Susan Haigney

INTERPHEX 2025: Challenges in Ultra-Rare Disease Treatment Development

At INTERPHEX 2025, which was held in New York City from April 1–3, Joe Katakowski, director of Research at the RTW Foundation, and Deanna Portero vice-president, Partnerships & Innovation, Orphan Therapeutics Accelerator, gave a presentation titled, “

A Novel Pathway for Ultra-Rare Disease Treatment Development, Commercialization and Access”,

 in which they discussed approaches to advancing treatments for ultra-rare diseases. PharmTech Group sat down with Katakowski to learn more about the RTW Foundation and its work to advance the development of treatments for ultra-rare diseases and the challenges faced by those involved in these efforts.

“[Ultra rare disease] is the most difficult space in which I've worked in preclinical drug development ever. [R]are disease foundations have challenges [to] getting access to the same resources and expertise that drug development companies would have; that's mainly due [to] their small patient population sizes,” Katakowski says. “Rare disease is defined as fewer than 200,000 individuals affected by a disorder in the [United States]. Ultra rare is [a] made-up term. I use that term to describe any rare disease that's too small to attract any commercial attention from biotech[s] or [the] pharmaceutical industry, and so there are really no prospects towards the traditional drug development avenues, such that the only way you can get a drug developed is if the parents form a foundation and become de facto drug developers themselves.”

The ultra-rare disease community must work withing the same regulatory structure despite not having the same access to resources as pharmaceutical companies and biotechs, according to Katakowski. “They can't seek the same investment opportunities from venture capital groups or other investment firms, so they have to self-fund these efforts through fundraising, through their own foundations. So, they have limited resources and finances at their disposal to develop these drugs, and they are sort of expected to develop them in the same regulatory structure and environment as other biotech and pharma developers,” he explains.

Click the video above to watch part one of PharmTech Group’s interview with Joe Katakowski.

Joe Katakowski is director of Research at the RTW Foundation.

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The Pharmaceutical Technology® Group sat down with Joe Katakowski, director of Research at the RTW Foundation about the challenges involved in the development of treatments for ultra-rare diseases. 

INTERPHEX 2025: Beta Bags and Sterile Manufacturing

Sterile manufacturing environments require that items be brought into the environment in a sterile manner. Traditionally, items would be taken through a transfer door into the sterile environment through a stainless-steel canister. Beta bags are a more flexible single-use method. To learn more about beta bags, the 

 Group spoke with Chris Gooding, general manager at CRL Solutions, during INTERPHEX 2025, which is being held in New York City from April 1–3.

According to Gooding, products that need to be transferred to a sterile environment come in different sizes that require different sterilization methods, which has resulted in the development of new methods for transfer. Beta bags can be made longer or for different sterilization methods, therefore, making them convenient.

The beta bag is really [designed] out of convenience for transferring either supplies, tools, or equipment into a sterile environment through an RTP [rapid transfer port] or a transfer door ... as opposed to the previous method, [which] would be a stainless-steel canister, but it was limited on the size and the depth and a number of different things,” Gooding says. “The bag allows it to be sized differently. It's much less expensive and is ultimately disposable after use.”

Click the video above to watch the interview.

Chris Gooding is the General Manager for Central Research Laboratories (CRL), headquartered in Red Wing, Minn. Chris has been the GM of CRL for five years. Prior to that, he worked in various leadership roles for many different manufacturing companies. CRL has 80 years of experience in the development of remote-handling systems, including telemanipulators, transfer systems, glove ports, and waste drum transfer systems. CRL's solutions promote operator safety and efficiency when performing various tasks in life sciences and nuclear applications around the world. For more information about CRL Solutions and its network of partners, visit 

The Pharmaceutical Technology® Group sat down with Chris Gooding, general manager at CRL Solutions, to learn more about how beta bags assist in the transfer of supplies and equipment into a sterile environment.

INTERPHEX 2025: Continuous and Controlled Freeze-Drying

Regulatory uncertainty has been a challenge to the implementation of continuous freeze-drying in the pharmaceutical industry, according to Thomas De Beer, co-founder and CEO of RheaVita. “The pharmaceutical industry is highly regulated, which makes it pretty difficult to innovate—[maybe] not difficult, but it makes it slow to innovate, especially new manufacturing technologies,” De Beer says in an interview with Pharmaceutical Technology® Group at INTERPHEX 2025. “And along with that, let's say, regulatory uncertainty. You also have the mindset of people within the pharmaceutical industry, they know that there is regulatory uncertainty to innovate, so their mindset is a bit oriented to that regulatory hesitance. However, on the other hand, there are also opportunities.”

The highly regulated nature of the pharma industry may make it difficult to innovate; however, regulators can often be the instigation for innovation. “They published several guidelines on process analytics and process analytical technology, but also on continuous manufacturing,” De Beer says. “And you see that this indeed stimulated the industry to innovate in pharmaceutical manufacturing, to leave the conventional batch wise manufacturing approach and to focus on continuous manufacturing. And at the same time, you see also new types of drug products coming up. And these new types of drug products, they become more and more complex. They require more flexible manufacturing. This also helps to stimulate innovation.”

RheaVita is exhibiting at Stand 2967 at INTERPHEX 2025, which is being held in New York City from April 1–3. De Beer's presentation, "

Revolutionizing Pharma: Continuous Manufacturing and GMP-Ready Freeze-Drying for Scaled Innovation

" is scheduled for Thursday, April 3 at 9:15AM.

Thomas De Beer, co-founder and CEO of RheaVita

Thomas De Beer graduated in pharmaceutical sciences in 2002 at the Ghent University in Belgium. He obtained his PhD at the same university in 2007. For his PhD research, he examined the suitability of Raman spectroscopy as a process analytical technology tool for pharmaceutical production processes. Within his PhD research period, he worked four months at University of Copenhagen in Denmark, Department of Pharmaceutics and Analytical Chemistry (Prof. Jukka Rantanen). After his PhD, he was an FWO-funded post-doctoral fellow at the Ghent University (2007-2010). Within his post-doc mandate, he worked nine months at the Department of Pharmacy, Pharmaceutical Technology and Biopharmaceutics from the Ludwig-Maximilians-University in Munich, Germany (Prof. Winter and Prof. Frieβ). In February 2010, he became professor in Process Analytics & Technology at the Faculty of Pharmaceutical Sciences from the university of Ghent. His research goals include bringing innovation pharmaceutical production processes (freeze-drying, hot-melt extrusion, continuous from-powder-to-tablet processing, etc.). More specifically, De Beer contributes to the development of continuous manufacturing processes for drug products such as solids, semi-solids, liquids and biologicals (continuous freeze-drying of unit doses). De Beer is also director of Ghent University’s Center of Excellence in Sustainable Pharmaceutical Engineering (CESPE) which was founded in 2016. In 2018, De Beer became co-founder and CEO at RheaVita, which provides a continuous freeze-drying technology for the pharmaceutical market.

Pharmaceutical Technology® Group sat down with Thomas De Beer, co-founder and CEO of RheaVita, to talk about his presentation at INTERPHEX 2025, “GMP-ready Continuous & Controlled Pharmaceutical Freeze-Drying”.

INTERPHEX 2025: AI and Automation in Pharma

Automation is being utilized in the areas of quality, throughput, and operator autonomy within the pharmaceutical industry, according to Peter Sarvey, head of Sales for Automation NTH, and the cost of using automation has decreased in the past two years. Meanwhile, the accuracy of these tools is increasing.

The use of artificial intelligence (AI), which has been used in the “vision side” of automation according to Sarvey, is also increasingly starting to be used in the inspection side of manufacturing.

“When we have a lot of part variation, we're not just programming pick and place, or motion based on preset points. So, we move to an area, and then we use the AI vision to tell us, where is that exact part or how much has that part changed? So, it actually allows the automation to move more seamlessly,” Sarvey says. “And then the other side of that is on the inspection side of things. There are a lot of inspections that previously were done manually, because it's not a simple pattern match for if this medical device or this pharmaceutical component is a reject. Now we can teach the AI what a good part looks like, what a bad part looks like, and then over time, shift that area too, all in the nature of increasing throughput and decreasing yield loss.”

® Group spoke with Peter Sarvey at INTERPHEX 2025, which is being held in New York City from April 1–3. Automation NTH can be found at Stand 3061.

Peter Sarvey is Head of Sales for Automation NTH, a specialist in semi-automated and fully automated solutions for a broad array of manufacturing applications, including pharmaceuticals and medical device production. With a focus on next-gen robotics and controls, the company’s services include custom automation, equipment optimization and automation consulting. 

Pharmaceutical Technology® Group spoke with Peter Sarvey, head of Sales for Automation NTH, at INTERPHEX 2025 about the growing use of automation and artificial intelligence in the pharmaceutical industry.

INTERPHEX 2025: Automation for Protein Manufacturing

Biomanufacturing involves complex operations and thousands of skilled workers, says Kerry Love, co-founder and CEO of Sunflower Therapeutics, a biotech company specializing in continuous and fully integrated equipment for the production of proteins. Automation can be a way to build quality and reduce human errors, while also allowing workers who might not be as skilled to participate in high-value manufacturing.

“When you use automation in general, you kind of make something that's usually highly complex, with a lot of handoffs and transitions, much less complicated, particularly in continuous bioprocessing," Love says in the interview. "That's actually a great place for automation, because already you're simplifying unit operations and making interfaces between unit operations that oftentimes haven't been there in the past. That's a great way to then layer automation on top of that and ultimately get to walk away solutions like those that we build at Sunflower.”

® Group spoke with Love at INTERPHEX 2025, being held April 1–3 in New York City. Sunflower Therapeutics can be found at Stand 1273.

Kerry Love, co-founder and CEO of Sunflower Therapeutics

Kerry Love, PhD, is the co-founder and CEO of Sunflower Therapeutics, a women-owned and led biotechnology company delivering next-generation protein manufacturing solutions that anyone can use to create innovative new medicines, vaccines, foods, and other bio-produced materials. Love is an organic chemist by training, performing her doctoral studies at MIT, and a biotech entrepreneur at heart, having founded two companies and contributed to the starting of many more over the past 20 years.Love has authored more than 40 scientific papers and patents.

The Pharmaceutical Technology® Group spoke with Kerry Love, co-founder and CEO of Sunflower Therapeutics at INTERPHEX 2025, to find out how automation enhances the manufacture of proteins.

FDA announced on April 1, 2025 that Martin A. Makary, MD, has been sworn in as the agency’s new commission after a bipartisan confirmation by the US Senate. Makary, a graduate of Bucknell University, Thomas Jefferson University, and the Harvard School of Public Health, completed his residency at Georgetown University and surgical oncology fellowship at Johns Hopkins.

He founded the Johns Hopkins Center for Surgical Trials and Outcomes Research and led cross-disciplinary research on cancer, obesity, Alzheimer’s, adverse event monitoring, and antimicrobial resistance. He is a member of the National Academy of Medicine and co-developer of the Surgery Checklist.

“I am honored and humbled to assume this role at the FDA under the leadership of President Trump and Secretary Kennedy,” said FDA Commissioner Martin A. Makary, in the press release (1). “I look forward to working with this Administration and the FDA workforce to advance our shared goals in meeting the agency’s public health mission. As Commissioner, I hope to ensure that the FDA holds to the gold standard of trusted science, transparency, and common sense so that we can Make America Healthy Again.”

“I am pleased to welcome Dr. Makary to the U.S. Department of Health and Human Services and the Food and Drug Administration,” said HHS Secretary Robert F. Kennedy, Jr., in the release. “He is a national leader in medicine with impeccable credentials. His extensive research, clinical experience, and national leadership make him uniquely qualified to lead the FDA as we work together to Make America Healthy Again.”

Back in January 2025, FDA leaders, Commissioner Robert Califf and the Director of the Center for Drug Evaluation and Research, Patrizia Cavazzoni, resigned their posts (2–4). And in March 2025, Peter Marks, director of the Center for Biologics Evaluation and Research resigned (5).

Califf warned of misinformation on his way out of office. "We are currently losing the battle due to the intersection of social media and cultural changes, which threatens the foundation of public health," Califf said. “A lot of people died who would not have died if they had gotten a free vaccine and not been misled [about the safety of vaccines]” (3).

The concern over misinformation was also on the mind of Marks, who stated the following in his resignation letter (6):

“Over the past 13 years, I have done my best to ensure that we efficiently and effectively applied the best available science to benefit public health,” Marks said. “As you are aware, I was willing to work to address the Secretary's concerns regarding vaccine safety and transparency by hearing from the public and implementing a variety of different public meetings and engagements with the National Academy of Sciences, Engineering, and Medicine. However, it has become clear that truth and transparency are not desired by the Secretary, but rather he wishes subservient confirmation of his misinformation and lies.”

1. FDA. Martin A. Makary, M.D., M.P.H., Sworn in as FDA Commissioner. Press Release. April 1, 2025.
2. Herper, M. Leaving FDA, Califf is Unapologetic—and Warns of Staff Departures. 

. Jan. 8, 2025.
3. Eglovitch, J. S. Goodbye Message: Califf Says FDA Needs Help in Battling Misinformation, Has Deep Talent Pool to Mitigate Departures. 

. Jan. 17, 2025.
4. Haigney, S. Cavazzoni Retires as CDER Director. 

https://www.pharmtech.com/view/cavazzoni-retires-as-cder-director


5. Haigney, S. FDA CBER Director Peter Marks Resigns. 

. March 31, 2025.
6. Marks, P. Letter to Sara Brenner, MD, MPH Acting Commissioner of Food and Drugs U.S. Food and Drug Administration. March 28, 2025. Posted by 

Articles

Martin A. Makary, confirmed by a bipartisan vote of the US Senate, has been sworn in as FDA’s new commissioner.