Digital Transformation in Pharma Manufacturing

Patent Cliff Deals, Tariff Pressures, and the In Vivo Cell Therapy Bet

Behind the Headlines, Episode 23

ICH Q12 and Post-Approval Changes

Speed, Flexibility Top Large-Molecule Manufacturing Trends

Drug Digest July 2025

Key Insights from Industry Survey Results

Bio/Pharma's Tariff Response

Genetic engineering concept. Collage with glowing DNA molecule over female hand on blue background | Image Credit: © Prostock-studio

 (Mirai) announced on May 20, 2025 that the company has entered into a strategic collaboration with 

 aimed at advancing the development and manufacturing of nucleic acid therapeutics (1). The agreement brings together Mirai’s technology platform for the design and optimization of genetic medicines with Thermo Fisher’s development and current good manufacturing practice (CGMP) capabilities.

The collaboration will initially focus on leveraging Thermo Fisher’s RNA and advanced formulations facility to support the industrialization of Mirai’s development candidates, according to the release (1). In addition to technical collaboration, the agreement includes a direct investment into Mirai as part of the broader partnership between Thermo Fisher and 

·Mirai Bio and Thermo Fisher are partnering to integrate design, delivery, and CGMP manufacturing for nucleic acid-based therapeutics.

·The collaboration will leverage Thermo Fisher’s RNA and advanced formulations facility to support scalable development of genetic medicines.

·Mirai’s machine intelligence platform will be combined with Thermo Fisher’s technical and manufacturing expertise to streamline drug development workflows.

“This collaboration is an expansion of our long-standing relationship with Thermo Fisher and a reflection of our shared vision for accelerating the development of enabling technologies to develop first-in-class therapies,” said Justine 

, executive partner, origination and corporate development at Flagship, in the release (1). “In this case, the combination of Mirai’s innovation in targeted delivery and cargo design and Thermo Fisher’s deep technical and commercial manufacturing expertise will maximize opportunities for the co-creation of novel medicines and help realize the full potential of both enterprises.”

Mirai offers end-to-end services for partners in the genetic medicine space. The company’s machine intelligence-based platform enables high-throughput design and 

 testing of targeted delivery systems, facilitating rapid and scalable development processes.

“Thermo Fisher is a trusted industry leader with deep technical expertise and proven developmental and GMP services and commercial manufacturing capabilities,” said Travis Wilson, executive chairman of Mirai and growth partner, Flagship Pioneering, in the release from Mirai (1). “Coupling these with Mirai’s unique delivery and genetic medicine optimization technologies will support accelerated program development and commercialization for our life science partners worldwide. We’re thrilled to enter into this collaboration to further strengthen Mirai’s platform and fully realize our vision of co-creating targeted, potentially curative programmable medicines with end-to-end optimization.”

, president, drug substance, pharma services at Thermo Fisher, stated, “Mirai is on a journey to break down the barriers currently limiting the full potential of genetic medicines and offer fully integrated design, delivery, and manufacturing capabilities to innovative partners. We’re excited to combine our life sciences expertise and manufacturing capabilities with Mirai’s machine intelligence-powered platform to help bring the next generation of medicines to patients in need.”

This new collaboration is part of an ongoing effort by both organizations to address key challenges in genetic medicine development and to jointly create integrated solutions that can support the next wave of therapeutic innovation (1).

Thermo Fisher is focused on working sustainably. Adam Goldstein, senior director of R&D Collaborations at Thermo Fisher Scientific, 

 that sustainability-by-design in bioprocess development involves integrating sustainable practices and principles from the very beginning of the development process, which includes considering environmental impacts, resource efficiency, and waste reduction during the design phase, to create processes that are not only efficient but also lower the environmental footprint (2).

Goldstein went on to emphasize the importance of the innovations that result from adopting a sustainability-by-design approach because they can lead to improved product quality and process efficiency. “Driving higher titer in smaller volumes means yielding higher throughput of product in a smaller footprint. The benefits are lower cost of goods in manufacturing while simultaneously delivering the product in a much more sustainable fashion with regards to emissions reductions,” he commented (2).

Mirai Bio Announces Partnership with Thermo Fisher Scientific to Accelerate Development of Genetic Medicines

Sustainability by Design in the Context of Bioprocess Development.

Articles

Mirai Bio and Thermo Fisher partner to integrate AI-driven design with CGMP manufacturing, aiming to streamline development of genetic medicines.

Mirai Bio and Thermo Fisher Partner to Advance Genetic Medicine Manufacturing

·Grifols received FDA clearance to initiate a Phase II trial of GRF312, an immunoglobulin (IG)-based eye drop for treating dry eye disease.

·The trial builds on Phase I/II data showing significant symptom reduction with IG drops and no difference in safety profile compared with placebo.

·This marks a potential first use of IG for an ocular surface indication, expanding its therapeutic scope beyond systemic applications.

FDA on paper texture | Image Credit: © Araki Illustrations.

, a global healthcare company specializing in plasma-derived medicines, announced on May 21, 2025 (1) that it has received clearance from 

 to initiate a Phase II clinical trial of its immunoglobulin-based eye drop, GRF312 Ophthalmic Solution, for the treatment of dry eye disease (DED).

Dry eye disease is a chronic, multifactorial inflammatory condition that affects more than 100 million individuals worldwide, according to a company press release (1). It is characterized by insufficient tear production or excessive tear evaporation, leading to ocular discomfort, pain, and in severe cases, irreversible damage to the ocular surface. Standard treatments—such as artificial tears, corticosteroids, cyclosporine, and lifitegrast—may take weeks or months to deliver meaningful relief and do not work for all patients.

The Phase II trial is expected to begin in the third quarter of 2025 and will evaluate the safety, tolerability, and efficacy of GRF312 in 100 patients with DED. The clinical program builds upon a Phase I/II pilot study led by Grifols’ partner 

University of Illinois College of Medicine, Department of Ophthalmology

, according to Grifols (1). That study demonstrated a significant reduction in both signs and symptoms of DED after eight weeks of treatment with the IG formulation, administered twice daily. The tolerability profile was comparable to that of placebo.

“We believe our immunoglobulin eye drops have the potential to make a qualitative leap in how dry eye disease is treated,” said Dr. 

Jörg Schüttrumpf, Grifols chief scientific innovation officer

 in the May 21 release (1). “Research suggests that the broad-spectrum anti-inflammatory and immunomodulatory properties of an ocular surface IG are superior to currently approved therapies, including artificial tears and anti-inflammatories. We are using our extensive and deep knowledge of immunoglobulins to benefit the millions of patients globally who aren’t receiving sufficient relief.”

Development of GRF312 stems from a 2023 collaboration and licensing agreement between Grifols and Selagine, under which Grifols acquired exclusive global rights to the IG-based ophthalmic treatment (2). The agreement leverages Grifols’ capabilities in developing and manufacturing immunoglobulin therapies and Selagine’s research and clinical experience in ophthalmology.

“Selagine and Grifols joining efforts and complementary expertise successfully led the treatment to a Phase II clinical trial,” said 

, founder and President of Selagine and BA field professor of ophthalmology at the 

University of Illinois College of Medicine

. “The results obtained to date and this promising development reinforce the potential of this novel treatment approach to significantly improve the quality of life of dry eye disease patients.”

The trial marks one of the first formal efforts to investigate immunoglobulin as a therapeutic agent for the ocular surface—a potential first-in-class application. If successful, GRF312 could represent a new mechanism of action for addressing inflammation and immune dysregulation in DED, with implications for both patient outcomes and therapeutic innovation.

 that its subsidiary Biotest had garnered its first FDA approval with the intravenous immunoglobulin therapeutic, Yimmugo (3). Set to be distributed by Kedrion, preparations for an early 2025 launch in the United States was underway as of October 

2024 for Yimmugo (4). No further news of the launch was available at the time of writing.

Grifols Receives FDA Clearance of IND Application for Phase 2 Trial of Immunoglobulin Drops for Dry Eye Disease

Grifols Enters into Global Collaboration and Licensing Agreement with Selagine to Develop Immunoglobulin Eye Drops to Treat Dry Eye Disease. Press Release

FDA Approves Immunoglobulin Therapeutic to Treat Primary Immunodeficiencies

Expanding Treatment Options: Yimmugo® Coming Soon for PID Patients in the US

Grifols received FDA clearance to begin a Phase II trial of GRF312, an immunoglobulin eye drop for dry eye disease, aiming to improve current treatments.

IND Application Cleared for Phase II Trial of Grifols’ Dry Eye Disease Drops

Chris Spivey is director of Industry Relations and Strategic Partnership, MJH Life Sciences.

Jamie Baumgartner, Jonathan Grinstein, and John Wilkerson go behind the headlines to discuss the implications of personalized gene-editing therapies, more HHS policy and funding updates, and the latest tariff-related investments. 

Behind the Headlines, Episode 17: Bespoke CRISPR Therapy, Executive Order Fallout, and more

 is a bi-weekly panel discussion examining the latest trends, readouts, and other factors that drive pharmaceutical news and innovation. Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seeking to highlight the more enduring lessons that hide behind the headlines.

In episode 17, we’re joined by panelists Jamie Baumgartner, President and CEO of Keystone Symposia on Molecular & Cellular Biology; Jonathan D. Grinstein, PhD, North American Editor for Inside Precision Medicine; and John Wilkerson, Washington correspondent for 

. The panelists dove into the beating heart of the life science ecosystem, discussing why researchers are currently attending conferences, what knowledge they are sharing, and what gaps in their knowledge they are looking to fill.

American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

, which was recently held in New Orleans, was the chosen vehicle to announce a world first in 

 therapeutics. Scores of people have already benefited from receiving CRISPR-based therapies for genetic conditions such as sickle cell disease. In this case, collaboration among clinicians, scientists, and physicians helped create a custom-tailored therapy to address an infant with CPS1 deficiency. “This compromised his ability to process the nitrogen-containing compounds produced when the body breaks down protein. As a result, his blood had high levels of ammonia, a compound that is particularly toxic to the brain,” according to a news briefing from 

. (1) “In just six months, Muldoon received his first dose. After that initial dose, Muldoon could safely eat the amount of protein recommended for his age.” (1). Although a big success, the panel considered what the probabilities are for cost lowering, alongside widespread access deployment.

Policy changes at the Department of Health and Human Services (HHS), funding cuts, and tariffs were also discussed, with Sanofi following suit by announcing a 5-year, $20 billion investment into US manufacturing (2). However, the “most favored nation” proposed executive order caused Roche to announce it is beginning to rethink its own $50 billion investment announcement a week earlier (3, 4). 

1. Ledfod, H. World’s first personalized CRISPR therapy given to baby with genetic disease. 

. May 15, 2025. https://www.nature.com/articles/d41586-025-01496-z

2. Sanofi to invest at least $20 billion in the US through 2030, growing investments in science and expanding domestic manufacturing. Press release. Sanofi. Published May 14, 2025. https://www.prnewswire.com/news-releases/sanofi-to-invest-at-least-20-billion-in-the-us-through-2030-growing-investments-in-science-and-expanding-domestic-manufacturing-302455566.html

3. Roche warns that it needs to reconsider its U.S. investments due to Trump's drug pricing order. Bloomberg. May 16, 2025. 

https://www.bloomberg.com/news/articles/2025-05-14/roche-warns-us-investments-at-risk-from-trump-drug-price-plans?embedded-checkout=true

4. Franco, M. Trump Administration Revives Most-Favored-Nation Drug Pricing: Here's What to Know. Holland and Knight. May 14, 2025. 

https://www.hklaw.com/en/insights/publications/2025/05/trump-administration-revives-most-favored-nation-drug-pricing

Videos

Behind the Headlines, Episode 17: Bespoke CRISPR Therapy, Executive Order Fallout, and More

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

Abnormal plasma cell or b-cell in multiple myeloma emitting paraprotein 3d illustration | Image Credit: © LASZLO - stock.adobe.com

CellCentric’s $120 million Series C funding supports late-stage development of inobrodib, a novel oral p300/CBP inhibitor for multiple myeloma.

Inobrodib’s mechanism targets MYC and IRF4 expression, addressing therapy resistance in relapsed or refractory multiple myeloma patients.

Planned trials include phase II/III studies and combination therapies with bispecific antibodies, advancing toward potential regulatory approval.

A $120 million Series C funding round was completed by CellCentric, a Cambridge, UK-based clinical-stage biotechnology company, for the advancement of inobrodib, which CellCentric is developing as a potential first-in-class oral treatment for several specific types of cancer, notably multiple myeloma (1).

CellCentric describes inobrodib as a novel, oral p300/CBP inhibitor (1). The p300/CBP coactivator family is comprised of p300—also known as adenovirus early region 1A, or E1A, binding protein p300—and CBP, or CREB-binding protein, in which CREB represents cAMP response element-binding protein, with cAMP standing for cyclic adenosine monophosphate (2,3). Targeting this has a desired end result of lowering the expression of transcription factor MYC and interferon regulatory factor 4 (IRF4), two key drivers of cancer (1,4).

New pathway for multiple myeloma treatment

According to CellCentric, the majority of patients diagnosed with multiple myeloma eventually succumb to the disease, and many are either not eligible for, or become resistant to, available therapies (1). Inobrodib’s oral delivery and mechanism of action provide another option, and a phase IIa dose optimization clinical study is currently ongoing that is combining inobrodib with two oral treatments, pomalidomide and dexamethasone.

The Series C funding will be used to support initiation of a phase II/III study in heavily pretreated patients with multiple myeloma, development activities for a phase III program to start in mid-2026, and initiation of trials to start almost instantaneously, within the second quarter of 2025, combining inobrodib with bispecific antibodies (1).

Financing was co-led by RA Capital Management and new investor Forbion, with participation from Avego Bioscience Capital and BrightEdge, the venture capital and impact investment arm of the American Cancer Society.

“We are delighted to secure the investment required to continue to advance inobrodib fully and as effectively as possible,” Will West, CellCentric CEO, said in a company press release issued May 19, 2025 (1). “This is a significant raise in a challenging market. Today’s announcement is a testament to the data we have in hand, the clear clinical and commercial opportunity inobrodib represents, and the strength of our expanded team.”

“CellCentric has developed an innovative and impactful therapy for multiple myeloma with inobrodib, a first-in-class oral p300/CBP inhibitor,” Jasper Bos, general partner at Forbion, said in the release; Bos is joining the CellCentric board (1). “This novel agent has demonstrated promising efficacy and a manageable safety profile in early clinical trials. We are enthusiastic about supporting CellCentric as it advances inobrodib into registration studies, aiming to transform multiple myeloma treatment across various stages of the disease.”

The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has moved forward several potential treatments for multiple myeloma since late 2024. 

 resulted in a recommendation for approval for a generic form of pomalidomide made by Teva, while conditional marketing authorization was granted for a trabectedin generic, from Accord Healthcare, for treatment of relapsed or refractory multiple myeloma at CHMP’s February 2025 summit (6,7).

CellCentric Secures $120 Million Investment to Advance Inobrodib for Treating Multiple Myeloma.

 Press Release. May 19, 2025.
2. National Cancer Institute. 

Definition of p300/CBP Bromodomain Inhibitor CCS1477.

 (accessed May 20, 2025).
3. ScienceDirect. 

 (accessed May 20, 2025).
4. Bae, S.; Park, P. S. U.; Lee, Y.; et al. MYC-Mediated Early Glycolysis Negatively Regulates Proinflammatory Responses by Controlling IRF4 in Inflammatory Macrophages. 

Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 16-19 September 2024.

Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 February 2025.

Inobrodib is being developed as a first-in-class oral cancer drug that the company says will be able to treat not only multiple myeloma, but other specific cancers as well.

CellCentric Completes $120 Million Series C Funding Round for Multiple Myeloma Treatment Inobrodib

· ACG’s PCM enables on-demand production of personalized capsules using real-time health data, precise dosing, and automated manufacturing.

· The ACG–AoY partnership combines advanced capsule tech and formulation expertise to create science-backed, customized supplements.

· With 1,500+ machines planned in Europe and €500M revenue forecasted, PCM shows strong commercial potential for scalable personalized nutrition.

A group of antibiotic pill capsules falling. Healthcare and medical 3D illustration background | Image Credit: © James Thew - stock.adobe.com

Thanks to an exclusive partnership with Art of You (AoY)—a personalized supplement development company—the ACG Group (ACG) announced its world-first Personalised Capsule Machine (PCM) for creating custom-formulated supplement capsules that can be individualized on demand to the specific needs of a given patient (1). As a fully integrated manufacturing solutions provider for the global pharmaceutical and nutraceutical industries, ACG developed PCM to create individualized supplements by merging precise dosing, real-time health data, and automated capsule production.

The AoY–ACG partnership integrates AoY’s formulation knowledge and experience along with ACG’s beadlet and capsule technologies to create a one-of-a-kind system for delivering bespoke supplement solutions that match individuals’ biomarkers, lifestyle choices, and health goals.

“A True Leap Forward in Personalized Nutrition”

“After five years in development, we are thrilled to launch the PCM and collaborate with Art of You on this groundbreaking initiative. It represents a true leap forward in personalised [sic] nutrition,” said Aaron Quinn, senior manager for Europe at Vantage Nutrition, part of the ACG Group. “This partnership underscores our commitment to growth through innovation and focus on advancing health solutions globally. With over 1,500 PCM machines planned for deployment across Europe in the next five years and projected revenue of more than €500 million over the next three, this initiative highlights ACG’s leadership in delivering cutting-edge capsule and beadlet technologies to the market.”

Adding to Quinn’s sentiments, AoY Co-Founder Pablo Smolders said “We are proud to showcase this exciting new collaboration at Vitafoods Europe. By leveraging ACG’s exceptional capsule technologies alongside our own expertise, we are bringing our vision of science-backed, personalised wellness closer to reality.”

The news comes as AoY team members are joining ACG at Vitafoods Europe, held May 20-22 in Barcelona (2), where they’re discussing the science, vision, and technology that helped that innovation come to life.Also at the show, ACG’s Vantage Nutrition division is presenting “advanced ingredient delivery technologies including controlled release beadlets, liquid-filled hard capsules, and cap-in-cap dosing solutions,” according to a press release shared by ACG (1).

PCM, according to ACG, is able to deliver:

Scientifically validated, research-based compositions

Enhanced absorption and nutrient delivery

Plant-based, flavor-neutral, and free from microplastics

Eco-conscious production meeting pharmaceutical standards

showcased it’s line of sustainable packaging solutions

 at Pharmapack Europe. In particular, ACG highlighted its CelluPod paper-based, recyclable, and compostable blister system for products that require low water vapor transmission rates (WVTR). The company also featured its packaging solutions that are free of polyvinyl chloride (PVC). These include its PVC and Halogen-Free Thermoforming Blister System for products requiring low to medium WVTR. ACG also provides an eco-friendly alternative in its Bio D PVC. The company’s Halogen-Free Cold Form Blister System also provides a high-performance sustainable solution without halogen and PVC.

 sat down Anil Andrade, vice-president of Global Sales at ACG World, during CPHI Milan to discuss growth in demand in the pharma industry and efforts put forth to achieve sustainable operations. In the interview, Andrade talked about the changing perceptions of the industry and the importance of sustainability in pharmaceutical operations. During the past 10 to 15 years, ACG has seen global growth in demand, and more so in Europe, North America, and Asia. Andrade also emphasized efforts to achieve sustainability to reduce the impact on the environment (3).

“Manufacturing capsules consumes a lot of water, and we have been trying to make our operations more efficient so that they consume less water, less paper, less plastic,” Andrade explained. “In Croatia, as an example, we have reduced the paper consumption by almost 25 tons in a year. Same with plastic, we have reduced the consumption of plastic by 25 tons, and that has related to the benefits that we see going forward.”

ACG unveils patented personalised capsule technology

CPHI Milan 2024: Growth and Sustainability in the Pharma Industry

ACG partnered with Art of You to launch the world-first Personalised Capsule Machine, enabling on-demand, customized supplement capsules based on individual health data.

Personalised Capsule Machine Unveiled by ACG

Coronavirus Covid-19 background - 3d rendering | Image Credit: © Production Perig - stock.adobe.com

After consulting with the World Health Organization, marketing authorization holders, and international partners and examining worldwide and animal study data, the European Medicines Agency (EMA) announced on May 16, 2025, that its Emergency Task Force (ETF) would be recommending that COVID-19 vaccines be updated for the 2025-2026 vaccination campaign (1).

The recommendation is based on ETF’s desire to target LP.8.1, a variant of the SARS-CoV-2 virus that caused COVID-19, which is the most prevalent variant circulating worldwide as of the first third of 2025 (1). It has passed the previously dominant JN.1 variant, for which previous vaccine formulas were updated, and is different from the JN.1 family.

 detailing the recommendation, EMA said LP.8.1 and XEC—the latter being another variant that had become dominant as 2024 turned to 2025—were both part of the BA.2.86 family of Omicron variants, antigenically different from the XBB variant that circulated in 2023 and others that circulated previously (2).

“A large body of evidence is now available to anticipate that, even if COVID-19 vaccines remain effective at preventing severe disease and death caused by new emerging variants, protection tends to decrease as the virus evolves into more antigenically distant variants,” the document said (2). “Studies have shown that matching the content of vaccines to the circulating viruses improves protection against the disease.”

Vaccines for other variants still approved

Novavax’s recombinant protein-based, non-messenger RNA (mRNA), marketed as Nuvaxovid, has gained full market approvals in the European Union and other countries and areas around the world as a COVID-19 treatment, and yet has only been available to date under emergency use authorization in the United States. On May 19, 2025, FDA approved the Biologics License Application for this vaccine in adults aged 65 and older, and individuals aged 12-64 who have at least one underlying condition putting them at high risk for severe outcomes (3).

However, the Novavax vaccine is formulated to target the JN.1 variant. While JN.1 sublineages are still emerging, EMA said, LP.8.1 has been shown to exhibit strong humoral immune evasion and holds potential to have the greatest growth advantage and increased transmissibility compared with other variants (2).

Despite that, the ETF said that vaccines targeting the JN.1 or KP.2 strains could still be considered for vaccination campaigns beginning later in 2025, until treatments specifically targeting LP.8.1 become available (1,2).

In the United States, the 2024-2025 COVID-19 vaccination campaign was launched with 

 that it had granted an emergency use authorization for updated versions of the mRNA vaccines manufactured by Moderna and Pfizer, both of which targeted the KP.2 strain, another Omicron descendant (4). Following FDA’s approval, those vaccines were available to the general public within days.

EMA said in its May 16, 2025, release that marketing authorization holders contact the agency immediately to discuss updates, as should companies still developing new vaccines which may be targeting other strains (1).

ETF Recommends Updating COVID-19 Vaccines to Target New LP.8.1 Variant.

 Press Release. May 16, 2025.
2. EMA, EMA/166854/2025, 

EMA Recommendation to Update the Antigenic Composition of Authorised COVID-19 Vaccines for 2025-2026

US FDA Approves BLA for Novavax's COVID-19 Vaccine.

FDA Approves and Authorizes Updated mRNA COVID-19 Vaccines to Better Protect Against Currently Circulating Variants.

The LP.8.1 variant of the SARS-CoV-2 virus differs from the JN.1 family that previous vaccines had been designed to target, and has surpassed JN.1 as the variant circulating most widely worldwide.

EMA Recommends Update to COVID-19 Vaccines for 2025-26 Campaign

IRVINE, CALIFORNIA - 16 APRIL 2020: Student Center and Visitor Center Building on the campus of the University of California Irvine, UCI. | Image Credit: © Steve Cukrov - stock.adobe.com

The laboratory of University of California, Irvine (UC Irvine) Charlie Dunlop School of Biological Sciences assistant professor Reginald McNulty has published research on the current drug candidates that target the nucleotide-binding oligomerization domain (NOD)-like receptor pyrin domain-containing 3 (NLRP3) inflammasome, a protein complex that plays a central role in causing inflammation in response to infection or tissue damage. The research team has stated that it has discovered the first and only known drugs that bind directly to the pyrin domain (1).

No such drug has yet been approved by FDA despite years of investigation, according to a UC Irvine press release (1). And when left unchecked, the McNulty lab said, NLRP3 can contribute to chronic inflammatory diseases affecting millions globally—everything from arthritis to Alzheimer’s disease to rare genetic disorders—or otherwise damage the body rather than protecting it.

“The NLRP3 inflammasome is a key mediator of the innate immune response to pathogen infection or tissue damage,” McNulty said in the press release (1). “Some humans have mutations in NLRP3 that make them more susceptible to the plethora of disorders and diseases NLRP3 is involved in, ranging from autism and arthritis to liver disease. Yet there are currently no FDA-approved drugs that target it.”

McNulty’s team concluded that the present crop of candidates to treat it, their discovery excluded, falls short in numerous areas, such as poor targeting or unwanted side effects (1).

“A significant challenge is to design an NLRP3 inhibitor that doesn’t bind domains previously shown to have ill-desired off-target effects,” McNulty said (1). “We showed that the pyrin domain has a similar structure to human glycosylase, and that inhibitors targeting one could be repurposed to inhibit the other.”

 describes its design of molecules that resemble damaged pieces of DNA, using oxidized guanine, which were deployed to block NLRP3 at the pyrin domain, preventing it from jump-starting a series of reactions that result in inflammation (1,2).

With an eye on next-generation anti-inflammatory therapies, the lab is now using cryo-electron microscopy to visualize the binding of the molecules to the pyrin domain, with safety and efficacy testing in a clinical setting being the next step (1).

“Progress in clinical trials will be key in advancing our understanding of mitigating inflammasome-based diseases,” McNulty said (1).

Investigation into the NLRP3 inflammasome can be lucrative. In September 2022, 

 for exclusive, worldwide rights to develop and commercialize candidates from Ventus Therapeutics’ portfolio of peripherally restricted NLRP3 inhibitors (3). At that time, Ventus’ lead NLRP3 inhibitor program had been indicated for nonalcoholic steatohepatitis, chronic kidney disease, and several other cardiometabolic conditions. Ventus retained the right to develop NLRP3 inhibitors for certain systemic diseases, including specific inflammatory and respiratory diseases, as well as worldwide rights to the company’s brain-penetrant NLRP3 inhibitor program.

UC Irvine Researchers Explore Promising New Pathway for Treating Inflammatory Diseases.

 Press Release. May 15, 2025.
2. Cabral, J. E.; Wu, A.; Zhou, H.; Pham, M. A.; Lin, S.; and McNulty, R. Targeting the NLRP3 Inflammasome for Inflammatory Disease Therapy. 

Ventus Therapeutics Enters Exclusive Development and License Agreement with Novo Nordisk for NLRP3 Inhibitor Program.

Researchers say they have discovered the first drug mechanism that binds to the pyrin domain to block NLRP3 and prevent a series of reactions resulting in inflammation.

UC Irvine Lab Studies NLRP3 Inflammasome for Inflammatory Disease Treatment Potential

Darmstadt, Germany - November 10, 2021: A building at the headquarters of science and technology company Merck KGaA. | Image Credit: © Anne Czichos - stock.adobe.com

Merck KGaA (Merck), headquartered in Darmstadt, Germany, and Israel-based Peregrine Ventures have announced a collaboration agreement in which Merck will become a strategic partner in one of Peregrine Ventures’ dedicated investment vehicles, called the Incentive Incubator (1,2). Peregrine is a global specialist in investments in transformative medical technologies. Merck, meanwhile, employs approximately 63,000 people in 65 countries.

A press release issued about the launch of the collaboration said that Merck will gain early exposure to certain opportunities for and development of innovation from pharmaceutical and life science startup companies, helping to foster those companies’ future success (1). The partnership has the goal of initiating early-stage collaborations drawn from Merck’s access to market trend insights, opportunity hotspots, and startups in pharma as well as bioconvergence and biotechnology.

Bioconvergence ventures, as defined in the press release, combine biology, engineering, and materials science, having the capability to integrate into Merck’s three global divisions: healthcare, life science, and electronics (1).

Other partners involved in the Incentive Incubator at this point include Bristol-Myers Squibb, Becton Dickinson, Elbit Systems, Tel Aviv University, Ben-Gurion University, and Shaare Zedek Medical Center, according to the press release (1).

“Merck’s scientific, technological, and business strength will greatly support the growth of startups in the incubator,” Lior Shahory, CEO of Incentive Incubator and general partner at Peregrine Ventures, said in the release (1). “Early-stage Israeli startups that catch Merck’s eye and align with its roadmap will receive a major boost in the global market, including access to investments, scientific expertise, clinical trial resources, and strategic assets. We are proud to welcome Merck and believe it will significantly contribute to the incubator’s success and deliver real value to entrepreneurial teams in the coming years.”

Growing partnerships locally and globally

Merck and Peregrine Ventures said they envision the collaboration as a chance for local and international stakeholders to work together on early-stage developments, benefiting from capital investments, access to professional networks, accumulated guidance and strategic guidance, among other factors, with Merck adding that it saw an opportunity to strengthen the company’s local presence (1). Investments, regulatory and business support, and potentially even the planning of early-phase trials were all predicted by the two partners as outcomes of the agreement.

"This partnership reflects our strategic commitment to fostering early-stage innovation and deepening our footprint in Israel’s vibrant pharma and life science ecosystem,” Ezequiel Garfinkel, head of Merck’s Science & Technology office in Israel, said in the release (1). “It builds on Merck’s long-standing scientific and strategic engagement in Israel, where we have been actively supporting research, technological development, and acceleration of local innovation for many years. By working closely with the startups in the Incentive Incubator, we aim to identify transformative technologies that align with our global R&D and business priorities. We are excited to join this dynamic network of partners and contribute to shaping the next generation of healthcare and technology solutions."

1. Merck KGaA. Merck Becomes a Strategic Partner in Peregrine Ventures’ Incentive Incubator. Press Release (received via email). May 15, 2025.
2. Peregrine Ventures. 

Merck will have early-stage opportunities to help startups set up for future success in the pharmaceutical and life sciences fields.

Merck KGaA Becomes Strategic Partner in Peregrine Ventures’ Incentive Incubator

At the 2025 edition of the International Society of Pharmaceutical Engineering (ISPE) Europe Annual Conference, held in London from May 12–14, ISPE marked the 20th anniversary of its Facility of the Year Awards (FOYA) by announcing the 2025 winners in six categories, plus the awarding of two honorable mentions (1).

FOYA was established in 2005, according to a press release from ISPE, to recognize state-of-the-art projects that utilize new and innovative technologies to improve product quality, reduce production costs for high-quality medicines, and showcase advancements in delivery (1). Facility design, construction, and operation are also weighed in the judging process.

Touchlight, in Hampton, UK, was named the winner in the Innovation category, for its development and manufacturing of novel synthetic DNA in an effort to enable the future of genetic medicines (1). Johnson & Johnson Innovative Medicine (formerly Janssen Pharmaceuticals) won in two categories under the umbrella of Operations, for Operational Excellence for its flexible bottle packaging line in Latina, Italy, and Project Execution for the BioCork 3.2 Expansion Project in Ringaskiddy, Ireland.

CSL Behring was given the nod in the Pharma 4.0 category for its “Project Aurora—Facility F Base Fractionation,” in Broadmeadows, Australia (1). 

 in 2017, to describe a drug manufacturing ecosystem that represents the evolution and revolution of drug discovery, manufacturing, and supply chain logistics, helping to ensure that drugs are delivered precisely when needed, and improving overall patient care (2).

Awards in two categories related to social impact were given to Bayer Healthcare’s United States cell therapy launch and manufacturing facility in Berkeley, Calif., for “Unmet Medical Needs,” and Sanofi’s wastewater treatment and recovery plant in Toronto, for “Sustainability Excellence” (1).

Erbe Elektromedizin, in Rangendingen, Germany, and United Therapeutics, in Christiansburg, Va. in the US, garnered honorable mentions.

“The 2025 FOYA category winners exemplify innovation and excellence that continues to elevate the pharmaceutical industry,” Parag Sane, assistant vice-president of Global Capital Projects at Amgen and 2025 FOYA judging chair, said in the press release (1). “Their forward-thinking projects not only advance manufacturing standards and champion sustainability but also reflect a commitment to improving human lives around the world. These winners are setting new benchmarks for what’s possible in delivering both operational excellence and global impact.”

One overall winner will be announced during the 2025 ISPE Annual Meeting & Expo in Charlotte, NC. The 2025 FOYA Banquet and Awards Celebration is to be held on Sunday, Oct. 26, the first night of the show; the annual meeting runs through Oct. 29, 2025 (1).

“Innovation propels our industry forward,” Mike Martin, ISPE president and CEO, said in the press release (1). “ISPE is committed to continuing to champion new ideas that are helping to shape the future of the industry.As the ISPE FOYA program celebrates its 20th anniversary year, we look forward to coming together for the 2025 FOYA Banquet and Awards Celebration to honor this year’s highly accomplished category winners and announce the overall 2025 FOYA winner.”

ISPE Announces 2025 Facility of the Year Awards (FOYA) Category Winners, Celebrating FOYA’s 20th Anniversary Year.

 Press Release. May 12, 2025.
2. Roy, C. 

Validating Pharma 4.0 for Smart Manufacturing.

Six winners and two honorable mentions were revealed at ISPE’s Europe Annual Conference in London.

ISPE Announces Six 2025 FOYA Category Winners

Pharmacy, hands and prescription medicine for customer with paper bag for healthcare, drugs and pharmaceutical. Closeup of a pharmacist or medical worker with person in drugstore for retail service | Image Credit: © Azee Jacobs/peopleimages.com - stock.adobe.com

United States President Donald Trump has signed an executive order designed to reduce prescription drug prices for Americans, bringing the prices in line with those paid by similar nations. Among other stipulations, the order, issued May 12, 2025, directs the Secretary of Health and Human Services (HHS) to establish a mechanism by which US patients can buy drugs directly from manufacturers who sell at a “most favored nation” price, bypassing middlemen (1).

“In case after case, our citizens pay massively higher prices than other nations pay for the same exact pill, from the same factory, effectively subsidizing socialism aboard [abroad] with skyrocketing prices at home,” Trump said in a Fact Sheet provided by the White House (1). “So, we would spend tremendous amounts of money in order to provide inexpensive drugs to another country. And when I say the price is different, you can see some examples where the price is beyond anything—four times, five times different.”

Further directions given in the order include directing the US Trade Representative and Secretary of Commerce to take measures to ensure foreign countries are not unfairly or purposefully undercutting market prices, therefore driving price hikes in the US (1). The Trump administration also plans to communicate price targets to pharmaceutical manufacturers with the goal of securing the best deal for the US, which is both the world’s largest funder and purchaser of prescription drugs.

If drug manufacturers fail to offer most favored nation pricing—within 30 days, 

—the HHS Secretary would be authorized to propose rules that impose such pricing, and take other “aggressive measures” to significantly reduce prescription drug costs and end anticompetitive practices (1,2). What specific measures the Commerce or HHS departments might potentially take under these circumstances were not elaborated upon in the order.

 said that Trump is hoping to cut the prices that US taxpayers pay for prescription drugs by 

 (3,4). According to the Fact Sheet, the executive order builds on actions taken by Trump during his first term in office to reduce domestic drug price disparities but now expands those efforts by including Medicaid in addition to Medicare (1).

However, the order came on the same day that the Republican-led US House of Representatives rolled out a plan to trim $880 billion from Medicaid (5).

Social media reacted swiftly to news of the executive order.

“This is as disastrous as it gets for biopharma,” John Carroll, founder of Endpoints News, 

. “Pure chaos. So, everybody is OK with that? Most favored nation pricing is cyanide to Big Pharma.”

 was BiotechTV founder Brad Loncar: “Pharma [companies] should publicly embrace MFN [most favored nation pricing] generally because getting foreign countries to pay a higher share is obviously a good thing, but more important, pharma is so hated that being viewed as a team player rather than obstructionist needs to be the industry’s new modus operandi.”

According to the White House, the order builds on actions taken in the president’s first term to reduce price disparities domestically.

Featured Content

Digital Transformation in Pharma Manufacturing

Behind the Headlines, Episode 23

ICH Q12 and Post-Approval Changes

Drug Digest July 2025

Bio/Pharma's Tariff Response