2025 AAPS National Biotechnology Conference round-up

28 May 2025

Written by Prathap Nagaraja Shastri (Johnson & Johnson)

Biopharmaceuticals Conference reports Drug development

The 2025 American Association of Pharmaceutical Scientists (AAPS) National Biotechnology Conference showcased cutting-edge science in biologics drug development, notably revolutionary advances in novel modalities. Held 4–7 May 2025, at The Westin Boston Seaport District (MA, USA), scientific sessions and posters highlighted breakthrough innovations in multispecific antibodies, CAR-T therapies, AAVs, and mRNA/siRNA therapeutics — treatments that are fundamentally transforming patient care across multiple therapeutic areas.

The following summary represents a small sample of the high-quality sessions that defined the 2025 program:

Opening Plenary: Translation triumphs: harnessing biotechnology to redefine the future of myeloma therapy
Delivered by Dr Nikhil C. Munshi (Harvard Medical School; MA, USA), the opening plenary provided a comprehensive overview of the current landscape and future potential in multiple myeloma treatments. Dr Munshi highlighted significant therapeutic accomplishments while identifying promising opportunities for continued advancement in this critical oncology area. He concluded his presentation with an inspiring quote from Adlai Stevenson stating, “It’s not the years in your life but the life in your years that counts” — a powerful reminder of the human impact driving biotechnology innovation.

Keynote: Delivering biotherapeutics to the CNS via different routes of administration
Dr Robert Thorne (Denali Therapeutics; CA, USA) addressed the critical challenge of delivering biotherapeutics to the central nervous system (CNS) for treating Alzheimer’s, Parkinson’s and other CNS disorders. He explored how physical and biochemical barriers, including the blood-brain barrier, limit the effectiveness of protein, oligonucleotide and gene therapies, despite their vast therapeutic potential. The presentation also highlighted current advances in alternative administration routes and emerging technologies that could overcome these delivery obstacles to unlock new treatment possibilities for devastating neurological diseases.

Hot Topic: Industry discussions on companion diagnostics for AAV-mediated gene therapy
This session, featuring Dr Yanmei Lu (Sangamo Therapeutics; CA, USA) and Dr Manuela Braun (Bayer; Leverkusen, Germany), offered their insights into a critical challenge in AAV-based gene therapies: managing pre-existing patient immunity that can compromise treatment effectiveness and trigger adverse reactions. The presentation and subsequent discussions addressed the complex regulatory landscape surrounding companion diagnostics for patient screening, proposing an innovative “Class IVD” framework that could streamline AAV serotype-specific diagnostic development.

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Hot Topic: AI/ML + FDA: best practices, themes & considerations for regulatory success
Dr Alex Cadotte (MCRA, an IQVIA business; Washington D.C., USA) and Dr Eric Luellen (Turing Biosciences; MA, USA) offered critical insights into FDA regulatory expectations for artificial intelligence and machine learning (AI/ML) applications. They provided practical guidance on best practices for engaging with the FDA on AI/ML products across drugs, diagnostics and devices, addressing a rapidly evolving regulatory landscape that pharmaceutical companies must navigate to successfully integrate AI technologies into their development programs.

Closing Plenary: Can financial engineering cure all diseases?
Delivered by Dr Andrew Lo (MIT Sloan School of Management; MA, USA), the closing plenary provided a venture capital perspective into the financial dynamics driving pharmaceutical drug development. Dr Lo explored how rare disease therapies are transforming patient lives by addressing life-threatening conditions, while also examining the economic incentives that encourage continued investment in rare disease development. He offered novel perspectives on how venture capitalists and scientists can collaborate to accelerate therapeutic innovation through strategic financial approaches.

The 2025 National Biotechnology Conference reinforced AAPS’s leadership in advancing pharmaceutical sciences while demonstrating unprecedented collaboration among industry, academia, and regulatory agencies in developing life-changing therapies.