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

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

 was released during CPHI Barcelona. This report indicates a rise in biotech funding and subsequent growth for pharma contract services. This year’s CPHI Barcelona reportedly sees the largest gathering of contract development and manufacturing organizations (CDMOs).

Analysis on the report was given by CPHI Report expert Brian Scanlan of Edgewater Capital. According to Scanlan, liquidity in the biotech and mid-sized pharma market is typically a proxy of growth for the contract research organization (CRO)/CDMO sector. Over the past two quarters, funding into the sector has seemingly stabilized at levels last seen right before the pandemic. Total funding in 2023 is trending ahead of 2022, with the exception of initial public offerings (IPOs), which remain weak.

Over the past few years, the spike in market saturation combined with decreased funding led to CDMOs slowing development pipelines. But Scanlan predicts that the market is turning. “There are signs of an improving VC [venture capital] funding environment, but this needs to coincide with increasing pharma M&A and a healthier IPO environment,” he wrote. “We believe softer demand, particularly from emerging pharma and in earlier phases of development, will extend for a period of 12–18 months. However, the investment banking community is already signaling a pickup on deal activity starting in late 2023 and accelerating into 2024.”

The noise around onshoring has slowed, as there are now two competing forces at work. The forces that initially ignited the supply chain re-alignment surge such as COVID-19, geopolitical tensions, and risk mitigation have been partially offset by calming geopolitical tensions, decreasing supply chain disruptions, and renewed focus on cash management (prompted by funding challenges).

Scanlan stated that an important (and positive) factor for CDMOs is the workforce shedding in the industry over 2022 and the early part of 2023, which he predicts will bolster demand for CRO/CDMOs in 2024. He concluded that the long-term fundamentals for CRO/CDMOs are very good, especially given the number of compounds in all phases of development and market dynamics that favor increases in outsourced penetration rates.

Articles

The report indicates a rise in biotech funding and subsequent growth for pharma contract services.

CPHI Annual Report: VC Capital Investment and Contract Services Growth in 2024

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

FDA announced on Oct. 19, 2023 that it had issued the final guidance, 

Development and Licensure of Vaccines to Prevent COVID-19

, to help sponsors with the clinical development and licensure of COVID-19 vaccines. The document does not address every consideration for satisfying requirements because some vaccines may need specific data, according to the agency. The final guidance reflects FDA’s current thinking on the issue.

“Given the need to ensure that sponsors are aware of our current recommendations to facilitate timely development of vaccines to prevent COVID-19, this guidance is being implemented without prior public comment because FDA has determined that prior public participation for this guidance is not feasible or appropriate (see section 701(h)(1)(C) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), (21 U.S.C. 371(h)(1)(C)), and 21 CFR 10.115(g)(2)). This guidance document is being implemented immediately, but it remains subject to comment in accordance with the Agency’s good guidance practices,” the agency stated in the document.

The final guidance explains key considerations for chemistry, manufacturing, and controls including facilities and inspections; nonclinical data, such as toxicity studies; clinical trials, such as trial populations and trial design; post-licensure safety evaluations; and diagnostic and serological assays. In addition, the guidance discusses other safety considerations that should be addressed including demonstrating effectiveness, surrogate endpoints, and adequate datasets. “If it is no longer possible to demonstrate vaccine effectiveness by way of conducting clinical disease endpoint efficacy studies, the use of a controlled human infection model to obtain evidence to support vaccine efficacy may be considered. However, many issues, including logistical, human subject protection, ethical, and scientific issues, would need to be satisfactorily addressed,” FDA stated in the guidance.

The final guidance document was issued to assist bio/pharma companies in the clinical development and licensure of COVID-19 vaccines. 

FDA Finalizes Guidance on Development of COVID-19 Vaccines

Communication between the manufacturing plant and retail stores. | Image Credit: © Cagkan - © Cagkan - stock.adobe.com

Bettersize US announced on Oct. 22, 2023 that it is showcasing three of its instruments—the BeNano Series, Bettersizer 2600, and BetterPyc 380—at the AAPS 2023 PharmSci 360 event. The company is also highlighting its FLOW MODE coupled with high-performance liquid chromatography (HPLC), “which enables high-resolution nanoparticle size testing tailored to the precise needs of pharmaceutical professionals,” according to the company.

The BeNano Series features nanoparticle size and zeta potential analysis by three light scattering techniques. The Bettersizer 2600 offers particle size analysis by laser diffraction, with wet/dry dispersions, and the BetterPyc 380 features powder analysis by gas displacement method.

Bettersize Instruments is a provider of analytical instruments for material characterization, comprehensive solutions, and exceptional customer service. In August 2023, the company opened a new facility in Costa Mesa, Calif. to support North American material scientists, researchers, and engineers.

Visit Bettersize at PharmSci 360 at booth number 2135.

Bettersize US showcases their BeNano Series, Bettersizer 2600, and BetterPyc 380 instruments at the AAPS 2023 PharmSci 360 event. 

Bettersize Unveils New Capabilities at AAPS 2023 PharmSci 360

Feliza Mirasol is the science editor for 

On Oct. 19, 2023, Waters Corporation announced that it has launched OligoWorks solid phase extraction (SPE) Kits and components, which are meant to improve sample preparation for liquid chromatography (LC)–mass spectrometry (MS)-based bioanalytical quantitation of therapeutic oligonucleotides.

The kits are designed to deliver up to 2x higher oligonucleotide recovery from biofluids, according to a company press release. The kits’ design also delivers improved LC­MS sensitivity and reproducibility for supporting robust quantitation. Because these capabilities enable lower limits of quantitation, the kits can reduce the need for repeat experiments.

The kits also use an innovative, enzyme-enhanced, and detergent-free sample pre-treatment that, according to Waters in the press release, reduces washing steps and improves mass spectrometer compatibility. This design enables for direct injection LC–MS analysis and is also what allows for the lower limits of quantitation. The kits can also be fully automated, which can improve productivity or simply help scientists gain back time via the reduction of manual steps and oversight.

The OligoWorks SPE Kits are available immediately and complement other Waters tools, including MaxPeak Premier Columns, ACQUITY Premier LC System and Arc Premier LC System, Andrew+ Pipetting Robot. The new kits also support the sensitivity of the Waters Xevo TQ Absolute Mass Spectrometer.

" … Waters is committed to solving customer pain points and enabling greater analytical performance and productivity. Our new OligoWorks SPE Kits directly address oligonucleotide bioanalysis sample prep challenges across diverse oligo therapeutics," said Erin Chambers, vice-president, Consumables and Lab Automation, Waters Corporation. "The science and societal effects of these therapies are exploding given their ability to address human health issues. Our workflow and kits will drive an acceleration of oligo bioanalysis projects and drug development timelines; ultimately improving the experience for drug candidates."

Waters has launched OligoWorks SPE Workflow and Kits, which is aimed at accelerating the bioanalysis of oligonucleotides.

Waters Launches New Oligonucleotide Bioanalysis Kits

Science laboratory test tubes, laboratory equipment | Image Credit: © BillionPhotos.com - © BillionPhotos.com - stock.adobe.com

On Oct. 17, 2023, Lisata Therapeutics announced that the European Medicines Agency (EMA) granted orphan drug designation to LSTA1, Lisata’s lead product candidate for pancreatic cancer treatment. As of the publication of this article, LSTA1 has several ongoing and planned clinical studies conducted globally. These studies use a variety of solid tumor types, including pancreatic cancer, with a range of anti-cancer regimens.

LSTA1 is an investigational drug intended to aid co-administered or tethered anti-cancer drugs to more effectively penetrate solid tumors by activating a novel uptake pathway. LSTA1 activates the pathway in a tumor-specific way, potentially modifying the tumor microenvironment and making tumors more venerable to immunotherapies. There is significant non-clinical data showing LSTA1 enhancing delivery of a variety of existing and emerging anti-cancer therapies, including immunotherapies, chemotherapeutics, and RNA-based therapeutics.

The orphan drug designation is given to drugs and biologics that treat rare disease, which affect no greater than five in 10,000 people in the European Union. The designation is based on a positive opinion from the EMA Committee for Orphan Medicinal Products. The companies of eligible products receive EU marketing exclusivity for 10 years after approval, possible fee reductions, and protocol assistance from EMA.

“To date, LSTA1 has demonstrated favorable safety, tolerability, and activity to enhance delivery of standard-of-care chemotherapy for patients with metastatic pancreatic cancer,” said Kristen K. Buck, M.D., executive vice president of R&D and Chief Medical Officer of Lisata, in a press release. “Obtaining orphan drug designation from the EMA reinforces our belief that LSTA1 offers major improvement in treating patients with this terrible disease. We are excited by the promise of LSTA1 for the treatment of pancreatic cancer and other solid tumors and are committed to advancing our development programs with the goal of providing a benefit to patients.”

The designation will grant Lisata EU marketing exclusivity for 10 years after approval and protocol assistance from EMA.

Lisata Therapeutics Announces EMA LSTA1 Orphan Drug Designation for Pancreatic Cancer Treatment

TOP NEWS on grunge world map | Image Credit: © Sean K - stock.adobe.com

Royalty Pharma announced on Oct. 19, 2023 that it has signed an agreement with PTC Therapeutics to acquire additional royalties on Roche’s Evrysdi. Royalty will pay $1.5 billion upfront, with the possibility of purchasing the remaining royalties for up to $500 million at a later date. Evrysdi, which is marketed by Roche, is an “orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy [SMA].” It received FDA approval in 2020 and has since treated more than 11,000 patients internationally. In 2022, Evrysdi generated approximately $1.2 billion in sales, 87% higher than in 2021.

As of the publication of this article, Roche’s royalty payment on Evrysdi is tiered, ranging from 8% to 16%. Following this transaction, Royalty Pharma’s ownership of the Evrysdi royalty is increasing from 43% to 81%. Until Dec. 31, 2025, PTC will have the option to sell its remaining Evrysdi royalties to Royalty Pharma for $500 million in five equal tranches. If PTC uses less than three of these options, Royalty Pharma will have the opportunity until March 31, 2026 to purchase 50% of the remaining royalties for $250 million.

“We are excited to acquire an additional royalty interest in Evrysdi, a convenient, oral therapy that has transformed the lives of SMA patients worldwide,” said Pablo Legorreta, founder and chief executive officer of Royalty Pharma in a Royalty Pharma press release. “This is our second transaction with PTC, which builds on our longstanding partnership, and highlights our ability to structure creative, win-win funding solutions for our partners. Further, our unmatched scale and deep knowledge of SMA positioned us to provide PTC with substantial capital in a timely fashion, another unique aspect of our business model. This transaction is expected to further enhance Royalty Pharma’s long-term growth and diversification. We remain incredibly excited for our future given our robust transaction pipeline.”

The spinal muscular atrophy treatment, currently marketed by Roche, generated $1.2 billion in sales in 2022.

Royalty Pharma to Purchase up to $1.5 Billion of PTC Therapeutics’ Royalty on Roche’s Evrysdi

The trend toward digital transformation in biopharmaceutical manufacturing continues as advanced technologies enter the scene and work their way through to manufacturing operations. With the availability of technologies to take automated biomanufacturing from preclinical to commercial-scale, the industry may start to see a wave of changeovers from manual processes to an automated, digital environment. This episode of the 

Podcast will discuss the realities of switching over from manual to digital, and what to expect.

Ajit Yeole, senior director, Life Sciences Manufacturing, Cognizant

Ajit Yeole is the senior director for Life Sciences Manufacturing at Cognizant, based in London, UK.He is responsible for strategy, smart manufacturing, developing new service offerings, and digital accelerators targeted towards addressing the industry needs. Has been closely working on engagements around manufacturing and quality involving emerging technologies (Industrial IoT, advanced analytics, AR/VR , and others) using agile and design-thinking-led approaches. He has led the manufacturing operations management practice for Cognizant in Europe and has delivered large manufacturing programs while collaboratively building tools and templates for consulting offerings. Previously, he worked in manufacturing operations management, IoT (M2M) and OT/ industrial automation as a consultant, solutions architect, and business unit head.

Laks Pernenkil, principal and practice leader, US Life Sciences Product & Supply Operations, Deloitte

Laks Pernenkil, PhD, is a principal in Deloitte’s Life Sciences Product, Supply, Manufacturing and Quality Operations practice based in McLean, Va. He has more than 15 years of consulting and technical operations experience in pharmaceutical, biologics and medical device sectors. At Deloitte, his experience includes smart/digital manufacturing and plant floor operations, plant engineering, facilities management, material sourcing, distribution, performance improvement, operational excellence, and market entry operations strategy.

 presents the Drug Solutions podcast, where the editors will chat with industry experts from across the pharmaceutical and biopharmaceutical supply chain. Join us as experts share insights into your biggest questions—from the technologies, to strategies, to regulations related to the development and manufacture of drug products.

Podcasts

In this episode of the Drug Solutions Podcast, industry experts discuss the challenges and advantages of the digitalization trend in the biopharma industry, with particular emphasis on the impacts to biomanufacturing.

Drug Solutions Podcast: Digital Transformation in the Biopharma Industry

On Oct. 18, 2023, SCHOTT announced that it has launched a new component for biopharmaceutical process analysis, the ViewCell flow cell. The purpose of this new component is to optimize downstream processes in the biopharmaceutical industry by enabling safe, continuous process control directly in the production line.

The introduction of the ViewCell flow cell follows the company’s launch of its ViewPort sensor port for bioreactors. Through a sterile-safe optical window, the flow cell allows for 

 measurements of biological and physical parameters in downstream processing. The flow cell can be used during purification and filtration as well as during subsequent formulation. This capability thus reduces the need for both physical sampling and storage tanks, according to a company press release.

According to the press release, optical sensors or spectrometers are connected to the flow cell for monitoring, which means that the measuring instruments do not come into contact with the medium, and thus do not penetrate the sterile barrier. This allows for the sensors or spectrometers to be easily replaced during ongoing production, which the company states in its press release is a major advancement compared to fixed probes. In addition, a glass-to-metal connection allows for a sterile-safe design, which also increases the safety and efficiency of process analysis, contributing to an overall optimized yield.

The company stated in the press release that ViewCell allows biomanufacturers to quickly build and test small-scale flow systems for Raman analysis, which are used to mimic real-world industrial processes. The compact design of the device reduces the overall volume of sample material required, which cuts down on waste and costs—key considerations in the handling of expensive or difficult-to-obtain test materials.

SCHOTT now offers ViewCell, a new component meant to add flexibility to biopharmaceutical process control.

SCHOTT Launches ViewCell Flow Cell for Biopharma Process Control

Key highlights from the CPHI Pharma Survey were announced as of Oct. 13, 2023, and the full findings will be published at CPHI Barcelona as part of the CPHI Annual Report. These results will provide insights from more than 250 global pharma executives, including discoveries such as pharma confidence dipping below the record scores of 2022, although it has remained extremely resilient despite wider macro uncertainly, according to the press release.

Further, the CPHI Pharma Index reported its second highest ever total, which was 2.5% down on the 2022 all-time high. These results highlight the medium-term trajectory of the industry and the strength of market fundamentals. The United States remained the top overall ranked nation, with India also continuing to make year-on-year inroads for significant growth in 2024. India did, however, remain at the top of the “growth” category for the second year in a row, scoring 7.8 out of 10. It also saw improvements in the score for “biologics quality” and finished behind the United States for “overall competitiveness”. According to the report, India is predicted to undergo a fast transformation from a generics hub to an innovation-focused pharma economy, driven by the incremental steps of large contract development and manufacturing organizations.

One of the more startling discoveries, according to the press release, is that the report predicts that an “India plus one” strategy will be adopted in the next five years. “When you look collectively at the data points in the Annual Survey, you see that after a reset from record scores in 2022—when many reported record profits—the industry is now well positioned for growth in 2024,” said Tara Dougal, Content Director–Pharma, in a press release.

The report also identified that many of the industry’s growth drivers that were seen in 2019 are still present but are now supplemented by the breakthroughs in microbiome therapeutics, while artificial intelligence now looks like the next efficiency used in the industry over the next five years.

“Our analysts predict outsourcing will continue its growth trajectory in 2024 and beyond—which is obviously a tremendously positive message for the industry as we approach CPHI Barcelona,” Dougal said in the press release. “It is also why we have ensured that ‘outsourcing strategies’ are a central theme of this year’s content, and the overall takeaway is of quiet and confident optimism. In fact, this will remain an ongoing theme for CPHI content, helping our audience to meet their objectives around partnering and supplier selection year-round.”

These results will provide insights from more than 250 global pharma executives, including discoveries such as pharma confidence dipping below the record scores of 2022, although it has remained extremely resilient despite wider macro uncertainly, according to the press release.

CPHI Annual Survey Finds Global Pharma Resilient with India the Chief Beneficiary of Macro Changes

On Oct. 17, 2023, SpliceBio, a genetic medicines company headquartered in Spain, announced that it has signed an exclusive collaboration and licensing agreement with Spark Therapeutics under which SpliceBio will use its proprietary protein splicing platform to develop a gene therapy for treating an inherited retinal disease. Details about the disease target were not disclosed.

Under the agreement, both companies will conduct a research collaboration utilizing SpliceBio’s technology platform. The protein splicing platform offers the potential to address diseases that currently cannot be treated with gene therapies. In those cases, the gene required for the treatment is too large to be delivered by adeno-associated virus (AAV) vectors. With this agreement, Spark will have exclusive worldwide rights to develop, manufacture, and commercialize a gene therapy resulting from this research collaboration; the gene therapy will target an undisclosed inherited retinal disease, according to a company press release. The agreement makes SpliceBio eligible to receive upfront, opt-in, and milestone payments of up to $216 million as well as royalties on net sales.

“This research collaboration and license agreement is an exciting opportunity to develop a novel gene therapy in an area of high unmet medical need. We are proud that Spark Therapeutics recognizes the potential of our pioneering [p]rotein [s]plicing platform and the profound impact it could have in the treatment of inherited retinal diseases that are unable to be effectively addressed by other gene therapy approaches,” said Miquel Vila-Perelló, CEO and co-founder of SpliceBio, in the press release. “In addition to the Spark collaboration, we continue to develop our lead program in Stargardt disease and further build our capabilities and pipeline of wholly owned gene therapy programs to develop life-changing therapies for patients in need.”

“This partnership builds on Spark’s leadership in gene therapies for inherited retinal diseases. Our breakthrough gene therapy LUXTURNA [voretigene neparvovec-rzyl] demonstrated how we can change the lives of patients with biallelic mutations in the RPE65 gene whose physicians have determined their eligibility for treatment. At the same time, there are many more people with other inherited retinal diseases that need treatment options,” said Federico Mingozzi, chief science and technology officer of Spark Therapeutics, in the release. “With our complementary capabilities, combined deep technical knowledge, and SpliceBio’s impressive protein splicing platform we hope to make further progress in the treatment of inherited retinal diseases, bringing new gene therapies into the clinic and to commercial availability.”

Spark Therapeutics and SpliceBio have formed a collaboration to develop a gene therapy that can treat an inherited retinal disease.

Featured Content

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

Behind the Headlines Episode 28

Ask the Expert

Drug Digest December 2025

A “Plan, Prevent, Prove” Approach