovery Collaboration with Pfizer Inc. Focused on GPCR Targets Across Multiple Therapeutic Areas

. Press Release. Nov. 30, 2015.
3. Nxera Pharma. 

Sosei Heptares and Lilly Enter Multi-target Collaboration and License Agreement in Diabetes and Metabolic Diseases

. Press Release. Dec. 16, 2022.
4. McKinsey & Co. 

GLP-1s Are Changing Obesity Care. What Comes Next

? mckinsey.com, June 1, 2025 (accessed Aug. 11, 2025).
5. Waldron, J. Pfizer's Embattled Obesity Program Loses Another GLP-1 Over Poor Data and Strong Competition. 

, Aug. 5, 2025 (accessed Aug. 11, 2025). 

https://www.fiercebiotech.com/biotech/pfizers-embattled-obesity-program-loses-another-glp-1-drug

Tokyo-based Nxera Pharma has launched an expanded drug discovery pipeline targeting obesity and related metabolic diseases, leveraging collaborations with Pfizer and Eli Lilly. Their lead asset is a proprietary oral GLP-1 agonist program, developed independently from Pfizer's discontinued PF-06954522. Nxera is advancing multiple GPCR-targeted programs, including small-molecule agonists and antagonists, using their NxWave platform. The pipeline aims to address long-term weight maintenance, obesity-related co-morbidities, and reduced side effects. The company continues research in neurology, gastroenterology, and immunology, applying GPCR-targeted methods.

The new targeted pipeline will advance Nxera’s oral GLP-1 agonists, novel GPCR targets, and structure-based drug design to address obesity and related health conditions.

Nxera’s New GLP-1 and GPCR-Targeted Drug Programs Aim to Advance Obesity and Metabolic Disorder Therapies

, PhD, is senior manager, scientific affairs at bioMérieux. Email: 

Stem cells - undifferentiated (immature) cells on the background of a DNA helix, 3d rendering | Image Credit: © ustas - stock.adobe.com

As personalized, life-saving treatments advance with the widespread adoption of cell and gene therapies (CGTs), diagnostic quality control partners face a critical challenge: adapting traditional methods to meet faster production timelines and the limited shelf life of cell therapy products while ensuring accuracy and reliability in results. The urgency of delivering these therapies to patients necessitates a reevaluation of existing quality control frameworks. Traditional methods that may have been sufficient for conventional pharmaceuticals often fall short when applied to the dynamic and sensitive nature of cell and gene therapies. This adaptation requires not only technological innovation but also a shift in mindset toward more agile and responsive quality control processes.

New therapies, new quality control processes

Cell and gene therapy manufacturers face significant quality control testing challenges due to the complex and highly variable nature of these therapies. One of the primary challenges is the need to develop novel testing methods that can accurately assess the unique attributes of these products. Unlike traditional pharmaceuticals, cell and gene therapies are living products, which means their characteristics can change over time and under different conditions. This variability necessitates the development of testing methods that can provide reliable and reproducible results despite these fluctuations.

Additionally, the limited number of viable sampling strategies poses a significant hurdle. Given the precious and often scarce nature of cell and gene therapy products, extensive sampling for quality control purposes is not always feasible. Manufacturers must therefore devise strategies that maximize the information obtained from minimal sample volumes, ensuring that enough product remains available for therapeutic use. Concerns over raw material consistency further complicate the quality control landscape. The biological nature of these therapies means that raw materials can vary significantly from batch to batch, potentially impacting the final product's quality and efficacy. Ensuring consistency in raw materials is crucial for maintaining the integrity of the therapy.

The potential for cross-contamination is another critical concern. The manufacturing processes for cell and gene therapies often involve handling multiple products simultaneously, increasing the risk of cross-contamination. Implementing stringent contamination control measures is essential to prevent this and ensure the safety and purity of the final product. Moreover, the need for rapid results to minimize patient wait times adds another layer of complexity. Patients awaiting cell and gene therapies often have urgent medical needs, making timely delivery of these treatments critical. Quality control processes must therefore be optimized to provide quick and accurate results without compromising on thoroughness.

All these challenges must be addressed while adhering to strict regulatory standards. Regulatory bodies have established rigorous guidelines to ensure the safety, efficacy, and quality of cell and gene therapies. Manufacturers must navigate these regulations carefully, ensuring that their quality control processes meet all necessary requirements.

Minimizing 'over-testing' to maximize product

An emerging consideration unique to cell and gene therapies is the need to develop a testing plan that minimizes over-testing, thereby maximizing the amount of sample, or product, available for patient use. This is particularly important given the often-limited availability of these highly specialized and valuable products. Strategic decisions in testing—such as choosing the right testing partner, streamlining upstream and downstream processes, and ensuring automation and accuracy—can facilitate a faster, more efficient path from lab to patient. By carefully selecting a testing partner with expertise in cell and gene therapies, companies can leverage specialized knowledge and advanced technologies to optimize their testing protocols.

Furthermore, because no two facilities or products are identical in the cell and gene therapy space, diagnostic partners are uniquely positioned to holistically analyze a customer’s development and manufacturing processes. They can provide tailored recommendations to enhance testing efficiency and develop new analytical methods. For instance, next-generation sequencing can be employed to accurately assess critical quality attributes such as cell viability, purity, potency, and genetic integrity. This level of precision is crucial for novel cell and gene therapy products where standard assays may not be suitable. Additionally, diagnostic partners can assist in the validation and implementation of these new methods, ensuring they meet regulatory requirements and industry standards.

Moreover, the dynamic nature of cell and gene therapy development necessitates a flexible and adaptive approach to testing. As new technologies and methodologies emerge, continuous collaboration with diagnostic partners can help companies stay at the forefront of innovation. These types of ongoing partnerships can lead to the discovery of more efficient testing strategies, ultimately reducing time-to-market and improving patient outcomes. By investing in robust testing frameworks and fostering strong relationships with diagnostic partners, companies can navigate the complexities of cell and gene therapy development more effectively.

Recently, bioMérieux has collaborated with various customers to develop customized testing plans that address the unique needs of individual facilities and products across multiple categories in relation to mycoplasma testing. This collaboration has been instrumental in ensuring that the testing protocols are tailored to the specific requirements of each facility, thereby enhancing the accuracy and reliability of the results.

During these conversations, several themes common to a collaborative approach have been regularly mentioned. They are as follows:

 When a sample has a higher cell density, testing an entire batch becomes impractical due to volume and resource constraints. High cell density can lead to challenges in sample handling and processing, making it difficult to obtain representative results from a single batch. The rapid advancement of cell and gene therapy has resulted in limited FDA guidance on the appropriate batch volume to test and whether the focus should be on the cells or the matrix. This lack of clear regulatory direction can create uncertainty for manufacturers, who must balance the need for thorough testing with the practical limitations of their processes. In such scenarios, an experienced partner can provide valuable guidance as new regulations are introduced. By leveraging their expertise and staying abreast of the latest regulatory developments, these partners can help manufacturers navigate the complexities of mycoplasma testing. They can offer insights into the most effective sampling strategies, ensuring that the testing is both comprehensive and efficient.

Selection of relevant species for validation.

 For mycoplasma detection method validation, testing every compendial species is unnecessary. Instead, using a representative selection of common and relevant species encountered in cell culture environments streamlines the process, making it more efficient and cost-effective. This targeted approach ensures that the validation process remains practical while maintaining adequate sensitivity and specificity across potential contaminants. By focusing on the most prevalent and relevant mycoplasma species, laboratories can optimize their resources and reduce the time and cost associated with extensive testing. This approach also allows for a more manageable and focused validation process, ensuring that the methods are robust and reliable for detecting the mycoplasma species most likely to be encountered in the specific cell culture environment.

The validation process should demonstrate sufficient coverage against likely mycoplasma species in the samples. This involves carefully selecting species that are representative of the broader mycoplasma population, ensuring that the detection methods can identify a wide range of potential contaminants. The goal is to achieve a balance between thoroughness and efficiency, providing confidence in the testing results without the need for exhaustive species-by species validation.

With the increasing use of molecular methods like polymerase chain reaction (PCR) for mycoplasma detection, the need to test against every species might be less critical. PCR assays can often detect a broad range of mycoplasma species based on conserved genetic sequences. These sequences are shared among different mycoplasma species, allowing for the detection of multiple species with a single assay. This capability significantly enhances the efficiency of the validation process, as it reduces the need for extensive species-specific testing. Moreover, the use of molecular methods like PCR offers additional advantages, such as increased sensitivity and faster turnaround times compared to traditional culture-based methods. These benefits are particularly important in the context of cell and gene therapies, where timely and accurate detection of contaminants is crucial for ensuring product safety and efficacy.

Analytical testing strategies for upstream and downstream processes. 

The advancement of cell and gene therapies (CGTs) necessitates a reevaluation of traditional quality control methods to accommodate faster production timelines and the unique characteristics of these therapies. Manufacturers face challenges such as developing novel testing methods, managing limited sampling strategies, ensuring raw material consistency, and preventing cross-contamination. Collaborations with diagnostic partners are crucial for optimizing testing protocols, leveraging advanced technologies, and ensuring regulatory compliance. By focusing on efficient quality control processes, the industry can enhance the speed and reliability of delivering life-saving CGTs to patients.

The scaled adoption of cell and gene therapies demands a new era of agile, precise, and efficient quality control methods. Manufacturers and diagnostic partners must collaborate to create innovative, compliant testing strategies that preserve product integrity, meet tight timelines, and deliver life-saving treatments faster.

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