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www.biopharminternational.com Next-Generation Biotherapeutics April 2025 BioPharm International ® 7 Innovation tory compliance, and long-term commercialization potential. She anticipates that the most promising therapies will demonstrate clear patient benefits, with transformative outcomes already observed in conditions such as sickle cell disease and hemato- logic malignancies. Franklin furthermore observes that the future of these therapies is intrinsically linked to collaborative efforts between drug developers, contract develop- ment and manufacturing organizations (CDMOs), and regulatory bodies. Meanwhile, FDA is exploring adaptive regulatory frameworks, including platform technology guidelines that may be able to better ac- commodate the rapid pace of innovation while main- taining rigorous safety standards, she explains. Serina highlights the fact that advance modali- ties such as ADCs and next-generation messenger RNA (mRNA) platforms—especially in oncology— are gaining significant traction. In particular, these innovative treatments are attracting investments and driving further research because of their high efficacy and ability to meet public health needs. It is this precision and efficacy that positions these mo- dalities at the forefront of the biopharmaceutical landscape, she remarks. Oncolog y and cardiovascular diseases are cur- rently major forces shaping the biopharmaceutical and pharmaceutical markets, and so significant in- novation is occurring in both dr ug discover y and in manufacturing processes to suppor t advanced treatments for these therapeutic markets, Serina also notes. Meanwhile, Tucker discusses how advancements in antibody design are becoming a defining feature of the biopharmaceutical industry. This is supported by the continually improving understanding that researchers are working toward in the areas of an- t ibody stabi l it y, i nteract ions, a nd f unct iona l it y. Progress in antibody t herapeutic development is suppor ted by increasingly sophisticated AI tools, which are helping to predict how antibodies will bi nd to ta rget molec u les. T h rough t he use of A I tools, the development process for new antibodies can be accelerated, and antibody-based therapies can be designed to be more efficient and precise. Looking to the future, Tucker observes that there is growing interest in making mRNA therapies more accessible and user-friendly—potentially through oral tablets or nasal sprays. Such deliver y formats would reduce the need for traditiona l injections, m a k i ng pre vent ive a nd t herapeut ic t reat ment s more convenient for the public. As technology ad- vances, developing stable, effective delivery systems for mRNA therapies could potentially revolutionize how vaccines and other therapies are administered in everyday healthcare, he asserts. ■ INTERPHEX 2025: The Challenge of Developing Ultra-Rare Disease Treatments 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," at INTERPHEX 2025, which was held in New York City from April 1–3. In their presentation, Katakowski and Portero discussed approaches to advancing treatments for ultra-rare diseases. PharmTech Group sat with Katakowski to learn more about the RTW Foundation and its work to advance the development of treatments for ultra-rare diseases as well as 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." About the speaker Joe Katakowski is director of Research at the RTW Foundation Watch Now! https://www.biopharminternational.com/view/ interphex-2025-the-challenge-of-developing-ultra- rare-disease-treatments SCAN TO WATCH