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

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

Silver Spring, MD, USA - June 25, 2022: The FDA White Oak Campus, headquarters of the United States Food and Drug Administration, a federal agency of the Department of Health and Human Services (HHS). | Image Credit: © Tada Images - stock.adobe.com

In a June 10, 2025 update, FDA posted seven updated Warning Letters, issued between the beginning of 2025 and the present, to its 

 which stores more than 3400 such letters dating back to 2020 (1). The warnings encompassed various forms of lack of compliance in the agency’s view, including product mislabeling, false or misleading claims on packaging, and shortfalls in current good manufacturing practice (CGMP) standards found during laboratory inspections.

Highlights of five of the seven letters, the companies they were addressed to, and the significant red flags found by FDA are presented below.

American Behavioral Research Institute (ABRI) was inspected in April and May 2024 relative to its capacity as the sponsor of a clinical investigation of an investigational drug, Relaxium Sleep (Protocol ABRI-002, “A Study to Evaluate the Effects of Relaxium in Subjects with Sleep Disorder”) (2). While ABRI did respond to FDA correspondence following this inspection, dated May 24, 2024, FDA said the institute failed to submit an Investigational New Drug application for the conduct of a clinical investigation, such as the one described, subject to Title 21 of the Code of Federal Regulations, part 312. However, FDA acknowledged that there may have been misrepresentation as to whether Protocol ABRI-002 was intended as a drug or a dietary supplement, and has given ABRI more time to reply.

FDA cited CGMP violations in multiple facilities, including inadequate quality systems, lack of equipment cleaning procedures, and missing production records.

Companies marketing unapproved or misbranded products, such as dietary supplements and topical analgesics, were warned for noncompliance with labeling and authorization rules.

Improper or misleading promotional claims, particularly on social media, led to FDA scrutiny and warning letters regarding drug representation and investigational compliance.

FDA charged numerous violations with respect to pain relief creams, ointments, and sprays produced under the brand umbrella “Amish Origins Deep Penetrating” (3). The violations ranged from CGMP standards, to misbranded or unapproved new drugs marketed as external analgesics, to misbranded drugs sold as sunscreen. Among the CGMP violations were failures to conduct testing to verify the identities of certain components of drug products, notably glycerin, and to adequately prepare and perform batch production and control records, as well as quality control. FDA suggested that Amish Origins retain a consultant to address its CGMP issues, and that some of the company’s products may be classified as cosmetics, which fall under different sets of regulations.

Pointe-Claire, Quebec, Canada – May 29, 2025

The warning letter to this Canadian manufacturing facility also deals with CGMP violations, in this instance tied to regulations for finished pharmaceuticals; according to FDA, these products include over-the-counter drug products such as sunscreens and other skin protectants, as well as hand sanitizer (4). In addition to the same recommendations regarding a CGMP consultant and cosmetics designation made to Amish Origins, FDA said Laboratoire Druide’s quality systems were inadequate, and that the Pointe-Claire facility failed to establish, and then follow, appropriate written procedures for the cleaning and maintenance of its equipment.

An Instagram post by Sprout Pharmaceuticals’ CEO, Cindy Eckert, was the impetus for this warning letter. FDA alleges that the post, which was reviewed by the agency’s Office of Prescription Drug Promotion but also received complaints through FDA’s Bad Ad Program, was false or misleading when it came to the effects of the oral tablet flibanserin, marketed as Addyi, within the meaning of the federal Food, Drug, and Cosmetic Act (FD&C Act) (5). Among other claims, the post referred to Addyi as “the sex pill for women”; while it has been indicated for treatment of premenopausal women with acquired, generalized hypoactive sexual desire disorder (HSDD), it is not intended for postmenopausal women, or men, with HSDD, and is not indicated to enhance sexual performance.

Two products offered for purchase through Bio Wellness 444’s website and Instagram account, both varieties of vaginal douches, are available without prescriptions (6). FDA says these are unapproved new drugs that were introduced, or delivered for introduction, into interstate commerce, in violation of multiple sections of the FD&C Act. The products, labeled “Silver Bullet elite douche” and “Silver D elite douche,” contain colloidal silver intended for use in treating vaginal infections. As such, FDA said, the products would be deemed new drugs and require new drug applications, but no FDA-approved applications are currently in effect for those products. Bio Wellness 444 has been asked to respond to FDA with its plan for how to address the violations alleged.

While FDA warning letters generally do not authorize the execution of any definitive actions by the agency, they do specify actions that may be taken if FDA’s suggestions and requests are not adhered to, usually to be communicated by a company within a window of 15 business days of receipt of FDA’s letter. After that period of time, measures cited by FDA may be enacted, or further warnings can be issued.

, last updated June 10, 2025 (accessed June 12, 2025).
2. FDA. 

Warning Letter—American Behavioral Research Institute, LLC.

, last updated June 10, 2025 (accessed June 12, 2025).
3. FDA. 

Warning Letter—Amish Origins Management, LLC.

, last updated June 10, 2025 (accessed June 12, 2025).
4. FDA. 

, last updated June 10, 2025 (accessed June 12, 2025).
5. FDA. 

Warning Letter—Sprout Pharmaceuticals, Inc.

, last updated June 10, 2025 (accessed June 12, 2025).
6. FDA. 

, last updated June 10, 2025 (accessed June 12, 2025).

Articles

The warning letters deal with a range of violations involving items from personal hygienic products to mismarketed analgesics.

FDA Posts Slate of Seven Warning Letters Following Company Inspections

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

 a sterile injectables contract development and manufacturing organization out of Bloomington, Ind. in the United States, has announced a strategic alliance with the Life Science division of 

—which is known as MilliporeSigma in the US and Canada—to assist companies in the biopharmaceutical industry seeking solutions related to antibody-drug conjugates (ADCs) and bioconjugates, including linker and payload manufacturing, drug product formulation development, and fill/finish services (1).

Simtra and Merck KGaA's alliance integrates bioconjugation and fill/finish services, streamlining ADC manufacturing and reducing complexity for clients.

With 70% of ADCs manufactured by CDMOs and over 200 in trials, the global ADC market is forecast to grow from $1.79 billion to $7 billion by 2035.

The partnership assigns dedicated program managers to improve coordination, aiming to accelerate timelines and uphold high quality standards.

As a press release from Simtra explained, the manufacture of an ADC is a complex process that involves combining a monoclonal antibody, cytotoxic payload, and linker into one stable and effective therapeutic (1). The development and contract manufacturing value chain created by this new partnership ties bulk drug substance made by MilliporeSigma together with Simtra’s drug product fill/finish capabilities. Dedicated product managers at each of the partners’ sites will be available to expedite customer needs and requests.

“By bringing together two experts in the ADC value chain, biopharmaceutical companies should benefit from shorter timelines and less complexity, allowing them to focus their efforts on drug discovery,” said 

 CEO of Simtra BioPharma Solutions, in the press release (1). “Harmonizing our processes with designated program management from start to finish will give customers greater confidence that their product will be delivered on time and to the highest quality standards—ultimately accelerating the delivery of life-changing therapies to patients who need them most.”

According to the press release, the ADC pipeline has been growing in recent years, driven by early successes against diseases such as human epidermal growth factor receptor 2 (HER2)-positive breast cancer; currently, 70% of these drugs are manufactured by CDMOs (1). A study by Roots Analysis values the ADC manufacturing market at $1.79 billion right now, but projects it to reach $7 billion by 2035 given a compound annual growth rate of 13%.

“Patients can’t wait. With over 200 new ADCs in active clinical trials, it is critical that we work to speed up the manufacturing process, reduce the risk of knowledge or time-loss during handoffs, and enable our clients to advance their programs,” said 

 head of Life Science Services, Life Science business of Merck KGaA, in the release (1). “Connecting the bioconjugation and fill/finish steps will be a value-add for our clients, ultimately meeting their ambitious timelines and allowing the broadest number of patients to benefit in the fastest and safest possible way.”

Just one day before the June 11 partnership announcement, Merck KGaA said that China’s Center for Drug Evaluation of its National Medical Products Association (NMPA) had 

 for marketing authorization for pimicotinib, a novel, orally administered small-molecule inhibitor of the colony stimulating factor 1 receptor, or CSF-1R, for treatment of adult patients with tenosynovial giant cell tumors (TGCTs) requiring systemic treatment (2).

And in March 2025, Simtra head of Development Services 

® Group at the Drug, Chemical & Associated Technologies Association 

 about the key challenges in aseptic processing for sterile injectables. 

Simtra BioPharma Solutions and MilliporeSigma Announce Strategic Alliance for Antibody Drug Conjugates Drug Substance and Drug Product Manufacturing Services.

 Press Release. June 11, 2025.
2. Merck KGaA. 

China’s Center for Drug Evaluation Accepts Merck KGaA, Darmstadt, Germany’s Application for Marketing Authorization of Pimicotinib for Treatment of Tenosynovial Giant Cell Tumor.

 Press Release. June 10, 2025.
3. Mirasol, F. 

DCAT Week 2025: Maintaining Aseptic Integrity and Stability in Sterile Injectable Manufacturing.

The agreement has been designed to offer a turnkey service to companies seeking faster time to market.

MilliporeSigma and Simtra Launch Five-Year Agreement for Drug Substance, Drug Manufacturing Services

Neurons, Active nerve cells, Conceptual illustration of neuronal cells with bright connecting nodes in the abstract dark space, illustration in high resolution, 3D rendering | Image Credit: © Eric - stock.adobe.com

, has raised €11.25 million (US$12.9 million) in an oversubscribed seed round to advance a novel therapeutic approach targeting treatment-resistant depression (1). The financing round was co-led by 

, and the French Tech Seed Fund managed by 

, on behalf of the French government, under the 

 initiative. Additional participants included 

The company is developing a first-in-class neuroplastogen based on pioneering academic research that aims to restore neuroplasticity without the drawbacks of current treatments like psychedelics or ketamine derivatives (1). This effort targets the intracellular protein Elk1, a central modulator of brain circuits involved in reward and emotional regulation. The approach stems directly from translational work conducted at the 

Linking Neuroscience to Therapeutic Development

·European biotech startup Elkedonia secures €11.25M (US$12.9M) in seed funding, integrating Sorbonne University research with Argobio’s drug development expertise.

·Novel Elk1-targeting antidepressants aim to restore neuroplasticity without sedation or hallucinations, addressing treatment-resistant depression.

·Elkedonia develops first-in-class, non-addictive neuroplastogens that avoid synaptic targets, offering a safer alternative to ketamine-based therapies.

Elkedonia was founded by Argobio, a biotech venture studio focused on translational science, and is built on research led by 

, director of research at CNRS. Her studies have identified Elk1 as a key regulatory protein in brain plasticity, disruptions of which are implicated in depression, PTSD, addiction, and anhedonia (2).

“Trauma, chronic stress, substance abuse and other factors can contribute to major depressive disorders and anhedonia (the inability to experience pleasure), driven by an impaired neuroplasticity,” the company stated (1). “This disruption in the brain’s ability to adapt leads to a breakdown in its normal functioning.”

Elkedonia’s therapeutic platform seeks to inhibit Elk1, restoring neural adaptability without inducing hallucinations, sedation, or dependence. The company points to encouraging findings from preclinical and early clinical research, suggesting that targeting Elk1 may offer rapid antidepressant effects, in contrast to the delayed onset of conventional treatments.

Proceeds from the seed financing will support the optimization of small-molecule Elk1 inhibitors and the development of companion biomarkers (1). These efforts are central to Elkedonia’s strategy to deliver precision medicine in mental health, matching therapies to patient subtypes and tracking treatment response with objective measures.

“I am thrilled to welcome our founding seed investors and thank my colleagues at Argobio for their support in creating Elkedonia,” said 

, CEO and co-founder of Elkedonia (1). “The novelty of our neuroplastogen approach offers a strong value proposition supported by groundbreaking neuroscience research. We are positioned to make a significant impact on the treatment of major depressive disorders, bringing relief to all patients, including young people, adolescents and the elderly. In addition, our development of biomarkers will allow us to pursue a precision medicine for patient selection and treatment efficacy monitoring.”

The company’s focus on treatment-resistant depression addresses a growing and underserved population. Globally, major depressive disorder affects approximately 280 million people, with many patients failing to respond to existing therapies (3). This public health burden is particularly severe among adolescents and young adults, where depression-related suicide is a leading cause of death.

“Major depressive disorder is a large market of 300 million people globally that remains poorly addressed, with roughly one third of patients resistant to currently available treatments,” said 

, chairman of the management board of Argobio and managing partner at Kurma Partners. “Elkedonia serves as a first proof-of-concept for Argobio where we are extremely proud to have identified this opportunity to benefit human health and to have provided industry competencies to accelerate and de-risk product development.”

, investment director at WE Life Sciences, said, “Investing in Elkedonia demonstrates WE Life Sciences' commitment to advancing the best cutting-edge innovations through collaboration with top-tier European life sciences investors. We are proud to support the realization of this promising innovation from Argobio, while fostering its growth in Wallonia, Belgium, a region increasingly recognized for its excellence in health and biotechnology.”

“Bpifrance, as a founding investor of Argobio, is delighted to support the seed round of this promising new venture,” said 

, investment director Life Sciences at Bpifrance. “We are thrilled to develop the foundational research that comes from CNRS at Sorbonne University. This investment underscores our commitment to advancing mental health solutions, which is currently a priority for France.”

Elkedonia Closes EUR 11 Million Seed Round to Advance Neuroplastogens as Novel Therapeutics for Depression and Neuropsychiatric Disorders

Elkedonia will develop first-in-class Elk1 inhibitors for treatment-resistant depression, targeting neuroplasticity without psychedelic side effects.

Elkedonia Raises €11.25M Seed Round to Advance Neuroplastogen Therapy for Depression

Logo of Merck KgA, Merck is a German multinational science and technology company. | Image Credit: © Robert - stock.adobe.com

 announced in a press release on June 10, 2025 that the Center for Drug Evaluation (CDE) of the China National Medical Products Association (NMPA) has accepted the company’s application for marketing authorization for pimicotinib, a novel, orally administered small-molecule inhibitor of the colony stimulating factor 1 receptor, or CSF-1R (1). CDE’s approval is for the treatment of adult patients with tenosynovial giant cell tumors (TGCTs) requiring systemic treatment.

Pimicotinib, which is in development by Abbisko Therapeutics, was granted priority review by CDE in May 2025, as well as breakthrough therapy designation by NMPA (1).

China’s CDE has accepted Merck KGaA’s application for pimicotinib, a CSF-1R inhibitor developed for TGCT, following its priority review designation.

Phase III trial data support pimicotinib’s once-daily use to reduce joint tumors and improve mobility and pain, addressing a major unmet need in TGCT care.

Merck KGaA’s filing follows FDA approval of a competing oral TGCT therapy, vimseltinib, and aligns with its broader innovation strategy via Peregrine Ventures.

TGCT is described as a locally aggressive, often reoccurring tumor of the joints that can cause swelling, pain, stiffness, and limited mobility of the affected joints, potentially resulting in high morbidity in recurrent cases or if left untreated (1). Merck KGaA’s application—and the resultant actions of NMPA and CDE—was based on results from the first part of a global Phase III trial (MANEUVER) that demonstrated superiority in a once-daily administration of pimicotinib versus placebo at 25 weeks.

“With the acceptance of our application for pimicotinib and the initiation of the priority review, we aim to offer patients in China the first approved systemic therapy for TGCT, addressing a tremendous unmet need in this country,” said 

 head of China and International, Healthcare business of Merck KGaA, in the press release (1). “Pimicotinib has demonstrated the ability to not only shrink tumors that affect their joints but also improve outcomes like mobility, pain and stiffness, highlighting its potential to be a best-in-class treatment for TGCT. In parallel, we are working to file a New Drug Application to [FDA], with additional filings planned in other markets.”

FDA has already granted full approval to at least one treatment for TGCT. In February 2025, 

’s vimseltinib, a kinase inhibitor (brand name Romvimza), in adult patients with TGCT for which surgical resection could potentially cause worsening functional limitation or severe morbidity (2). This treatment comes in oral, capsule form and had previously received fast track designation and priority review from FDA.

As for Merck KGaA, the news of China’s approval of pimicotinib comes on the heels of the German company’s announcement 

 that it would partner with Israel-based Peregrine Ventures, becoming a strategic partner in one of Peregrine’s dedicated investment vehicles, the Incentive Incubator (3). Under this agreement, 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.

 Press Release. June 10, 2025.
2. Ono Pharmaceutical. 

US FDA Grants Full Approval of Deciphera’s Romvimza (vimseltinib) for the Treatment of Symptomatic Tenosynovial Giant Cell Tumor (TGCT).

 Press Release. Feb. 14, 2025.
3. Merck KGaA. Merck Becomes a Strategic Partner in Peregrine Ventures’ Incentive Incubator. Press Release (received via email). May 15, 2025.

NMPA’s Center for Drug Evaluation accepted Merck KGaA’s application for marketing authorization of pimicotinib as a Class 1 innovative drug for adult patients with TGCTs that require systemic treatment.

China’s NMPA Approves Merck KGaA Treatment for Tenosynovial Giant Cell Tumor

Abstract vertical illustration of targeting cancer cell made of glowing neon particles. Blue geometric background depicting cancer cell screening and disease treatment medical concept. Digital art | Image Credit: © artacet - stock.adobe.com

The biopharmaceutical industry has seen exciting advancements in the past decade, with cell and gene therapies, personalized medicine, messenger RNA (mRNA) vaccines, and new treatments for autoimmune diseases. The global biopharma industry is expected to increase at a compound annual growth rate of 8.97% from 2025 to 2030; this growth is suspected to result from a demand for targeted therapies, new alliances, and increased innovation (1). The oncology field is also making an impact, with immunotherapies, cell therapies, and gene therapies advancing quickly (2).

As more and more new modalities reach regulatory approval and enter the market, David Kuntin, CEO and cofounder of Mesenbio, sees the biopharmaceutical market becoming more diverse with a number of highly targeted treatments, such as antibody–drug conjugates (ADCs) and next-generation antibody therapies (e.g., bispecific antibodies) being developed. “Cell and gene therapies (CGTs) are also advancing, though interest in early-stage assets is waning; we can expect more gene therapies for inherited diseases and CAR [chimeric antigen receptors]-T or other cell-based therapies expanding beyond rare cancers into more common conditions, if pricing is something that can be addressed,” Kuntin says. “Messenger RNA-based products will certainly move beyond traditional vaccines—for instance, there is significant effort in mRNA therapeutics for cancer vaccines and protein replacement therapies. Crucially, these modalities won’t exist in isolation. Many will become established alongside traditional biologics. For example, an autoimmune condition might in the future have treatment options ranging from monoclonal antibodies to an mRNA therapy or even a cell-derived product.”

Other approaches, such as extracellular vesicle (EV) therapeutics, are in development and are a promising new class of regenerative or delivery agents, according to Kuntin. “Overall, the future commercial landscape will not be dominated by one modality but characterized by a wide array of therapeutic approaches, each tailored to different medical needs. This rich diversity is exciting and marks a shift toward more personalized and precise medicine; though, it also means our industry must support a broader range of manufacturing and regulatory strategies to bring these treatments to patients.”

“I’m excited to see multispecific antibody- like biologics synergistically block multiple cancer cell survival pathways or help induce immune responses, such as cell-killing we are currently seeing with cell therapies,” Christian Cobaugh, chief scientific officer and founder, Vernal Biosciences, says. “Multispecifics are also offering more favorable target product profiles and potentially better outcomes in oncology.”

Lipid nanoparticles (LNPs) and mRNA have become “derisked,” according to Cobaugh, and the clinical data from LNP-mRNA for gene editing and oncology are impressive. “Most of the 

 gene-editing programs that we see from companies like Intellia, Beam, and Verve rely on LNP-mRNA (plus guide RNA) and can be dosed to effect,” Cobaugh says. “Further, the therapeutic cancer vaccines are another great way to leverage the speed to clinic that mRNA manufacturing offers.”

While a significant number of illnesses will still be addressed by small-molecule drugs, according to Miguel Forte, president, ISCT, board member, ARM, and CEO Kiji Therapeutics, biologics offer the ability to target certain diseases with specificity and mechanism of action. “If a patient has a genetic disease, it is likely that the best approach would be gene therapy,” Forte says. “I would expect cell, and gene-edited cells to play a major role in oncology—as is already the case—and also in autoimmune diseases, in parallel and possible combination with biologics.”

Diversification in the bio/pharmaceutical industry that provides a mix of traditional small-molecule drugs, biologics, and vaccines may drive more targeted therapies and intensify market competition, according to Max Baumann, cofounder, Treehill Partners.

“As we observed in an industry analysis we performed comprising 1200 clinical programs, only a minority of companies currently optimize their development for commercial success,” Baumann says. “Combined with increased segmentation of clinical end markets, this has a significant impact on the return that potential pharmaceutical drug development offers for investors, and absent a clear investment thesis, not all the products we currently see in development will be successful in securing funding to market entry and break-even.”

Innovation often comes at a cost, however, and this must be balanced. While the industry benefits from the cycle of innovation, formulation, and production, says Oury Chetboun, CEO, Seekyo Therapeutics, new drugs also must be cost effective for patients and to develop and produce. “You may have a fantastic drug in the lab which costs a massive amount to be scaled up, but if you can’t get that into a large-scale production or at a reasonable cost, then you can’t do anything,” Chetboun cautions.

Each patient has different needs, and each disease is different; therefore, a mix of formulation and drug delivery methods is needed for optimal treatment, according to Chetboun. Patient adherence is also important, however. Complying with a treatment plan is easier for patients when fewer doses or administrations are required. Side effects may also impact patient adherence, making multiple delivery route options more attractive, suggests Chetboun.

Creative formulation and drug delivery strategies are necessary for new modalities to reach patients, Kuntin says, and he sees less invasive administration as a major trend. Nasal and inhaled formulations for vaccines and antibody therapies might advance, which could target drugs to mucosal tissues. “There is also significant interest in achieving oral delivery of biologics; traditionally, proteins and nucleic acids get digested in the gut, but new techniques like protective coatings, nanoparticle carriers, or special capsule devices are showing promise in allowing antibodies or peptide therapies to be taken as a pill,” Kuntin explains. “While not every biologic will become a tablet, even a few successes in oral biologic delivery would be paradigm-changing for chronic treatments.”

 cell therapies, mostly CAR-Ts and HSCs [host cell proteins], have validated some key mechanisms of action including gene editing. But those therapies are more akin to procedures, and highly invasive and risky ones at that, than they are products. I’m looking forward to advancements in non-viral genetic delivery technologies, such as LNPs and PNPs that can de-target the liver and retarget blood and immune cells to transform these procedures into products that can be dosed to effect.”

Stability and distribution of new modalities is also critical, says Kuntin, with development ongoing to formulate drugs that remain stable at refrigerator temperature or room temperature, therefore removing the need for ultra-cold storage.

“We’re likely to see more use of advanced carriers like lipid nanoparticles, which have already proven their worth in delivering mRNA vaccines and could be refined for other drugs,” Kuntin says. “Looking further ahead, there is exploration of novel delivery vehicles—for example, utilizing extracellular vesicles as a natural means to carry therapeutic molecules to target tissues. EVs in particular could signal a new paradigm in biologic delivery, with production approaches able to be intrinsically integrated into the EV production process.”

Advancements in technology, such as artificial intelligence (AI), machine learning (ML), and digitization, have played a part in the development of new and innovative modalities. The ability to work with large amounts of data and manage processes digitally may accelerate processes and reduce cost, according to Forte. “Importantly though are the opportunities to accelerate development by providing faster data analysis, scenario elaboration, and digital comparison approaches (digital twins) that will overall improve the process and speed of development,” he stresses. AI can also facilitate the exploration of different models and conditions by using digital twins, adds Forte. “In this context, the conditions and requirements for scaling up can be modeled and analyzed, accelerating the process overall,” he notes. 

“In biologics design, for example, machine learning algorithms can suggest modifications to an antibody or protein to improve its efficacy and stability, or even help design novel therapeutic proteins from scratch,” Kuntin says. “This means we can generate and evaluate a wider range of therapeutic options 

 before moving to lab experiments, saving time and resources. I’ve seen cases where an AI model predicts how a change in a molecule’s structure might affect its function or how a particular patient subgroup might respond to a therapy—insights that would have taken much longer to glean through trial and error in the lab.”

According to Cobaugh, AI is also helping design antisense oligonucleotide therapies and locating gene-editing sites. He also sees AI playing a role in predicting and designing antigens for personalized disease treatments.

“We’ve also seen how tools like Alpha-Fold3 are using machine learning to accurately predict the structures of most biomolecules, including proteins and nucleic acids, reducing what used to be a dissertation’s amount of work to a day or two of computing time. Labs, such as the David Baker lab, are now able to take a de novo approach to design completely synthetic proteins with specific functions in mind,” Cobaugh says.

The development pathway can be streamlined by digitization, using advanced data analytics and automation in preclinical research, says Kuntin. “AI-driven analysis helps interpret complex datasets, such as finding patterns in thousands of data points from early safety studies or optimizing formulation conditions for a new modality. In clinical development, digital tools assist in trial design and patient selection—for instance, algorithms can match eligible patients to clinical trial protocols more effectively or predict outcomes using real-world data, which helps in designing smarter trials. Overall, these technologies are accelerating development timelines and helping us make more informed decisions at each step. The end result is a higher probability that truly effective therapies make it through to approval by focusing our efforts on the most promising candidates and approaches.”

Digital technologies can “create a smoother bridge” across discovery and manufacturing, according to Kuntin, with critical factors and ideal settings for production identified faster with fewer errors. AI/ML can then play a bigger role in commercial manufacturing by monitoring production in real time.

Baumann adds that AI/ML can bridge the gap by facilitating tech transfer through digitally capturing and standardizing process knowledge. “This integrated approach supports faster, more reliable scale-up and reduces the risk of costly manufacturing setbacks,” he says. 

“Modern biologics facilities often use advanced sensors and control software that adjust process conditions on the fly, guided by AI models that have ‘learned’ from the development data,” Kuntin says. “This means we maintain consistency between a small lab batch and a large manufacturing batch. I’ve found that even for complex new modalities—like extracellular vesicles—having a strong digital data backbone from R&D through to production makes tech transfer much more seamless. It reduces the risk for surprises during scale-up and helps get therapies from the bench to the patient more reliably and efficiently. I would say, however, that caution must be exercised whenever these tools are used, and to avoid over-reliance on these, as there is a tendency to treat AI solutions as ‘black boxes’—a totally obscured machine with the ability to pump out data—without truly critically evaluating the outputs. There are clear and important distinctions between generative AI approaches and those that use existing data and draw connections and contextualize these into meaningful outputs, and that is a crucial thing to bear in mind when using AI in drug development, especially when interrogating large data sets.”

“There is significant value to be generated in two main areas,” says Bauman, “namely, ‘doing the right thing/development strategy—where AI/ML can be instrumental in identifying and supporting the best strategic direction and avoid mis-steps that may put an entire company on an inferior path—and ‘doing things the right way’/execution of key tasks along the development journey, where error rates can be reduced, equations be better solved, and ultimately better development outcomes be created through aggregating thousands of slightly improved puzzle pieces,” says Baumann.

New biopharma modalities, along with new technology, have given patients new opportunities and provide value to society, according to Forte, and they could lead to advancements in other areas and product types.

Kuntin sees some new modalities becoming established treatment options in the next decade. “Not every new modality will succeed commercially—each must prove not only clinical benefit but also that it can be delivered to patients reliably—but I am optimistic that a good number of them will become staples in the medical toolkit,” says Kuntin. “In my view, we’re seeing a co-evolution: as therapeutic modalities mature, they push the industry to develop new production capabilities, and as manufacturing technology leaps forward, it expands the realm of what therapies can successfully reach patients. The end result is that patients should benefit from a broader range of treatment options, and these will hopefully become more affordable and accessible as manufacturing efficiencies improve. The future biopharma landscape will be defined by this synergy between groundbreaking science and equally innovative manufacturing.”

“The interplay between modality innovation and manufacturing evolution will define the next decade of biopharma, driving both operational excellence and improved patient outcomes. We remain cautious about the current geopolitical environment inflicting fundamental changes to our industry, which range from choice of production location to pricing of the end product and with that affect the commercial attractiveness/viability of geographical markets across the globe,” Baumann says.

1. Grand View Research. Biopharmaceutical Market Size and Trends. 

2. Haigney, S. Emerging Treatments for Cancer. 

BioPharm International. Next-Generation Biotherapeutics

The quickly evolving development of biopharmaceuticals is impacting innovation in the industry overall.

Advancements in Biopharma Spur Innovation

Washington, DC, USA - June 24, 2022: The symbol of the U.S. Department of Health and Human Services is seen at the entrance to its headquarters in the Hubert H. Humphrey Building in Washington, DC | Image Credit: © Tada Images - stock.adobe.com.

On June 9, 2025, Secretary of Health and Human Services Robert F. Kennedy Jr. announced the dismissal of all 17 members of the Advisory Committee on Immunization Practices (ACIP), a Centers of Disease Control and Prevention (CDC)-affiliated body responsible for guiding vaccine policy in the United States (1). The decision was communicated through a Department of Health and Human Services (HHS) press release. While the Secretary holds the legal authority to appoint and remove committee members, the wholesale dismissal of the panel is an unprecedented step and has prompted concern across public health, scientific, and biopharmaceutical sectors. The news follows 

 of a restructuring to include the reduction of HHS workforce by 10,000 full-time employees.

·ACIP overhaul disrupts vaccine approval timelines, posing risks to clinical trial planning, FDA readiness, and seasonal launch strategies.

·Regulatory uncertainty from ACIP shake-up may delay vaccine reimbursement and market access, impacting commercial rollout and payer engagement.

·ACIP restructuring threatens US alignment with global vaccine policy, potentially undermining international regulatory harmonization and partnerships.

ACIP plays a foundational role in shaping immunization policy in the US. It provides evidence-based recommendations on the use of vaccines for children and adults, informs decisions about insurance coverage, and helps determine eligibility for publicly funded programs such as Vaccines for Children (VFC). In justifying the dismissals, Secretary Kennedy argued that a "clean sweep" was needed to "reestablish public confidence in vaccine science," asserting that future members would prioritize “public health and evidence-based medicine,” and avoid serving as a “rubber stamp for industry profit-taking agendas” (1).

Medical and scientific groups have pushed back against this characterization. The American Medical Association (AMA) stated that the decision “undermines trust and upends a transparent process that has saved countless lives,” while the Infectious Diseases Society of America called Kennedy’s accusations of bias “completely unfounded” (2,3). ACIP members are required to publicly disclose potential conflicts of interest and recuse themselves from votes where applicable. A government report previously cited by Kennedy to support his claims of systemic conflicts found no such widespread issues (4).

Industry groups have also raised serious concerns. The Biotechnology Innovation Organization (BIO), which represents biotechnology firms, including vaccine developers and manufacturers, criticized both the process and the timing of the dismissals (5). BIO President and CEO 

 emphasized that ACIP plays a legally mandated role not only in ensuring scientific rigor but also in determining how the US population accesses immunizations. Crowley noted that the removal of the committee members less than three weeks before ACIP’s next scheduled meeting risks derailing ongoing deliberations on critical vaccines, including those for respiratory syncytial virus (RSV), influenza, and human papillomavirus.

In particular, BIO highlighted that ACIP’s reviews involve months of preparation, including technical assessments by specialized working groups, and that a sudden overhaul disrupts this process. Crowley warned that such disruption could impair the committee’s ability to deliver timely, well-informed guidance—potentially putting public health at risk.

Impact on vaccine development and manufacturing

For vaccine developers and manufacturers of, the overhaul introduces short- and long-term uncertainty in the regulatory landscape. ACIP recommendations significantly influence vaccine uptake by guiding clinical practice, shaping insurance coverage decisions, and signaling public health priorities. An abrupt change in committee composition may delay decisions on new vaccines or introduce new criteria for evaluating safety and efficacy. This could directly affect investment planning, clinical trial design, and launch timelines, particularly for candidates in late-stage development or approaching FDA review.

Market access strategies are also likely to be affected. Reimbursement pathways for newly recommended vaccines depend in part on ACIP endorsement. Delays or inconsistencies in the committee’s operations could hinder payer engagement and commercial rollout, especially for seasonal or time-sensitive products. Manufacturers currently preparing for launches of RSV or updated influenza vaccines in the upcoming cycle may need to adjust expectations depending on how quickly the new committee is formed and operational.

Beyond immediate regulatory impacts, the restructuring raises broader strategic questions for vaccine research and development in the US. The longstanding credibility of ACIP has supported productive collaboration between government, academia, and industry, while also helping to align US policy with global regulatory norms. A perceived politicization of ACIP’s role or a reduction in scientific transparency may strain public–private partnerships and deter investment in vaccine-preventable disease areas.

In addition, international regulatory bodies often look to ACIP as a model for evidence-based immunization policy. If future decisions by the reconstituted committee are seen as politically motivated or inconsistent with global standards, this could affect regulatory harmonization and market access strategies for multinational firms.

The removal of all voting members of the ACIP introduces a period of heightened uncertainty for the pharmaceutical industry, particularly for vaccine developers, manufacturers, and regulatory affairs professionals. While the long-term implications will depend on who is appointed to the committee and how it operates going forward, the abruptness and scale of the change have disrupted a long-standing scientific and policy framework. Continued attention will be required to assess how future ACIP deliberations evolve and what this means for US immunization policy and pharmaceutical innovation.



Department of Health and Human Services. 

HHS Takes Bold Step to Restore Public Trust in Vaccines by Reconstituting ACIP

AMA Statement on Advisory Committee on Immunization Practices

Statement on the Advisory Committee on Immunization Practices. Press Release

RFK Jr. Boots All Members of the CDC’s Vaccine Advisory Committee

BIO Statement on HHS Dismissal of CDC Advisory Committee Members

The dismissal of all 17 members of the Advisory Committee on Immunization Practices has far-reaching impacts for the industry and beyond.

Implications of RFK Jr.’s CDC Vaccine Committee Overhaul for the Pharmaceutical Industry

Orlando, Florida, USA - February 8, 2020: Hesperos headquarters in Orlando, Florida, USA. Hesperos is a biotechnology company that develops human-on-a-chip systems. | Image Credit: © JHVEPhoto - stock.adobe.com

 a biotechnology company with a focus on developing therapies for neurological disorders that are difficult to treat, and 

 an industry leader in organ-on-a-chip technology, have announced a strategic agreement for the purpose of accelerating preclinical development of Psilera’s lead compound that targets frontotemporal dementia (1).

Psilera and Hesperos have partnered to accelerate preclinical development of PSIL-006, a neuroplastogen candidate targeting frontotemporal dementia.

Hesperos’ organ-on-a-chip system uses patient-derived stem cells to model disease physiology, supporting data generation for FDA submissions.

FDA policy shifts promoting human-based lab models highlight organ-on-a-chip as a viable alternative to animal testing, aiding faster, safer drug development.

The compound, known as PSIL-006, integrates next-generation neuroplastogen, and the partnership with Hesperos will leverage that company’s advanced biological platform to develop a targeted treatment for unmet medical needs relating to frontotemporal dementia, a progressive neurological disorder for which treatment options are currently limited (1).

“Our collaboration with Hesperos represents a significant step forward in our mission to develop new treatments for neurodegenerative diseases,” said Chris Witowski, co-founder and CEO of Psilera, in a press release (1). “Utilizing their cutting-edge platform allows us to gain deeper insights into PSIL-006’s mechanism of action as we’re nearing first-in-human trials.”

The Hesperos platform, which integrates patient-derived induced pluripotent stem cells into interconnected, multi-organ systems, has been recognized for its ability to replicate human physiological responses, according to Hesperos, and has successfully supported multiple FDA Investigational New Drug and Orphan Drug Designation applications (1).

“We are excited to partner with Psilera as they advance next-generation neuroplastogens to potentially treat a range of hard-to-treat diseases,” said 

 PhD, co-founder and chief scientist at Hesperos, in the press release (1). “Our platform offers a unique opportunity to assess the compound’s effects on neural tissues derived from patients with neurodegeneration, potentially accelerating the development of a much-needed therapy utilizing clinically relevant functional readouts.”

Organ-on-a-chip technology was at the center of another partnership, 

 (2). Under that agreement, Pharmaron is tasked with validating CN Bio’s PhysioMimix technology across existing applications, while integrating organ-on-a-chip technologies into its R&D platform. Also in the deal, the partners agreed to explore potential new applications that would address unmet needs in drug discovery and development (3).

 as one of the human-based lab models that FDA would be shifting to, specifically those systems that mimic the heart and liver, in its objective to reduce and eventually replace animal testing in monoclonal antibody safety evaluations (4).

“For patients, it means a more efficient pipeline for novel treatments,” FDA Commissioner Marty Makary, PhD, said at the time (4). “It also means an added margin of safety, since human-based test systems may better predict real-world outcomes. For animal welfare, it represents a major step toward ending the use of laboratory animals in drug testing. Thousands of animals, including dogs and primates, could eventually be spared each year as these new methods take root.”

In the press release announcing the agreement between Hesperos and Psilera, that policy adjustment by FDA was cited as a means of promoting the benefits of organ-on-a-chip—for not only reducing the reliance on animal testing, but also enabling precise evaluation of drug efficacy and safety that has the potential to expedite clinic-ready datasets (1).

Psilera Collaborates with Drug Development Leader Hesperos to Advance Preclinical Modeling of PSIL-006 for Frontotemporal Dementia.

 Press Release. June 9, 2025.
2. CN Bio. 

CN Bio and Pharmaron Establish Long-Term Strategic Partnership to Develop OOC Technologies on a Global R&D Platform.

 Press Release. April 24, 2025.
3. Mirasol, F. 

CN Bio and Pharmaron Partner to Develop OOC Technologies for Global R&D.

FDA Announces Plan to Phase Out Animal Testing Requirement for Monoclonal Antibodies and Other Drugs.

The partnership leverages the Hesperos organ-on-a-chip platform in the preclinical development of Psilera’s lead compound targeting the progressive neurological disorder for which treatment options are few.

Hesperos, Psilera Agree to Accelerate Organ-on-a-Chip Treatment for Frontotemporal Dementia

Cynthia A. Challener is a contributing editor to Pharmaceutical Technology.

Efficient and effective delivery of complex and challenging drug substances is an ongoing issue facing developers of modern medicines. Delivery systems must overcome poor solubility and bioavailability while achieving targeted delivery to achieve maximum efficacy with lower doses while also minimizing unwanted side effects. Nanoscale technology is proving to be an attractive solution on all accounts, particularly for realizing targeted delivery into specific types of cells and across biological barriers.

Many of the unique properties of nanoscale delivery systems can be attributed to their small size (< 100 nanometers). At this scale, the surface area/volume ratio is high, leading to unique physical, chemical, and biologic properties. The ability to tune the surface properties of nanomaterials through functionalization further increases their utility for drug delivery, particularly with respect to their targeting capabilities. Nanoscale delivery systems can not only increase the bioavailability of poorly water-soluble drugs; they can also protect sensitive APIs from degradation and, when appropriately designed, remain stable in the bloodstream, allowing long-term sustained release and more consistent pharmacokinetics (1). The greater cellular uptake of nanoscale delivery systems also contributes to improved efficacy and reduced side effects.

Another important advantage of nanoscale delivery systems is the ability to deliver two or more APIs simultane

Nanoscale co-delivery systemsoffer improved performance for combination therapies because they deliver the different drug substances in a homogeneous manner (2). Ideal nanoscale carriers are stable, small, but able to support sufficient drug loading of both hydrophobic and hydrophilic APIs; protect the loaded drug from degradation; are suitably functionalized to exhibit a high binding affinity to target cells/ tissues; and have good biocompatibility.

Many different types of nanoscale delivery systems (e.g., lipidic, polymeric, inorganic, biomimetic) provide opportunities for designing solutions with high specificity for the particular API, disease target, and route of administration. In fact, they support the loading of most types of drug substances, including small molecules, proteins, nucleic acids, and diagnostic agents (1). They can also be customized in support of personalized medicines, and some have been developed that are capable of exhibiting both therapeutic and diagnostic activities (theranostics).

As more information is gained about the benefits of nanoscale delivery systems, researchers are exploring the applicability of a growing variety of nanoscale materials for this purpose (1–3). Nanoparticles have attracted particular attention, including those based on lipids, polymers (natural and synthetic), lipid-polymer hybrids, dendrimers, inorganic materials (metals, magnetic compounds, carbon nanotubes, silica-based systems, etc.). Other nanocarriers include nanocrystals; DNA-based systems; extracellular vesicles such as exosomes, microvesicles, apoptotic bodies, and phagosomes; nanoemulsions; and micelles.

Like any drug delivery technology, nanoscale systems are not perfect solutions and, depending on the particular nanotechnology, have different sets of limitations (1). Some of the nanomaterials used for drug delivery can, if the systems are not designed appropriately, pose toxicity and biocompatibility issues. One example is metallic nanoparticles if they accumulate in the body.

Similarly, while some nanoscale delivery systems can exhibit prolonged lifetimes in the bloodstream, others have short circulation times or can be viewed as foreign materials by the immune system and rapidly cleared. Some nanomaterials may also exhibit unexpected/unpredictable interactions with biological systems that lead to detrimental effects. These uncertainties and complexity of nanoscale drug delivery systems often lead to regulatory challenges.

The development of robust, predictable large-scale manufacturing processes can be a significant hurdle for some nanomaterials/nanoscale delivery systems. In addition, stability during storage can be an issue for some nanoscale systems, requiring careful formulation development. All these challenges tend to lead to high costs for therapeutics and vaccines leveraging nanoscale drug delivery systems.

Highly targeted delivery with nanoscale systems can be achieved using passive or active mechanisms. Passive delivery involves accumulation of the nanoscale system in the targeted cell/ tissue through enhanced permeability and retention (EPR), which is dictated by the shape, size, charge, and other surface properties of the unfunctionalized nanomaterial (4). This approach does not, however, completely reduce delivery to healthy cells, which can result in off-target effects.

Active targeting, meanwhile, is realized through functionalization of the surface of the nanocarrier system to both increase cellular uptake and ensure delivery to specific cells/tissues (4). Ligands are attached to the surfaces of the nanocarriers that have high binding affinity to receptors specific to the target cells. These ligands can include a range of compounds, including antibodies, aptamers, peptides, small molecules, and biomimetic materials such as cell membranes, among others. As an example, nanoscale delivery systems can be functionalized with PD-L1 monoclonal antibodies in anticancer therapies.

The targeting ability of nanoscale systems can benefit treatments for all disease classes, but this capability is of even greater importance in certain applications. Targeting of cancer cells is a major goal in oncology, as traditional systemic treatment result in severe side effects. The ability of nanocarriers to selectively attack targeted tumor cells allows for delivery of higher doses to improve efficacy while reducing cytotoxicity (1).

Therapeutic hyperthermia is a unique treatment made possible by use of nanoparticle delivery systems (1). Once the nanoparticles are localized in the tumor, they are exposed to laser radiation or an applied magnetic field, leading to an increase in temperature sufficient to kill the cancer cells. The improved targeting capability of nanoscale delivery systems also leads to improved activation of immune cells in cancer vaccines.

Unique surface properties and ligand functionalization provide real advantages.

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