Issue link: https://www.e-digitaleditions.com/i/1534558
6 BioPharm International ® Next-Generation Biotherapeutics April 2025 www.biopharminternational.com Innovation Meanwhile, Mar yland Frank lin, PhD, vice-pres- ident and enterprise head, Cell and Gene Therapy, Labcor p, emphasizes the significant contribution of cell and gene therapies (CGTs) to the transforma- tion of the biopharma R&D landscape. The entrance of the first CGTs on the market were characterized by groundbreaking scientific and technological ad- vancements, which continue in the ongoing devel- opment of new CGTs. "Most of the cell and gene therapy products being developed today are highly innovative compared to traditional therapeutics. You're using live cells and/or adding, deleting, or even correcting genetic material, and this is so different from small molecules or even traditional monoclonal antibodies," Franklin states. Franklin points to two particularly promising ap- proaches in CGT that are emerging in clinical develop- ment: in-vivo chimeric antigen receptor T-cell (CAR-T) delivery and advanced gene editing strategies. She emphasizes that these technologies aim to address critical challenges in safety and manufacturing com- plexity that have historically limited CGT progress. "Two of the most new and innovative approaches very early in clinical development are in-vivo CAR-T delivery, and some of the newer gene editing strate- gies that improve on limitations with CRISPR/Cas9 [clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9] systems, [such as] base editing and prime editing," says Franklin. "In both instances, we're seeing the industr y work on solutions to address safet y concerns and sub- stantial complexities and challenges that we face in manufacturing these products." Innovation drivers Lidia Serina, PhD, head of Development Services at Simtra BioPharma Solutions, highlights a few key drivers of innovation in new modality development for biopha r maceut ica ls. T hese d r ivers i nclude a strong interest by industr y for targeted molecular delivery mechanisms coupled with increased invest- ment in precision therapeutics. In addition, advanced technologies such as artificial intelligence (AI) and digitalization technologies are pushing the drug dis- covery process to reach the next generation of these therapeutics. Also, current market dynamics and the regulatory support environment are favorable toward development of next-generation therapeutics. "Recently, FDA has, I would say, accelerated the approvals of next-generation ADCs that are more se- lective, that decrease the level of toxicity. So, there is a real potential on the market; we see that from the market demand," Serina states. Sean Tucker, PhD, founder and chief scientific of- ficer of Vaxart, concurs that the current landscape of biopharma development is characterized by a drive toward more targeted t herapeutics wit h reduced side effects and innovative deliver y mechanisms. From h is perspective and experience work ing in the immunology field, Tucker notes that emerging technologies are transforming vaccine and drug de- velopment, with a particular focus on convenience and mucosal site targeting. Tucker points to how AI is playing a critical role in accelerating drug discovery and design processes. For nucleic acid-based vaccines, for example, AI en- ables rapid optimization of genetic sequences, which allows researchers to design complex antigens more efficiently. Although experimental validation re- ma i ns essent ia l, A I ca n sign i f ica nt ly reduce t he time required for initial design stages, Tucker says. In addition to vaccines, antibody development continues to be a pivotal area of innovation. This is another area where AI tools enhance the under- standing of molecular interactions, stability, and de- sign. Tucker anticipates that the future of biophar- maceuticals lies not just in creating new molecules, but in developing advanced delivery platforms that improve patient accessibility. "I think [where] the future lies is not [in] basically being able to deliver these … molecules themselves [such a s messenger R NA], but potent ia l ly usi ng [them] for the next modalities, where you're trying to figure out not just how to make the [molecule], but how to package it in a way that It can be used in a tablet or a spray, or something that's a lot more convenient," Tucker says. Strategic portfolio development Dondarski f ur ther notes that the most successf ul biopharmaceutica l organ izations w i l l not neces- sarily focus on a single modality but rather develop diverse, adaptable portfolios. He explains that the key strategic imperative is maintaining a compre- hensive pipeline that spans multiple disease areas a nd le verages va r iou s tech nolog ica l pl at for m s. Using this approach has a better chance of mitigat- ing the inherent serendipity in biopharmaceutical research while maximizing potential therapeutic breakthroughs. The biopharmaceutical industr y stands at a piv- otal moment, Dondarski explains, which is charac- terized by technological diversity, sophisticated mo- dalities, and increasingly personalized therapeutic approaches, and he anticipates that success will be defined not by adherence to traditional paradigms, but by organizational f lexibility, technological inte- gration, and strategic portfolio management. Franklin agrees, also noting that success in this domain will depend on comprehensive development strategies that consider not just scientific innova- tion, but a lso manufact uring sca labi lit y, reg ula- Innovation